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oai:repisalud.isciii.es:20.500.12105/273082026-03-10T01:15:55Zcom_20.500.12105_19586com_20.500.12105_2202col_20.500.12105_19587

00925njm 22002777a 4500 dc Byrne, Barry J author Flanigan, Kevin M author Matesanz, Susan E author Finkel, Richard S author Waldrop, Megan A author D'Ambrosio, Eleonora S author Johnson, Nicholas E author Smith, Barbara K author Bönnemann, Carsten author Carrig, Sean author Rossano, Joseph W author Greenberg, Barry author Lalaguna, Laura author Lara-Pezzi, Enrique author Subramony, Sub author Corti, Manuela author Mercado-Rodriguez, Claudia author Leon-Astudillo, Carmen author Ahrens-Nicklas, Rebecca author Bharucha-Goebel, Diana author Gao, Guangping author Gessler, Dominic J author Hwu, Wuh-Liang author Chien, Yin-Hsiu author Lee, Ni-Chung author Boye, Sanford L author Boye, Shannon E author George, Lindsey A author 2025-06-04 Currently, there are an estimated 8,000 genetic disorders that cumulatively affect approximately 10% of the population. Even among the 5% of patients with genetic disease that have treatment options, these therapeutics rarely address the underlying cause of disease but rather focus on managing or modifying symptoms and typically require recurrent, lifelong therapy. A therapeutic approach to genetic disease that in vivo delivers a functional copy of the aberrant gene is an intuitive solution that has thus far taken 3 decades to reduce to clinical practice, predominantly using adeno-associated viral (AAV) vectors. Among available viral and non-viral gene delivery approaches, AAV vectors remain the most efficient means for in vivo delivery of DNA to the nucleus. AAV vectors now constitute a bone fide novel therapeutic drug class composed of seven US Food and Drug Administration-approved products with over 10-fold more in clinical development for an expanding number of disease indications and an identified list of problems to overcome for widespread clinical application. Here, we review current progress in clinical AAV gene therapy, including for neuromuscular disorders, hemophilia, primary cardiovascular disorders, or disorders with cardiovascular manifestations, lysosomal storage disorders, mucopolysaccharide disorders, primary central nervous systemic disorders, and ocular disorders. Mol Ther. 2025 Jun 4;33(6):2479-2516. Molecular Therapy 40329530 https://hdl.handle.net/20.500.12105/27308 adeno-associated viral vectors in vivo gene therapy Current clinical applications of AAV-mediated gene therapy.