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 ...

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.

Understanding human gene function in living organisms has long been hampered by fundamental differences between species. Although mice share most ...

Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...

In a new study, the researchers have shown that the CRISPR–Cas3 system can induce reliable, extensive deletions of the ...

CHANGE-seq-BE was developed to enable scientists to better understand base editors, an important class of CRISPR precise genome editors.

Powered by gene editing advances like CRISPR and base editing, cell and gene therapy (CGT) is delivering on the promise of genomic medicine. First-generation CAR T-cell therapies, for example, have ...

Restricted access to genome-editing technologies poses serious challenges for countries like India that urgently need such ...