Posted: Jan 26, 2023
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.