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

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

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

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.

How to protect the first ‘CRISPR babies’ prompts ethical debate
S Mallapaty, Nature, February 25, 2022

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.

Disclaimer: Articles listed in the Public Health Genomics and Precision Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the 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 update, 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.