Researchers found CAR T-cell therapy may serve as effective alternative for patients with these disorders.

The two studies, presented at ASHG 2023, identified base editing and prime editing approaches for treating the rare newborn genetic disease

CHOP researchers find exposing cells to a small molecule drug improves cell fitness during hematopoietic stem cell transplants, which could improve ex vivo gene therapy.

For decades, doctors and researchers have been working diligently to discover new treatments and provide more options for patients with Duchenne muscular dystrophy (DMD), a debilitating genetic disease that ravages the muscles in the body and leads to a loss of movement and coordination.

A Penn Medicine and CHOP team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health.

CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.

CHOP has treated its 500th patient with CAR T-cell immunotherapy, a “living drug” that harnesses the power of a patient’s own immune system to fight cancer.

CHOP researchers will develop gene therapies for multiple sulfatase deficiency, a devastating lysosomal storage disorder, with the goal of first-in-human trials.

CHOP gene therapy expert Dr. Lindsey George reacts to a phase 3 hemophilia gene therapy study in an editorial in the New England Journal of Medicine.

CHOP will be a provider in Independence Blue Cross’s new Advanced Network for Gene-Based Therapeutics, effective March 1, 2023.