Gene Therapy in Rats with a Lentiviral Vector Containing the Human Coagulation Factor IX Gene / 대한수혈학회지
Korean Journal of Blood Transfusion
;
: 1-8, 2008.
Artigo
em Coreano
| WPRIM
| ID: wpr-57133
ABSTRACT
BACKGROUND:
Hemophilia B is an inheritable X-linked bleeding disorder that occurs as a consequence of genetic alterations within the factor IX (IX) gene. In the present study, pseudotyped HIV-I-derived lentiviral vectors expressing human IX (lentivirus-IX) were assessed for the ability to produce an active human IX in the animals transduced with lentivirus-IX.METHODS:
The IX concentrations and activated partial thromboplastin times (aPTT) were measured from the supernatants of HeLa cells that were transduced with lentivirus-IX. In an animal study, we injected 1microgram of lentivirus-IX into the hind limbs of Sparague-Dawley (SD) rats. The IX concentrations were measured from the plasma of the vehicle injected rats and the plasma of the lentivirus-IX injected rats for 8 weeks.RESULTS:
The in vitro expression of human IX was detected in a dose-dependent manner following the transduction of lentivirus-IX into the HeLa cells (control 10+/-3 vs. 100 ng of lentivirus-IX 1486+/-50 ng/mL, P<0.05). The aPTT also showed the tendency of dose-dependent decrease (control 83.9+/-0.5 vs. 50 ng of lentivirus-IX 80.1+/- 0.8 sec), but this was not statistically significant. In the animal experiment, the plasma IX concentration from the lentivirus-IX transduced rats (n=3) was significantly increased compared to the vehicle-injected rats (n=4) (5.9+/-3.9 vs. 46.4+/-20.6 ng/mL) at post-injection 1 week.CONCLUSION:
This study demonstrated that in vivo delivery of lentiviral vectors expressing human IX to the muscle cells has the potential to be a therapeutic modality for hemophilia B.
Texto completo:
DisponíveL
Índice:
WPRIM (Pacífico Ocidental)
Assunto principal:
Plasma
/
Fatores de Coagulação Sanguínea
/
Fator IX
/
Tromboplastina
/
Células HeLa
/
Terapia Genética
/
Hemofilia B
/
Lentivirus
/
Células Musculares
/
Experimentação Animal
Limite:
Animais
/
Humanos
Idioma:
Coreano
Revista:
Korean Journal of Blood Transfusion
Ano de publicação:
2008
Tipo de documento:
Artigo
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