Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.
CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...
In a new study, the researchers have shown that the CRISPR–Cas3 system can induce reliable, extensive deletions of the ...
The Phase 1/2 trial is a first-in-human, open-label, multi-national study designed to evaluate the safety, tolerability, and efficacy of TSRA-196 in adults with AATD. Trial participants will receive a ...
Scientists and physicians can better assess precision genome editing technology using a new method made public today by St.
Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...
In conjunction with the strategic evolution, the Company appointed Jian Irish, Ph.D., M.B.A., as Chief Executive Officer. Based on a revised capital allocation strategy, the Company extended its ...
CHANGE-seq-BE was developed to enable scientists to better understand base editors, an important class of CRISPR precise genome editors.
Back in 2016, a group of researchers led by Harvard’s David Liu, PhD, published a paper in Nature that reported the development of base editing. Base editing, they wrote, is “a new approach to genome ...
Researchers restored hearing in preclinical mouse models with a specific form of inherited deafness called DFNA50 caused by mutations in microRNA, by using a novel in vivo CRISPR genome editing ...