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

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

Picture CRISPR-Cas9, a gene editing technology, as a GPS-guided scalpel: gRNA directs the Cas9 enzyme, a protein that cuts ...

Scientists first read the human genome, a three-billion-letter biological book, in April 2003. Since then, researchers have steadily advanced the ability to write DNA, moving far beyond single-gene ...

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 ...

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 ...

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

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