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Current Opinion in Molecular Therapeutics|October 2, 2010
Adeno-associated virus for the treatment of muscle diseases: toward clinical trialsNina DiPrimio, Scott W J McPhee, R Jude SamulskiStereotactic and Functional Neurosurgery|August 19, 2011
Assessment of hippocampal adeno-associated viral vector gene delivery via frameless stereotaxis in a nonhuman primateCary H Leung, Michele A Kliem, Brenten L Heeke, et al.Science Translational Medicine|December 21, 2012
Long-term follow-up after gene therapy for canavan diseasePaola Leone, David Shera, Scott W J McPhee, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|November 10, 2011
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vectorDawn E Bowles, Scott W J McPhee, Chengwen Li, et al.Human Gene Therapy|November 25, 2014
Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trialPaul E Monahan, Junjiang Sun, Tong Gui, et al.Blood|October 17, 2020
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expressionBarbara A Konkle, Christopher E Walsh, Miguel A Escobar, et al.Pageof 1