Efficient gene transfer in skeletal muscle with AAV-derived bicistronic vector using the FGF-1 IRES
نویسندگان
چکیده
منابع مشابه
Long term expression of bicistronic vector driven by the FGF-1 IRES in mouse muscle
BACKGROUND Electrotransfer of plasmid DNA into skeletal muscle is a promising strategy for the delivery of therapeutic molecules targeting various muscular diseases, cancer and lower-limb ischemia. Internal Ribosome Entry Sites (IRESs) allow co-expression of proteins of interest from a single transcriptional unit. IRESs are RNA elements that have been found in viral RNAs as well as a variety of...
متن کاملAAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in...
متن کاملAAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells.
In a clinical trial for adeno-associated virus serotype 1 (AAV-1)-mediated gene transfer to muscle for lipoprotein lipase (LPL) deficiency, 1 subject from the high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneously, after an initial downward trend consistent with expression of LPL, plasma triglyceride levels...
متن کاملTranslational efficiency of EMCV IRES in bicistronic vectors is dependent upon IRES sequence and gene location.
The internal ribosomal entry site (IRES)from encephalomyocarditis virus (EMCV) is a popular RNA element used widely in experimental and pharmaceutical applications to express proteins in eukaryotic cells or cell-free extracts. Inclusion of the wild-type element in monocistronic or bicistronic messenger RNAs (mRNAs) confers a high level of cap-independent translation activity to appropriately co...
متن کاملDevelopment of a new bicistronic retroviral vector with strong IRES activity
BACKGROUND Internal Ribosome Entry Site (IRES)-based bicistronic vectors are important tools in today's cell biology. Among applications, the expression of two proteins under the control of a unique promoter permits the monitoring of expression of a protein whose biological function is being investigated through the observation of an easily detectable tracer, such as Green Fluorescent Protein (...
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ژورنال
عنوان ژورنال: Gene Therapy
سال: 2008
ISSN: 0969-7128,1476-5462
DOI: 10.1038/gt.2008.49