After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

Researchers at the University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's ...

While heritable human genome editing is banned in the United States, across Europe and much of the world, the technology ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's disease ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

OCU400 is Ocugen’s novel modifier gene therapy for retinitis pigmentosa (RP). The OCU400 (AAV5-h NR2E3) gene therapy platform ...