Functional Efficacy of Dystrophin Expression from Plasmids Delivered to mdx Mice by Hydrodynamic Limb Vein Injection
نویسندگان
چکیده
منابع مشابه
Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice
Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise for Duchenne muscular dystrophy (DMD) patients. However, recent failure with drisapersen, an AO candidate drug in phase 3 trial, highlights the importance of exploring other effective AO chemistries for DMD. Previously, we demonstrated the appreciable biological activity of peptide nucleic acid (PNA) AOs in r...
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Duchenne muscular dystrophy is a lethal recessive disease characterized by widespread muscle damage throughout the body. This increases the difficulty of cell or gene therapy based on direct injections into muscles. One way to circumvent this obstacle would be to use circulating cells capable of homing to the sites of lesions. Here, we showed that stem cell antigen 1 (Sca-1), CD34 double-positi...
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Background and Aims: Adenovector gene transfer induces inflammatory response that finally leads to vector removal from delivered site. The effect of hydrodynamic pre-injection on Adenovector mediated liver inflammation remained elusive. Materials and Methods: Different mice groups were pre-treated by hydrodynamic saline, dexamethasone or nothing prior to Adeno-Luciferase administration. Their s...
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Mitochondrial genetic disorders are a major cause of mitochondrial diseases. It is therefore likely that mitochondrial gene therapy will be useful for the treatment of such diseases. Here, we report on the possibility of mitochondrial gene delivery in skeletal muscle using hydrodynamic limb vein (HLV) injection. The HLV injection procedure, a useful method for transgene expression in skeletal m...
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ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2010
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2009.133