A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage, with human trials ...

In major step toward the clinic, Ring’s human viral vector delivers gene therapy to eyes of mice. By Helen Floersh Apr 2, 2024 8:10am. Cell & Gene Therapy Flagship Pioneering viral ...

New gene delivery vehicle shows promise for human brain gene therapy. ScienceDaily. Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2024 / 05 / 240516160501.htm.

SwanBio Advances to Higher-Dose Cohort in First-in-Human Study of Gene Therapy for Adrenomyeloneuropathy. DSMB has recommended dose-escalation. First patient in cohort 2 has received SBT101.

The first in-human trial of a CRISPR Cas system delivered by adeno-associated virus 9 gene therapy to treat HIV was able to target only the intended DNA and cleared the blood within 6 months. EBT ...

A new gene therapy has been shown to be safe and effective in 10 young children who were recently diagnosed with a rare immunodeficiency disorder. This disorder, Artemis-deficient severe combined ...

Infant Becomes First Human Ever To Receive Personalized CRISPR Gene Therapy Treatment The child started receiving the treatment when they were 6 months old and so far things are looking good. Dr ...

State-of-the-Art AI Predicts Gene Activity in Human Cells AI foundation model may help genetics, cancer, and complex disease research. Posted January 10, 2025 | Reviewed by Monica Vilhauer Ph.D.