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 child who received the world's first personalized CRISPR therapy is now taking his first steps after months in hospital ...
A miracle baby who was treated with a pioneering gene-therapy at CHOP has reached a major milestone as he took his first ...
A toddler who received the first-of-its-kind gene therapy for a potentially fatal disease has taken his first steps at 16 ...
On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...
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 ...