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List of works by Sandra Motas

Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy

scientific article published on 18 December 2014

CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)

scientific article

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

scientific article published on 17 February 2017

Insulin-like growth factor I (IGF-I)-induced chronic gliosis and retinal stress lead to neurodegeneration in a mouse model of retinopathy

scientific article published on 25 April 2013

Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC.

scientific article published on 04 August 2016

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

scientific article

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