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Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.

Human molecular genetics - 15 Apr 2017

Roca Carles, Motas Sandra, Marcó Sara, Ribera Albert, Sánchez Víctor, Sánchez Xavier, Bertolin Joan, León Xavier, Pérez Jennifer, Garcia Miguel, Villacampa Pilar, Ruberte Jesús, Pujol Anna, Haurigot Virginia, Bosch Fatima

Abstract excerpt

Gene therapy is a promising therapeutic alternative for Lysosomal Storage Disorders (LSD), as it is not necessary to correct the genetic defect in all cells of an organ to achieve therapeutically significant levels of enzyme in body fluids, from which non-transduced cells can uptake the protein correcting their enzymatic deficiency. Animal models are instrumental in the development of new treatments for LSD. Here...

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PMID: 28334745
DOI: 10.1093/hmg/ddx058

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