The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas. KJ Muldoon is walking and getting ready to celebrate ...

A baby born with a rare and life-threatening genetic disorder has taken his first steps after receiving a groundbreaking, ...

A miracle baby who was treated with a pioneering gene-therapy at CHOP has reached a major milestone as he took his first ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

A child who received the world's first personalized CRISPR therapy is now taking his first steps after months in hospital ...

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...

JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...

ZURICH (Reuters) - Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to "step up", Chief Executive ...

Talk about a Christmas miracle!A medical breakthrough came just in time and changed one baby’s life — and gave his family a ...