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Neonatal Systemic AAV-Mediated Gene Delivery of GDF11 Inhibits Skeletal Muscle Growth.

Mol. Ther.. 2018-04; 
JinQuan,QiaoChunping,LiJianbin,LiJuan,Xiao
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Gene Synthesis … Sequences encoding codon-optimized GDF11 and GDF11Pro-Fc-1 were synthesized (GenScript, Piscataway, NJ, USA) and cloned into the AAV backbone under the transcriptional control of the ubiquitous CAG promoter (consisting of the cytomegalovirus [CMV] enhancer … Get A Quote

摘要

Growth and differentiation factor 11 (GDF11; BMP11) is a circulating cytokine in the transforming growth factor beta (TGF-β) superfamily. Treatment with recombinant GDF11 (rGDF11) protein has previously been shown to reverse skeletal muscle dysfunction in aged mice. However, the actions of GDF11 in skeletal muscle are still not fully understood. Because GDF11 activates the TGF-β-SMAD2/3 pathway, we hypothesized that GDF11 overexpression would inhibit skeletal muscle growth. To test this hypothesis, we generated recombinant adeno-associated virus serotype 9 (AAV9) vectors harboring the gene for either human GDF11 (AAV9-GDF11) or human IgG1 Fc-fused GDF11 propeptide (AAV9-GDF11Pro-Fc-1) to study the effec... More

关键词

adeno-associated virus,gene delivery,viral ve