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Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

Molecular therapy. Methods & clinical development. 2020-09; 
Piras G,Montiel-Equihua C,Chan YA,Wantuch S,Stuckey D,Burke D,Prunty H,Phadke R,Chambers D,Partida-Gaytan A,Leon-Rico D,Panchal N,Whitmore K,Calero M,Benedetti S,Santilli G,Thrasher AJ,Gaspar HB
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Codon Optimization An hGAA cDNA sequence was codon optimized and commercially synthesized by GenScript Biotech. Get A Quote

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