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Last Posted: Jan 26, 2023
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The CRISPR Patent Ruling and Implications for Medicine.
Paradise Jordan et al. JAMA 2023 1

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

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

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

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.

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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.

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