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Hot Topics of the Day are picked by experts to capture the latest information and publications on public health genomics and precision health for various diseases and health topics. Sources include published scientific literature, reviews, blogs and popular press articles.

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404 hot topic(s) found with the query "Genome editing"

Sickle Cell Disease Approvals Include First CRISPR Gene Editing Therapy
E Harris, JAMA, January 3, 2024 (Posted: Jan 03, 2024 2PM)

From the article: "The US Food and Drug Administration (FDA) recently greenlit 2 cell-based gene treatments for sickle cell anemia, including the first therapy involving the genome editing technology known as CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats–CRISPER-associated protein 9). Both treatments are approved for people aged 12 years or older and work by modifying people’s own blood stem cells and then transplanting them into their bodies via a single-dose infusion. They change blood cells in different ways, though. "

How CRISPR gene editing could help treat Alzheimer’s
T Thompson, Nature, December 11, 2023 (Posted: Dec 12, 2023 9AM)

From the article: "Last month saw the first-ever approval of a gene therapy that uses the CRISPR–Cas9 gene-editing tool, a treatment for the blood conditions sickle-cell disease and ß-thalassaemia that works by precisely cutting out a faulty gene in people’s stem cells. Now, researchers in search of new treatments for Alzheimer’s disease are hoping to deploy similar strategies against forms of the disease that are caused by genetic mutations."

In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease
A Feurstein, StatNews, December 8, 2023 (Posted: Dec 08, 2023 2PM)

From the article: "The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine, called Casgevy,'s authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases. "

CRISPR 2.0: a new wave of gene editors heads for clinical trials Landmark approval of the first CRISPR therapy paves the way for treatments based on more efficient and more precise genome editors.
H Ledford, Nature, December 7, 2023 (Posted: Dec 08, 2023 2PM)

From the article: "Less than a month after the world’s first approval of a CRISPR-Cas9 genome-editing therapy, researchers are hoping that the therapy will win its second authorization this week — this time from the United States, with its famously stringent regulators and lucrative health-care market. The therapy, which UK regulators approved on 16 November, disables a gene as a means of treating a genetic blood disorder called sickle cell disease. A host of other CRISPR-Cas9 therapies that work on the same principle are in clinical trials as treatments for a range of diseases. "

A new age of precision gene therapy.
Axel Schambach et al. Lancet 2023 11 (Posted: Nov 29, 2023 9AM)

From the abstract: "Gene therapy has become a clinical reality as market-approved advanced therapy medicinal products for the treatment of distinct monogenetic diseases and B-cell malignancies. This Therapeutic Review aims to explain how progress in genome editing technologies offers the possibility to expand both therapeutic options and the types of diseases that will become treatable. "

'Treasure trove' of new CRISPR systems holds promise for genome editing.
Sara Reardon et al. Nature 2023 11 (Posted: Nov 27, 2023 10AM)

From the paper: "CRISPR–Cas9 is best known as a laboratory tool for editing DNA, but its natural function is as part of the immune system that helps certain microorganisms to fight off viruses. Now, researchers have used an algorithm to sort through millions of genomes to find new, rare types of CRISPR system that could eventually be adapted into genome-editing tools. "

UK first to approve CRISPR treatment for diseases: what you need to know
C Wong. Nature. November 16, 2023 (Posted: Nov 17, 2023 8AM)

From the article: "In a world first, the UK medicines regulator has approved a therapy that uses CRISPR gene editing as a treatment for diseases. The decision marks another high point for a biotechnology that has regularly been lauded as revolutionary in the decade since its discovery. The therapy will treat the blood conditions sickle-cell disease and ß-thalassaemia. Sickle-cell disease, also known as sickle-cell anaemia, can cause debilitating pain, and people with ß-thalassaemia can require regular blood transfusion. "

First trial of 'base editing' in humans lowers cholesterol - but raises safety concerns.
Miryam Naddaf et al. Nature 2023 11 (Posted: Nov 14, 2023 9AM)

From the paper: "The first trial in humans of the precise gene-editing technique known as base editing has shown promising results for keeping cholesterol levels in check in patients with familial hypercholesterolemia. The approach injects into people a treatment called VERVE-101, which permanently deactivates a gene in the liver called PCSK9. That gene controls the level of low-density lipoprotein (LDL), or ‘bad’ cholesterol — a key contributor to heart disease. But the findings have also drawn criticism. Two serious adverse events in the trial, including a death, have raised safety concerns. "

Gene therapies for rare diseases are under threat. Scientists hope to save them- As industry steps aside, scientists seek innovative ways to make sure expensive treatments can reach people who need them.
H Ledford, Nature, October 6, 2023 (Posted: Oct 06, 2023 7AM)

From the article: "In the past two years, two gene therapies have been withdrawn from the European market for business reasons after earning regulators’ approval. Concern is mounting that other gene therapies for rare diseases will meet a similar fate, as might upcoming treatments that rely on the related technique of genome editing, which makes targeted DNA changes. "

Super-precise CRISPR tool enters US clinical trials for the first time.
Heidi Ledford et al. Nature 2023 9 (Posted: Sep 20, 2023 7AM)

From the article: "A high-precision successor to CRISPR genome editing has reached a milestone: the technique, called base editing, has made its US debut in a clinical trial. The trial tests more complex genome edits than those performed in humans to date. Trial organizers announced that the first participant had been treated using immune cells with four base-edited genes, equipping the cells to better target and destroy tumors. The hope is that the approach can tame trial participants’ difficult-to-treat form of leukemia and serve as a gateway to more complex edits in the future. "

Base-edited gene therapies ‘may be superior’ for sickle cell disease
Healio, July 2023 (Posted: Jul 24, 2023 11AM)

Genomic alteration using adenine base editors demonstrated the most potency in restarting fetal hemoglobin expression in cells of patients with sickle cell disease, study results showed. The genome editing technology proved more stable while producing higher and more uniform levels of fetal hemoglobin in human hematopoietic stem cells compared with CRISPR/Cas9-based editing approaches, according to findings published in Nature Genetics.

Potent and uniform fetal hemoglobin induction via base editing.
Thiyagaraj Mayuranathan et al. Nat Genet 2023 7 (Posted: Jul 05, 2023 7AM)

Inducing fetal hemoglobin (HbF) in red blood cells can alleviate ß-thalassemia and sickle cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor cells, using either Cas9 nuclease or adenine base editors. The most potent modification was adenine base editor generation of ?-globin –175A>G. Homozygous –175A>G edited erythroid colonies expressed 81?±?7% HbF versus 17?±?11% in unedited controls, whereas HbF levels were lower and more variable for two Cas9 strategies targeting a BCL11A binding motif in the ?-globin promoter or a BCL11A erythroid enhancer.

Base editing shows potential superiority for curing sickle cell disease
Medical XPress, June 29, 2023 (Posted: Jun 30, 2023 10AM)

Scientists at St. Jude Children's Research Hospital and the Broad Institute of MIT and Harvard used a next-generation genome editing technology, adenosine base editing, to restart fetal hemoglobin expression in SCD patient cells. The approach raised the expression of fetal hemoglobin to higher, more stable, and more uniform levels than other genome editing technologies that use CRISPR/Cas9 nuclease in human hematopoietic stem cells.

Unleashing CRISPR on Cancer.
et al. N Engl J Med 2023 6 (25) e78 (Posted: Jun 22, 2023 9AM)

"What if" should precede "whether" and "how" in the social conversation around human germline gene editing.
Diewertje Houtman et al. J Community Genet 2023 6 (Posted: Jun 20, 2023 7AM)

CRISPR Therapy Exceeds Targets in Thalassemia, Sickle Cell Disease
M Basset, Medpage today, June 13, 2023 (Posted: Jun 16, 2023 8AM)

Data from two pivotal trials suggest that a single infusion of the CRISPR-based gene therapy exagamglogene autotemcel (exa-cel) can provide a "functional cure" for patients with transfusion-dependent beta-thalassemia or severe sickle cell disease.

Emerging Trends in Gene Therapy: Thalassemia as a Case Study
CDC Seminar— June 22, 2023, 2:00–3:00 PM ET Brand (Posted: May 17, 2023 11AM)

Although relatively new in terms of clinical application, several gene therapy-based treatments have, in recent years, received approval from the Food and Drug Administration (FDA) and begun to be used in real world settings in the United States. In addition, clinical trials using either gene transfer or genome editing continue to show promise, with the potential to impact treatment for patients with a wide range of hereditary disorders in the future. This webinar will showcase thalassemia as a case example in emerging approaches in gene therapy.

CRISPR Editing Takes Aim at Ischemia/Reperfusion Injury.
Oscar E Reyes Gaido et al. JAMA Cardiol 2023 5 (Posted: May 12, 2023 6AM)

Human embryo science: can the world’s regulators keep pace? Concerned by the speed of research, policymakers are looking for alternative ways to establish rules on genome editing and other technologies.
P Ball, Nature, May 10, 2023 (Posted: May 10, 2023 6AM)

The World Health Organization currently recommends against the use of heritable (or germline) human genome editing, but has no powers of enforcement — and neither does any other international body. Some national bodies, such as the HFEA, can create rules to govern research and its outputs, but such centralized oversight is relatively rare. Most scientists comply with guidelines formulated by professional bodies such as the International Society for Stem Cell Research (ISSCR), but these principles have neither legal force nor a mandate from wider society.

The ethics, equity, and governance of human genome editing need greater consideration.
Sarojini Nadimpally et al. BMJ 2023 5 p996 (Posted: May 04, 2023 6AM)

Challenges exist around heritable gene editing, its potential medical applications, ethical implications, and the need for regulatory mechanisms in the field. Adopting a purely scientific view of human genome editing risks ignoring ethical, societal, and equity considerations.

In the business of base editors: Evolution from bench to bedside.
Elizabeth M Porto et al. PLoS Biol 2023 4 (4) e3002071 (Posted: Apr 13, 2023 6AM)

With the advent of recombinant DNA technology in the 1970s, the idea of using gene therapies to treat human genetic diseases captured the interest and imagination of scientists around the world. Years later, enabled largely by the development of CRISPR-based genome editing tools, the field has exploded, with academic labs, startup biotechnology companies, and large pharmaceutical corporations working in concert to develop life-changing therapeutics.

Ethical concerns temper optimism about gene-editing for human diseases
R Stein, NPR, March 8, 2023 (Posted: Mar 08, 2023 6PM)

It's still far too premature to try to use powerful new technologies to edit genes that can be passed down from generation to generation, according to the organizers of the Third International Summit on Human Genome Editing that concluded Wednesday in London. Techniques that have made it easier to manipulate DNA still produce too many mistakes for scientists to be confident any children born from edited embryos would be healthy, according to the organizers of the Third International Summit on Human Genome Editing.

Beyond CRISPR babies: How human genome editing is moving on after scandal
H Ledford, Nature, March 2, 2023 (Posted: Mar 03, 2023 8AM)

Researchers will discuss advances in genome-editing technologies — and the ethics of deploying them — at a major international summit. When researchers gather in London next week for the Third International Summit on Human Genome Editing, they will discuss the latest advances in deploying techniques such as CRISPR–Cas9 to treat genetic diseases, and will look ahead to the possible approval of the first genome-editing therapy later this year.

CRISPR technology: A decade of genome editing is only the beginning.
Joy Y Wang et al. Science (New York, N.Y.) 2023 1 (6629) eadd8643 (Posted: Feb 10, 2023 6AM)

In the decade since the publication of CRISPR-Cas9 as a genome-editing technology, the CRISPR toolbox and its applications have profoundly changed basic and applied biological research. The authors review the origins and utility of CRISPR-based genome editing, the successes and current limitations of the technology, and where innovation and engineering are needed.

The CRISPR Patent Ruling and Implications for Medicine.
Paradise Jordan et al. JAMA 2023 1 (Posted: Jan 18, 2023 1PM)

Looming over the medical promise of CRISPR is a series of high-profile patent challenges. The US Patent and Trademark Office (PTO) within the Department of Commerce issues patents and adjudicates certain types of patent disputes. The invention must be novel, useful, and nonobvious to a person skilled in the art and must disclose the way to make and use the invention. Once granted, a patent gives the inventor 20 years of exclusivity from the date of the filing of the application, and rights are limited to the enumerated claims within the patent.

Accounting for diversity in the design of CRISPR-based therapeutic genome editing
K Saha, Nature Genetics, January 2, 2023 (Posted: Jan 02, 2023 0PM)

CRISPR cell and gene therapy have been designed largely with respect to a single reference human genome. A new study reveals how human genetic diversity could lead to off-target effects and presents a new tool to identify these risks.

Human genetic diversity alters off-target outcomes of therapeutic gene editing.
Cancellieri Samuele et al. Nature genetics 2022 12 (Posted: Dec 16, 2022 8AM)

CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat disease. However, standard computational and biochemical methods to predict off-target potential focus on reference genomes. We developed an efficient tool called CRISPRme that considers single-nucleotide polymorphism (SNP) and indel genetic variants to nominate and prioritize off-target sites. We tested the software with a BCL11A enhancer targeting guide RNA (gRNA) showing promise in clinical trials for sickle cell disease and ß-thalassemia

CRISPR infusion eliminates swelling in those with rare genetic disease
J Kaiser, Science, September, 2022 (Posted: Sep 24, 2022 7AM)

CRISPR has already been shown to treat blood disorders via an ex vivo strategy in which a patient’s cells are harvested, edited in a lab, and then returned to the body. An in vivo approach for blindness disorders, where the gene editor is injected into the eye, is also showing tentative benefits. But directing CRISPR to specific organs or cells inside the body via an intravenous infusion is harder.

Treatment of Genetic Diseases With CRISPR Genome Editing
MJ Kan et al, JAMA, September 13, 2022 (Posted: Sep 14, 2022 3AM)

Ongoing clinical trials use CRISPR for somatic cell genome editing to treat hereditary diseases or cancer. In these studies, cells are either removed and edited in tissue culture and then readministered to the patient (ex vivo), or genome editors are packaged within viral vectors or lipid nanoparticles and given intravenously to home to specific tissues (in vivo). Ex vivo therapies have focused on blood disorders, including at least 6 trials aiming to cure sickle cell disease (SCD) or transfusion-dependent ß-thalassemia (TDT).

Changing our DNA: 'The age of human therapeutic gene editing is here'
S LaMotte, CNN, May 31, 2022 (Posted: May 31, 2022 8AM)

The arrival of CRISPR systems in the 1990s and specifically CRISPR-Cas-9 in 2013 heralded a new, more elegant way to edit genes. CRISPR uses what is called guide RNA to get the Cas-9 enzyme to a more precise spot on the DNA strand to make the cut. After years of vetting, the US Food and Drug Administration approved CRISPR-Cas-9 in 2021 for use in human clinical trials for sickle cell disease.

Changes in opinions about human germline gene editing as a result of the Dutch DNA-dialogue project
D Houtman et al, EJHG, May 12, 2022 (Posted: May 14, 2022 11AM)

Major CRISPR patent decision won’t end tangled dispute Fights over who invented the gene-editing technology are becoming more complex, and could carry on for years.
H Ledford, Nature, March 9, 2022 (Posted: Mar 10, 2022 9AM)

How to protect the first ‘CRISPR babies’ prompts ethical debate
S Mallapaty, Nature, February 25, 2022 (Posted: Feb 25, 2022 9AM)

Two prominent bioethicists in China are calling on the government to set up a research centre dedicated to ensuring the well-being of the first children born with edited genomes. Scientists have welcomed the discussion, but many are concerned that the pair’s approach would lead to unnecessary surveillance of the children.

The use of new CRISPR tools in cardiovascular research and medicine.
Nishiga Masataka et al. Nature reviews. Cardiology 2022 2 (Posted: Feb 21, 2022 7AM)

In this Review, we first provide an overview of the diverse new CRISPR tools that have been developed to date. Second, we summarize how these new CRISPR tools are being used to study biological processes and disease mechanisms in cardiovascular research and medicine. Finally, we discuss the prospect of therapeutic genome editing by CRISPR tools to cure genetic cardiovascular diseases.

Year in Review: Gene editing advances on all fronts
K O'Leary, Nature Medicine, December 2021 (Posted: Dec 17, 2021 6AM)

Trove of CRISPR-like gene-cutting enzymes found in microbes- The search for a CRISPR enzyme’s ancestors has revealed more than one million potential genome-editing tools.
H Ledford, Nature, September 10, 2021 (Posted: Sep 10, 2021 8AM)

A first step toward in vivo gene editing in patients
H Buning et al, Nature Medicine, August 30, 2021 (Posted: Aug 31, 2021 9AM)

The first preliminary evidence that in vivo gene editing in a clinical setting is feasible with no early signs of severe adverse events comes from an ongoing clinical trial to treat transthyretin amyloidosis, a fatal monogenetic disease.

Governing Human Germline Editing Through Patent Law.
Sherkow Jacob S et al. JAMA 2021 8 (Posted: Aug 31, 2021 8AM)

Even though patents are not a perfect solution to the ethical challenges of germline editing, they are a useful supplement to many of the approaches currently available, especially given fractured international regulation. Patents present an opportunity to combine the tools of commercialization and ethical behavior in a manner not readily present in other fields. It is an opportunity that should not be wasted; the perfect should not be the enemy of the good.

What If You Could Become Invisible to Mosquitoes? Using Crispr, scientists have taken the first step toward creating a mosquito that is blind to human hosts.
S Imbler, NY Times, August 2021 (Posted: Aug 22, 2021 7AM)

For the first time, scientists have used the gene-editing tool Crispr-Cas9 to render humans effectively invisible in the eyes of Aedes aegypti mosquitoes, which use dark visual cues to hunt, according to a paper. By eliminating two of that mosquito’s light-sensing receptors, the researchers knocked out its ability to visually target hosts.

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
Gillmore Julian D et al. The New England journal of medicine 2021 7 (6) 493-502 (Posted: Aug 05, 2021 11AM)

In a small group of patients with hereditary ATTR amyloidosis with polyneuropathy, administration of NTLA-2001 was associated with only mild adverse events and led to decreases in serum TTR protein concentrations through targeted knockout of TTR.

How Designer DNA Is Changing Medicine - A genomic revolution is poised to cure sickle cell and other genetic diseases
C Barber, Scientific American, July 17, 2021 (Posted: Jul 19, 2021 6AM)

The next-generation technology, gene editing, is another level altogether. Gene editing enables scientists to precisely target abnormal genes of many organisms (bacteria, plants, animals), snip the DNA, then remove, replace or add new DNA at the incision site. “Imagine you have a car with a flat tire. Gene therapy is taking a fifth wheel and putting it somewhere on the car and hoping it runs. Gene editing is repairing the flat.”

He Inherited A Devastating Disease. A CRISPR Gene-Editing Breakthrough Stopped It
R Stein, NPR, June 26, 2021 (Posted: Jun 27, 2021 7AM)

The advance is being hailed not just for amyloidosis patients but also as a proof-of-concept that CRISPR could be used to treat many other, much more common diseases. It's a new way of using the innovative technology.

CRISPR injected into the blood treats a genetic disease for first time
J Kaiser, Science, June 26, 2021 (Posted: Jun 26, 2021 1PM)

The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an enormous hurdle: getting the molecular scissors into the body and having it slice DNA in the tissues where it’s needed. Now, in a medical first, researchers have injected a CRISPR drug into the blood of people born with a disease that causes fatal nerve and heart disease and shown that in three of them it nearly shut off production of toxic protein by their livers.

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
JD Gillmore et al, NEJM, June 26, 2021 (Posted: Jun 26, 2021 1PM)

In a small group of patients with hereditary Transthyretin (ATTR) amyloidosis with polyneuropathy, administration of NTLA-2001 was associated with only mild adverse events and led to decreases in serum TTR protein concentrations through targeted knockout of TTR.

1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive
R Stein, NPR, December 15, 2020 (Posted: Dec 16, 2020 8AM)

"It is a big deal because we were able to prove that we can edit human cells and we can infuse them safely into patients and it totally changed their life."

Should you choose your baby's eye color?
K Swisher, NY Times, October 22, 2020 (Posted: Oct 23, 2020 7AM)

CRISPR-Cas9 is the kind of scientific breakthrough that could change human evolution. Scientists call it “genetic scissors” — a tool that snips DNA with powerful and scary precision. What does this technology mean for how we live — and die? And just because we can more precisely “edit” life, should we?

A cut above: pair that developed CRISPR earns historic award
J Cohen, Science, October 16, 2020 (Posted: Oct 17, 2020 0PM)

The pair built on discovery that bacteria have distinctive stretches of DNA—so-called clustered regularly interspaced short palindromic repeats, or CRISPR—embedded in their genomes. Retained from viruses that have infected the microbes in the past, the DNA allows them to recognize and defend themselves against invasions. They reconfigured CRISPR to turn it into a tool that could edit DNA in a cell-free system.

As genome-editing trials become more common, informed consent is changing
Science Daily, October 12, 2020 (Posted: Oct 14, 2020 8AM)

As public interest and expanded research in human genome editing grows, many questions remain about ethical, legal and social implications of the technology. People who are seriously ill may overestimate the benefits of early clinical trials while underestimating the risks. This makes properly understanding informed consent, the full knowledge of risks and benefits of treatments, especially important.

Heritable Genome Editing Not Ready for the Clinic, Panel Says
J Abbasi, JAMA, October 13, 2020 (Posted: Oct 13, 2020 11AM)

The report focuses on scientific considerations and lays out a “responsible pathway for clinical use” of heritable human genome editing. The group also called for the formation of an international scientific advisory panel of independent experts that can assess any technique’s safety and effectiveness before it’s used clinically.

Heritable Human Genome Editing- The International Commission Report
EY Adashi et al, JAMA< October 8, 2020 (Posted: Oct 09, 2020 6AM)

The commission firmly acknowledged the significant potential of HHGE in the prevention of heritable monogenic (but not polygenic) diseases. Near-term implementation of HHGE, however, was firmly ruled out because the efficiency and specificity of current embryo editing methods are not adequate for human clinical uses.

Pioneers of revolutionary CRISPR gene editing win chemistry Nobel
H Ledford et al, Nature, October 7, 2020 (Posted: Oct 07, 2020 1PM)

The technology allows precise edits to the genome and has swept through laboratories worldwide since its inception in the 2010s. It has countless applications: researchers hope to use it to alter human genes to eliminate diseases; create hardier plants; wipe out pathogens and more.

‘CRISPR babies’ are still too risky, says influential panel - The safety and efficacy of gene editing human embryos hasn’t been proven, researchers warn.
H Ledford Nature News, September 4, 2020 (Posted: Sep 04, 2020 9AM)

The international report — which reviewed the scientific and technical state of heritable gene editing, rather than ethical questions — advocates the formation of an international committee that evaluates developments in the technology and advises political leaders and regulators on its safety and utility.

A ‘Cure for Heart Disease’? A Single Shot Succeeds in Monkeys
G Kolata, NY Times, June 27, 2020 (Posted: Jun 29, 2020 8AM)

In the first gene-editing experiment of its kind, scientists have disabled two genes in monkeys that raise the risk for heart disease (PCSK9 and LDL). Humans carry the genes as well, and the experiment has raised hopes that a leading killer may one day be tamed.

CRISPR gene editing in human embryos wreaks chromosomal mayhem
H Ledford, Nature News, June 25, 2020 (Posted: Jun 26, 2020 8AM)

A suite of experiments that use the gene-editing tool CRISPR–Cas9 to modify human embryos have revealed how the process can make large, unwanted changes to the genome at or near the target site.

Scientists tap CRISPR’s search-and-detect skills to create a rapid Covid-19 test
E Brodwin, Stat News, April 16, 2020 (Posted: Apr 16, 2020 9AM)

A new method uses CRISPR to quickly spot the coronavirus in samples from nose or throat swabs. The test takes roughly 40 minutes, compared with four to six hours for traditional PCR-based Covid-19 tests, which are currently being used. But the test also appears to produce false-negatives slightly more often than existing coronavirus tests.

With First CRISPR Trials, Gene Editing Moves Toward the Clinic
T Hampton, JAMA, April 8, 2020 (Posted: Apr 09, 2020 7AM)

An experimental CRISPR-based therapy directly injected under the retina could potentially restore vision by eliminating a genetic alteration in photoreceptor cells that causes Leber congenital amaurosis.

In A 1st, Scientists Use Revolutionary Gene-Editing Tool To Edit Inside A Patient
R Stein, NPR, March 4, 2020 (Posted: Mar 05, 2020 8AM)

The groundbreaking procedure involved injecting the microscopic gene-editing tool into the eye of a patient blinded by a rare genetic disorder, in hopes of enabling the volunteer to see.

The Case for Remedial Germline Editing—The Long-term View
EY Adashi et al, JAMA Health Forum, Ferbuary 27, 2020 (Posted: Feb 28, 2020 8AM)

Numerous genetic diseases affect millions of people worldwide. This article compares two technologies under consideration to tackle this challenge: preimplantation genetic diagnosis & remedial germline editing.

Super-precise CRISPR tool enhanced by enzyme engineering: Improvements to a method known as base editing could pave the way for safer gene therapies.
H Ledford, Nature, February 11, 2020 (Posted: Feb 12, 2020 8AM)

CRISPR takes on cancer
J Couzin-Frankel, Science, February 7, 2020 (Posted: Feb 07, 2020 8AM)

Launching a new chapter in the fast-moving cancer immunotherapy field, scientists have blended two cutting-edge approaches: CRISPR, which edits DNA, and T cell therapy, in which sentries of the immune system are exploited to destroy tumors.

Step aside CRISPR, RNA editing is taking off: Making changes to the molecular messengers that create proteins might offer flexible therapies for cancer, pain or high cholesterol, in addition to genetic disorders.
S Reardon, Nature, February 4, 2020 (Posted: Feb 05, 2020 9AM)

Prime Time for Genome Editing?
Urnov Fyodor D et al. The New England journal of medicine 2020 01 (5) 481-484 (Posted: Jan 31, 2020 9AM)

Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects.
Li Hongyi et al. Signal transduction and targeted therapy 2020 1 (Posted: Jan 16, 2020 8AM)

Navigating 2020 and beyond
Nature Genetics editorial, January 7, 2020 (Posted: Jan 08, 2020 8AM)

As we usher in a new year of a new decade and ponder what the future will bring for the genetics field, we wish to reflect on some specific areas related to diversity, privacy and genome editing that require attention and vigilance from the community.

Quest to use CRISPR against disease gains ground
H Ledford, Nature, January 6, 2020 (Posted: Jan 08, 2020 8AM)

As the first clinical-trial results trickle in, researchers look ahead to more sophisticated medical applications for genome editing.

Gene Therapies Make It to Clinical Trials
L Marsa, Discover, December 2020 (Posted: Jan 06, 2020 9AM)

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

The case for banning heritable genome editing.
Botkin Jeffrey R et al. Genetics in medicine : official journal of the American College of Medical Genetics 2019 Nov (Posted: Nov 23, 2019 7AM)

This analysis suggests that the social benefits of heritable genome editing are insufficient to warrant the necessary resources and opportunity costs involved in technology development.

Time to start intervening in the human germline? A utilitarian perspective
K Smith, Bioethics, November 2019 (Posted: Nov 23, 2019 7AM)

Focusing on present-day possibilities raised by existing technology, I consider the normative aspects of genetically modifying the human germline from a utilitarian standpoint. With reference to a hypothetical case, I examine the probable consequences of permitting a well-conceived attempt to correct a disease-associated gene.

First CRISPR treatment for blood diseases shows early benefits in two patients
S Begley, StatNews, November 19, 2019 (Posted: Nov 21, 2019 7AM)

Human germline editing needs one message
Nature editorial, November 20, 2019 (Posted: Nov 21, 2019 7AM)

The speed of technological advance, coupled with some scientists’ determination to press ahead with editing human germline cells — eggs, sperm and embryonic cells — has been sounding alarm bells for nearly five years. Editing could produce unpredictable changes that an individual’s descendants will inherit — with potentially wide-reaching societal implications.

CRISPR's unwanted anniversary
J Doudna, Science, November 15, 2019 (Posted: Nov 16, 2019 7AM)

"It is encouraging that scientists around the globe responded by opening a deeper public conversation about how to establish stronger safeguards and build a viable path toward transparency and responsible use of CRISPR technology."

Heritable Genome Editing—Edited Eggs and Sperm to the Rescue?
E Adashi et al, JAMA, November 13, 2019 (Posted: Nov 14, 2019 8AM)

Heritable genome editing is widely predicted to render inborn afflictions a thing of the past. Topping the list of edit-worthy maladies are single-gene disorders. An insufficient number of viable embryos without the disease mutation is an important limitation in preimplantation genetic diagnosis, heritable genome editing might offer an alternative strategy.

CRISPR: the movie- New gene-editing documentary showcases biology’s hottest tool — up to the point when things went awry
A Maxmen, Nature, November 11, 2019 (Posted: Nov 12, 2019 8AM)

Gene editing enters ‘prime’ time
Nature Biotechnology, November 7, 2019 (Posted: Nov 11, 2019 6AM)

Early results suggest that prime editors are cleaner than CRISPR–Cas9 and more versatile than base editors, but many questions remain.

NIH Director Backs Moratorium for Heritable Genome Editing
Gen News, November 8, 2019 (Posted: Nov 09, 2019 8AM)

Doctors try CRISPR gene editing for cancer, a 1st in the US
M Marchione, Washington Post, November 6, 2019 (Posted: Nov 07, 2019 8AM)

CRISPR Approach To Fighting Cancer Called 'Promising' In 1st Safety Test
R Stein, NPR, November 6, 2019 (Posted: Nov 07, 2019 7AM)

Crispr Takes Its First Steps in Editing Genes to Fight Cancer
D Grady, NY Times, November 6, 2019 (Posted: Nov 07, 2019 7AM)

Doctors have for the first time in the United States tested a powerful gene-editing technique in people with cancer. The test, meant to assess only safety, was a step toward the ultimate goal of editing genes to help a patient’s own immune system to attack cancer. The editing was done by the DNA-snipping tool Crispr.

Gene-Editing Advance Puts More Gene-Based Cures Within Reach
F Collins, NIH Director Blog, November 5, 2019 Brand (Posted: Nov 06, 2019 8AM)

There’s been tremendous excitement about the potential of CRISPR and related gene-editing technologies for treating or even curing sickle cell disease (SCD), muscular dystrophy, HIV, and a wide range of other devastating conditions. Now comes word of another remarkable advance—called “prime editing”—that may bring us even closer to reaching that goal.

Is Crispr the Next Antibiotic?
K Sheikh, NY Times, October 2019 (Posted: Nov 05, 2019 8AM)

In nature, the gene-editing tool Crispr protects bacteria against viruses. Now it’s being harnessed in the fight against superbugs and the flu.

CRISPR-edited babies arrived, and regulators are still racing to catch up
A Opar, Nature, October 24, 2019 (Posted: Oct 25, 2019 1PM)

New CRISPR tool has the potential to correct almost all disease-causing DNA glitches, scientists report
S Begley. Stat News, October 21, 2019 (Posted: Oct 22, 2019 9AM)

Scientists Create New, More Powerful Technique To Edit Genes
R Stein, NPR, October 21, 2019 (Posted: Oct 22, 2019 9AM)

Scientists have created a new way to edit DNA that appears to make it easier to precisely and safely re-write genes. The new technique is designed to overcome some of the limitations of CRISPR. That technique, often described as a kind of molecular scissors for genes, has been revolutionizing scientific research by letting scientists alter DNA.

Before heritable genome editing, we need slow science and dialogue ‘within and across nations’
F Bayliss, StatNews, September 23, 2019 (Posted: Sep 24, 2019 8AM)

Nano-Sized Solution for Efficient and Versatile CRISPR Gene Editing
NIH Director blog, September 17, 2019 Brand (Posted: Sep 18, 2019 9AM)

Could editing the DNA of embryos with CRISPR help save people who are already alive?
A Joseph, StatNews, September 16, 2019 (Posted: Sep 16, 2019 9AM)

Good News, Bad News for CRISPR Gene Editing for HIV Cure- "Bigger story for the gene-editing field than the HIV field"
M Walker, Medpage today, September 11, 2019 (Posted: Sep 16, 2019 8AM)

CRISPR, the gene-editing technique, went 1-for-2 in a real world tryout, showing safety and successful engraftment of edited cells in a patient with HIV infection, but with no clinical benefit.

Many cancer drugs aim at the wrong molecular targets- Analysis using CRISPR gene-editing technology suggests that drugs’ mechanism of action are misunderstood.
H Ledford, Nature, September 12, 2019 (Posted: Sep 13, 2019 10AM)

CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia.
Xu Lei et al. The New England journal of medicine 2019 Sep (Posted: Sep 12, 2019 7AM)

Forget single genes: CRISPR now cuts and splices whole chromosomes
RF Service, Science News, August 29, 2019 (Posted: Sep 03, 2019 8AM)

Scientists Attempt Controversial Experiment To Edit DNA In Human Sperm Using CRISPR
R Stein, NPR, August 22, 2019 (Posted: Aug 22, 2019 7AM)

International Commission on Heritable Genome Editing Holds First Public Meeting
A Robinson, NASEM, August 20, 2019 (Posted: Aug 22, 2019 7AM)

Designer babies are on the way. We're not ready
R Klitzman, CNN, August 16,2019 (Posted: Aug 20, 2019 8AM)

CRISPR enters its first human clinical trials- The gene editor targets cancer, blood disorders and blindness
T Hesman, Science News, August 14, 2019 (Posted: Aug 17, 2019 9AM)

Since its debut in 2012, CRISPR gene editing has held the promise of curing most of the over 6,000 known genetic diseases. Now it’s being put to the test. In the first spate of clinical trials, scientists are using CRISPR/Cas9 to combat cancer and blood disorders in people.

For rules on creating ‘CRISPR babies’ from edited embryos, scientists call a do-over
S Begley, Stat News, August 12, 2019 (Posted: Aug 13, 2019 8AM)

China approves ethics advisory group after CRISPR-babies scandal
H Jia, Nature, August 8, 2019 (Posted: Aug 12, 2019 8AM)

Gene therapy for primary immunodeficiency.
Booth Claire et al. Human molecular genetics 2019 Jul (Posted: Aug 05, 2019 8AM)

This review provides an overall outcome and progress in gene therapy clinical trials for several primary immunodeficiency disorders (SCID-X, ADA-SCID, WAS, X- CGD), and the recent developments in genome editing technology for developing potential therapy, and highlights key studies.

Did CRISPR help—or harm—the first-ever gene-edited babies?
J Cohen, Science, August 1, 2019 (Posted: Aug 02, 2019 9AM)

Statement on governance and oversight of human genome editing
WHO, July 26, 2019 (Posted: Aug 01, 2019 9AM)

The WHO expert advisory committee on governance and oversight of human genome editing issued an interim recommendation to the WHO Director-general stated that “it would be irresponsible at this time for anyone to proceed with clinical applications of human germline genome editing.” WHO supports this interim recommendation.

CRISPR Could Be the Future of Disease Diagnosis,
by Emily Mullin, One Zero, July 25, 2019 (Posted: Jul 26, 2019 0PM)

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Disclaimer: Articles listed in Hot Topics of the Day are selected by Public Health Genomics Branch to provide current awareness of the scientific literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the Clips, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.
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