Toward a gene therapy for neurological and somatic MPSIIIA
Haurigot Mendonça, Virginia (Universitat Autònoma de Barcelona. Departament de Bioquímica i de Biologia Molecular)
Bosch i Tubert, Fàtima (Universitat Autònoma de Barcelona. Departament de Bioquímica i de Biologia Molecular)

Date:	2013
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.
Note:	Número d'acord de subvenció MICINN/SAF2008-00962
Note:	Número d'acord de subvenció MINECO/SAF2011-24698
Note:	Número d'acord de subvenció AGAUR/2009/SGR-224
Note:	Número d'acord de subvenció CLINIGENE/LSHB-CT-2006-018933
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Language:	Anglès.
Document:	article ; recerca ; publishedVersion
Subject:	MPSIIIA ; LSD ; CNS gene ; Therapy ; CSF ; AAV
Published in:	Rare Diseases, Vol. 1 Núm. 1 (desembre 2013) , p. e27209, ISSN 2167-5511

DOI: 10.4161/rdis.27209
PMID: 25003015

7 p, 651.7 KB

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