Skeletal muscle-specific expression of human blood coagulation factor IX rescues factor IX deficiency mouse by AAV-mediated gene transfer

Lihui Lai, Li Chen, Jianmin Wang, Hong Zhou, Daru Lu, Qi Wang, Xiaobo Gao, Xinfang Qiu, Jinglun Xue

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

The efficacy of recombinant adeno-associated virus (AAV) vector to deliver and express human blood clotting factor IX (hFIX) gene in skeletal muscle of coagulation factor IX deficiency mouse strain (FactorIX-knockout) is evaluated. The muscle creatine kinase enhancer (MCK) and β-actin promoter (βA) were used to drive the hFIX minigene (hFIXm1), which was flanked by AAV inverted terminal repeats (ITRs). Following intramuscular injection of high titer (2.5 × 10 11 vector genomes/mL) of rAAV, increased hFIX expression (256 ng/mL of plasma) was achieved. The time course of hFIX expression demonstrated that the expression level gradually increased over a period of two weeks before anti-hFIX antibodies developed in mouse circulating plasma. Those results provided a promising evidence that rAAV-mediated gene transfer and skeletal muscle-specific expression of hFIX is a feasible strategy for treating patients for hemophilia B.

Original languageEnglish (US)
Pages (from-to)633-634
Number of pages2
JournalScience in China, Series C: Life Sciences
Volume42
Issue number6
StatePublished - Dec 1 1999
Externally publishedYes

Fingerprint

Dependovirus
Gene transfer
Hemophilia B
blood coagulation factors
Factor IX
gene transfer
Blood Coagulation Factors
Viruses
coagulation
Blood Coagulation
Muscle
skeletal muscle
virus
Skeletal Muscle
muscle
blood
Blood
mice
Genes
hemophilia

Keywords

  • AAV
  • Direct-muscular gene therapy
  • Factor IX deficiency mouse
  • hFIX

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • Environmental Science(all)
  • Agricultural and Biological Sciences(all)

Cite this

Skeletal muscle-specific expression of human blood coagulation factor IX rescues factor IX deficiency mouse by AAV-mediated gene transfer. / Lai, Lihui; Chen, Li; Wang, Jianmin; Zhou, Hong; Lu, Daru; Wang, Qi; Gao, Xiaobo; Qiu, Xinfang; Xue, Jinglun.

In: Science in China, Series C: Life Sciences, Vol. 42, No. 6, 01.12.1999, p. 633-634.

Research output: Contribution to journalArticle

Lai, Lihui ; Chen, Li ; Wang, Jianmin ; Zhou, Hong ; Lu, Daru ; Wang, Qi ; Gao, Xiaobo ; Qiu, Xinfang ; Xue, Jinglun. / Skeletal muscle-specific expression of human blood coagulation factor IX rescues factor IX deficiency mouse by AAV-mediated gene transfer. In: Science in China, Series C: Life Sciences. 1999 ; Vol. 42, No. 6. pp. 633-634.
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