Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
SUMF1 positive cells in tissues following IT and combined IT/IV gene therapy. Credit: Communications Medicine (2025). DOI: 10.1038/s43856-025-00734-9 In two...
genetic
ASH: Gene therapy for rare genetic disorder, molecular insights into leukemia growth and more
Researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA will present their...
Horae Gene Therapy Center now Department of Genetic & Cellular Medicine
Guangping Gao, PhD, is founding chair of the Department of Genetic & Cellular Medicine.Photo: Faith Ninivaggi The Horae Gene Therapy Center...
Genetic engineering and the eye
Hoermann A, Habtewold T, Selvaraj P, Del Corsano G, Capriotti P, Inghilterra MG, et al. Gene drive mosquitoes can aid...
Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord
Introduction Classically, the majority of medical treatments have been developed for diseases affecting large number of patients and patients with...
Gene Therapy Promising for Rare Genetic Cardiomyopathy
CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or stabilize the...
Gene Therapy Technology Discovery Could Advance Genetic Disease Treatment
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Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders
IntroductionIn medicine, gene therapy is defined as therapeutic strategy that transfers DNA to a patient’s cells to correct a defective...
Gene therapy halts progression of rare genetic condition in young boy
Michael is checked by a nurse at SickKids before receiving gene therapy in a single-patient clinical trial for spastic paraplegia...
