Integration-deficient Lentiviral Vectors Expressing Codon-optimized R338L Human FIX Restore Normal Hemostasis in Hemophilia B Mice
نویسندگان
چکیده
منابع مشابه
Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice.
Gene therapy may provide a cure for hemophilia and overcome the limitations of protein replacement therapy. Increasing the potency of gene transfer vectors may allow improvement of their therapeutic index, as lower doses can be administered to achieve therapeutic benefit, reducing toxicity of in vivo administration. Here we generated codon-usage optimized and hyperfunctional factor IX (FIX) tra...
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The development of the next-generation gene therapy vectors for hemophilia requires using lower and thus potentially safer vector doses and augmenting their therapeutic efficacy. We have identified hepatocyte-specific transcriptional cis-regulatory modules (CRMs) by using a computational strategy that increased factor IX (FIX) levels 11- to 15-fold. Vector efficacy could be enhanced by combinin...
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4. Janssens W, Chuah MK, Naldini L, et al. Efficiency of onco-retroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent. Hum Gene Ther. 2003;14(3):263-276. 5. Serafini M, Naldini L, Introna M. Molecular evidence of inefficient transduction of proliferating human B lymphocytes by VSV-pseudotyped HIV-1-derived lentivectors. Virology. 2004;325(2):41...
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2014
ISSN: 1525-0016
DOI: 10.1038/mt.2013.188