Vertex Pharmaceuticals may have narrowed its focus this year, but the biopharma’s decision to stick with its remaining islet ...

From June 27 to 29 at the Holiday Inn Parco Dei Medici in Rome, the international congress "CDKL5 All InVolved" will be held, ...

PBFT02 continued to demonstrate robust, durable elevation in CSF PGRN levels and improvement in plasma NfL, a disease progression biomarker, compared to natural history Dose 2, 50% lower than Dose 1 ...

Repositioning genes awakens fetal hemoglobin to treat disease. CRISPR editing may change future gene therapy. Researchers ...

Skin grafts genetically engineered from a patient's own cells can heal persistent wounds in people with an extremely painful ...

A Northeast Ohio woman and a 22-year-old man from UAE are among the first patients to receive the innovative CRISPR treatment ...

Dr. Omar Llaguna, specialist in surgical oncology, programs a HistoSonics medical device for targeting a medium that mimics a liver tumor in a human body to demonstrate how a histotripsy procedure ...

Recessive dystrophic epidermolysis bullosa (RDEB) is a systemic disease of poor mucocutaneous basement membrane integrity downstream of bi-allelic pathogenic variants in COL7A1. Cutaneous clinical ...

Penn researchers believe new potential gene therapy for glioblastoma brain cancer offers ‘a lot of hope’ Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with ...

Skin grafts genetically engineered from a patient's own cells can heal persistent wounds in people with an extremely painful ...