Retroviral-mediated transfer and amplification of a functional human factor VIII gene
نویسندگان
چکیده
منابع مشابه
Expression of functional factor VIII in primary human skin fibroblasts after retrovirus-mediated gene transfer.
We have developed a retroviral-vector system for the transfer and expression of a cloned blood clotting factor VIII cDNA. Since inclusion of the complete cDNA into existing vectors is precluded by its large size, we deleted most codons for the B-domain, which is also excised during in vivo maturation of factor VIII. When inserted into the retroviral vector M5-neoR (Laker, C., Stocking, C., Berg...
متن کاملLong-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice.
Hemophilia A is caused by a deficiency in coagulation factor VIII (FVIII) and predisposes to spontaneous bleeding that can be life-threatening or lead to chronic disabilities. It is well suited for gene therapy because a moderate increase in plasma FVIII concentration has therapeutic effects. Improved retroviral vectors expressing high levels of human FVIII were pseudotyped with the vesicular s...
متن کاملTherapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy.
Factor X deficiency results in a rare but serious bleeding disorder that might be treated by expressing a normal factor X gene in patients. We generated an amphotropic retroviral vector with the human FX cDNA and delivered it to rat hepatocytes in vivo during liver regeneration. The human alpha1-antitrypsin promoter was chosen to direct expression because it was the most efficient of several te...
متن کاملBiomaterial-mediated retroviral gene transfer using self-assembled monolayers.
Biomaterial-mediated gene delivery has recently emerged as a promising alternative to conventional gene transfer technologies that focus on direct delivery of viral vectors or DNA-polymer/matrix complexes. However, biomaterial-based strategies have primarily targeted transient gene expression vehicles, including plasmid DNA and adenovirus particles. This study expands on this work by characteri...
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ژورنال
عنوان ژورنال: Blood
سال: 1990
ISSN: 0006-4971,1528-0020
DOI: 10.1182/blood.v75.5.1074.1074