McPhee, Scott W.J.

Dystrophin Immunity in Duchenne's Muscular Dystrophy

Mendell, J. R., Campbell, K., Rodino-Klapac, L., Sahenk, Z., Shilling, C., Lewis, S., Bowles, D., Gray, S., Li, C., Galloway, G., et al. (2010) · New England Journal of Medicine

Muscle Physiology and DisordersVirus-based gene therapy research

Cited by 655

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Bowles, D. E., McPhee, S. W., Li, C., Gray, S. J., Samulski, J. J., Camp, A. S., Li, J., Wang, B., Monahan, P. E., Rabinowitz, J. E., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 400

Long-term follow-up after gene therapy for canavan disease

Leone, P., Shera, D., McPhee, S. W. J., Francis, J. S., Kolodny, E. H., Bilaniuk, L. T., Wang, D.-J., Assadi, M., Goldfarb, O., Goldman, H. W., et al. (2012) · Science Translational Medicine

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 307

Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene ( ASPA ) to the Human Brain

Janson, C., McPhee, S., Bilaniuk, L., Haselgrove, J., Testaiuti, M., Freese, A., Wang, D.-J., Shera, D., Hurh, P., Rupin, J., et al. (2002) · Human Gene Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 255

Immune responses to AAV in a phase I study for Canavan disease

McPhee, S. W. J., Janson, C. G., Li, C., Samulski, R. J., Camp, A. S., Francis, J., Shera, D., Lioutermann, L., Feely, M., Freese, A., et al. (2006) · The Journal of Gene Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 241

BAX 335 hemophilia b gene therapy clinical trial results: Potential impact of CpG sequences on gene expression

Konkle, B. A., Walsh, C. E., Escobar, M. A., Josephson, N. C., Young, G., von Drygalski, A., McPhee, S. W. J., Samulski, R. J., Bilic, I., de la Rosa, M., et al. (2021) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 189

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 Trial

Monahan, P. E., Sun, J., Gui, T., Hu, G., Hannah, W. B., Wichlan, D. G., Wu, Z., Grieger, J. C., Li, C., Suwanmanee, T., et al. (2015) · Human Gene Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 108

Development of patient-specific AAV vectors after neutralizing antibody selection for enhanced muscle gene transfer

Li, C., Wu, S., Albright, B., Hirsch, M., Li, W., Tseng, Y.-S., Agbandje-McKenna, M., McPhee, S., Asokan, A., & Samulski, R. J. (2016) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 48