Gene Therapy for Huntington's Disease Begins Moving from Lab to Clinic
No evidence of adverse events or significant safety concerns has surfaced.
Genetics-Based Advances in Retinitis Pigmentosa Research
Gene therapy with the CRISPR/Cas9 system corrects mutations identified with patient-specific models that use stem cells generated from skin.
Milestone in Stem Cell Transplant Therapy for Advanced Age-Related Macular Degeneration
Study results show that transplanted human retinal stem cells rescue photoreceptors in rabbit retinas for 8 months.