Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.

Humanized mouse models are vital for studying human gene function, but fully replacing mouse genes with complete human ...

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St.

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 ...

CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...

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

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St. Jude Children's Research Hospital. Significant amounts of time and resources ...

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 ...

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 ...

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