Retroviral approaches to gene therapy of cystic fibrosis.
Ann N Y Acad Sci
; 953: 43-52, 2001 Dec.
Article
en En
| MEDLINE
| ID: mdl-11795422
Retroviral vectors are attractive as vectors for gene therapy of cystic fibrosis because of their ability to integrate into the host cell genome, which may lead to long-term expression and, perhaps, a cure. Nevertheless, retroviral applications for gene transfer to airway epithelia have been limited by low titers and a requirement for proliferating cells. Significant advances in pseudotyping of retroviruses and in retroviral production have reduced some of the concerns regarding titer. The development of lentiviral vectors that transduce nondividing cells has also helped to establish that retroviral approaches for gene therapy of cystic fibrosis are feasible. However, the apical membrane of the airway epithelium remains a formidable barrier to gene transfer. In this review, I will discuss limitations of current retroviral gene transfer vectors and strategies to improve retroviral gene transfer efficiency to airway epithelia in vivo.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Retroviridae
/
Terapia Genética
/
Fibrosis Quística
/
Vectores Genéticos
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Ann N Y Acad Sci
Año:
2001
Tipo del documento:
Article
País de afiliación:
Estados Unidos
Pais de publicación:
Estados Unidos