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 ...

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

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 ...

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 ...

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

Waking up this morning to news of the much-deserved Nobel Prize win for Emmanuelle Charpentier and Jennifer A. Doudna "for the development of a method for genome editing" confirms the importance of ...