Gene therapy for inherited diseases of liver metabolism.
Hum Gene Ther
; 26(4): 186-92, 2015 Apr.
Article
em En
| MEDLINE
| ID: mdl-25830689
ABSTRACT
Gene therapy is entering the stage of initial clinical development to treat a growing number of inherited metabolic diseases. This review outlines the development of liver-directed gene therapy for diseases caused by deficiencies of enzymes that are primarily expressed in the liver and discusses the disorders that appear most promising for clinical translation.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Hepatopatias
/
Doenças Metabólicas
Limite:
Animals
/
Humans
Idioma:
En
Revista:
Hum Gene Ther
Assunto da revista:
GENETICA MEDICA
/
TERAPEUTICA
Ano de publicação:
2015
Tipo de documento:
Article
País de afiliação:
Itália