Trends in development of self-complementary adeno-associated virus vector / 生物工程学报
Chinese Journal of Biotechnology
; (12): 658-664, 2009.
Article
en Zh
| WPRIM
| ID: wpr-286660
Biblioteca responsable:
WPRO
ABSTRACT
Numerous studies and clinical trials have demonstrated the efficacy of recombinant adeno-associated virus gene delivery vectors. However, prior to expression, it is necessary to convert the single-stranded DNA genome into double-stranded DNA, which hinders the efficiency of these vectors. We can entirely circumvent this step through the use of self-complementary recombinant adeno-associated virus vector (scrAAV). ScrAAV packages an inverted repeat genome that can fold into double-stranded DNA without the requirement for DNA synthesis or base-pairing between multiple vector genomes. By using scrAAV, we could increase expression efficiency and reduce immune response caused by vectors themselves. Therefore, it is a promising vector for gene therapy. So far, it has been used in the treatment of hepatic diseases, central nervous system diseases, and eye diseases. It has also been used in the modifications of stem cells and as vectors for siRNA/miRNA and ribozymes. In this review, we focused on the preparation, expression and location of scrAAV both in vitro and in vivo. We mainly introduced the recent progress of scrAAV based therapy of Hemophilia B, in order to elucidate the potential and prospects of scrAAV in gene therapy.
Texto completo:
1
Base de datos:
WPRIM
Asunto principal:
Terapéutica
/
ADN
/
ADN de Cadena Simple
/
Datos de Secuencia Molecular
/
Secuencia de Bases
/
Terapia Genética
/
Hemofilia B
/
Técnicas de Transferencia de Gen
/
ADN Complementario
/
Dependovirus
Límite:
Animals
/
Humans
Idioma:
Zh
Revista:
Chinese Journal of Biotechnology
Año:
2009
Tipo del documento:
Article