title: Toward a gene therapy for neurological and somatic MPSIIIA.
abstract: Mucopolysaccharidosis Type IIIA (MPSIIIA) represents an unmet medical need. MPSIIIA shares with many other lysosomal storage disorders (LSD) the characteristic of being a severe neurodegenerative disease accompanied by mild somatic involvement. Thus, the main target organ for the development of new treatments is the central nervous system (CNS), but overall clinical efficacy would be greatly enhanced by simultaneous correction of peripheral disease. We have recently developed a novel treatment for MPSIIIA based on the delivery to the cerebrospinal fluid of serotype 9 adeno-associated virus (AAV9)-derived vectors. This gene therapy strategy corrected both CNS and somatic pathology in animal models through widespread transduction of CNS, peripheral nervous system (PNS), and liver. The work set the grounds for the clinical translation of the approach to treat MPSIIIA in humans. Here we discuss some important considerations that further support the applicability of this treatment to MPSIIIA and other LSD with CNS and somatic involvement.
authors.affiliation: Center of Animal Biotechnology and Gene Therapy (CBATEG) and Department of Biochemistry and Molecular Biology; School of Veterinary Medicine; Universitat Autònoma de Barcelona; Barcelona, Spain.
authors.forename: Virginia
authors.initials: V
authors.lastname: Haurigot
authors.affiliation: Center of Animal Biotechnology and Gene Therapy (CBATEG) and Department of Biochemistry and Molecular Biology; School of Veterinary Medicine; Universitat Autònoma de Barcelona; Barcelona, Spain.
authors.forename: Fatima
authors.initials: F
authors.lastname: Bosch
keywords: AAV; CNS gene therapy; CSF; LSD; MPSIIIA