Gene delivery strategies for the treatment of mucopolysaccharidoses.
BACKGROUND
Mucopolysaccharidosis (MPS) disorders are genetic diseases caused by deficiencies in the lysosomal enzymes responsible for the degradation of glycosaminoglycans. Current treatments are not able to correct all disease symptoms and are not available for all MPS types, which makes gene therapy especially relevant. Multiple gene therapy approaches have been tested for different types of MPS, and our aim in this study is to critically analyze each of them.


METHODS
In this review, we have included the major studies that describe the use of adeno-associated retroviral and lentiviral vectors, as well as relevant non-viral approaches for MPS disorders.


CONCLUSIONS
Some protocols such as the use of adeno-associated vectors and lentiviral vectors are approaching the clinic for these disorders and, along with combined approaches, seem to be the future of gene therapy for MPS.
Centro de Terapia Gênica- Hospital de Clinicas de Porto Alegre , Ramiro Barcelos, 2350, Porto Alegre, RS 90035-903 , Brazil.
Guilherme
G
Baldo
Roberto
R
Giugliani
Ursula
U
Matte
D000818:Animals; D000229:Dependovirus / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D016086:Lentivirus / Q000235:genetics; D009083:Mucopolysaccharidoses / Q000628:therapy*; D012190:Retroviridae / Q000235:genetics