Gene Therapy for Lysosomal Storage Disorders: Recent Advances and Limitations
J. inborn errors metab. screen
;
5: e160028, 2017. tab
Artigo
em Inglês
|
LILACS-Express
| LILACS
| ID: biblio-1090937
ABSTRACT
Abstract Lysosomal storage disorders (LSDs) are a group of diseases with multisystemic features. Current treatments have limitations and gene therapy arises as a promising treatment option. Here, we discuss some of the most recent studies for gene therapy in LSD, vectors used, and outcomes. In particular, the approaches used in animal models aiming to correct the central nervous system, the eye, and the bones are highlighted. Finally, we discuss the recent reports of clinical trials using this technology for these diseases. We conclude that gene therapy for LSD has gathered a substantial amount of evidence from animal models to know its potential and limitations. First evidences from clinical trials using both adeno-associated and lentiviral vectors show that this approach is safe and efficient and therefore could provide an effective treatment for several LSD in the near future.
Texto completo:
DisponíveL
Índice:
LILACS (Américas)
Idioma:
Inglês
Revista:
J. inborn errors metab. screen
Assunto da revista:
Medicina Cl¡nica
/
Patologia
Ano de publicação:
2017
Tipo de documento:
Artigo
País de afiliação:
Brasil
Instituição/País de afiliação:
Hospital de Clínicas de Porto Alegre/BR
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