Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy

by Bartus, Raymond T, Weinberg, Marc S, Samulski, R. Jude
Published 2014

Global CNS Gene Delivery and Evasion of Anti-AAV Neutralizing Antibodies by Intrathecal AAV Administration in Non-Human Primates

by Gray, Steven J., Kalburgi, Sahana Nagabhushan, McCown, Thomas J., Samulski, R. Jude
Published 2013

Adeno-Associated Viral Vectors Show Serotype Specific Transduction of Equine Joint Tissue Explants and Cultured Monolayers

by Hemphill, Daniel D., McIlwraith, C. Wayne, Samulski, R. Jude, Goodrich, Laurie R.
Published 2014

Three-dimensional multipotent progenitor cell aggregates for expansion, osteogenic differentiation and “in vivo” tracing with AAV vector serotype 6

by Ferreira, Joäo R., Hirsch, Matthew L., Zhang, Li, Park, Yonsil, Samulski, R. Jude, Hu, Wei-Shou, Ko, Ching-Chang
Published 2012

AAV Exploits Subcellular Stress Associated with Inflammation, Endoplasmic Reticulum Expansion, and Misfolded Proteins in Models of Cystic Fibrosis

by Johnson, Jarrod S., Gentzsch, Martina, Zhang, Liqun, Ribeiro, Carla M. P., Kantor, Boris, Kafri, Tal, Pickles, Raymond J., Samulski, R. Jude
Published 2011

In Cellulo Examination of a Beta-Alpha Hybrid Construct of Beta-Hexosaminidase A Subunits, Reported to Interact with the GM2 Activator Protein and Hydrolyze GM2 Ganglioside

by Sinici, Incilay, Yonekawa, Sayuri, Tkachyova, Ilona, Gray, Steven J., Samulski, R. Jude, Wakarchuk, Warren, Mark, Brian L., Mahuran, Don J.
Published 2013

β-Cell–Specific IL-2 Therapy Increases Islet Foxp3+Treg and Suppresses Type 1 Diabetes in NOD Mice

by Johnson, Mark C., Garland, Alaina L., Nicolson, Sarah C., Li, Chengwen, Samulski, R. Jude, Wang, Bo, Tisch, Roland
Published 2013

Adeno-Associated Virus Vector Mediated Delivery of the HBV Genome Induces Chronic Hepatitis B Virus Infection and Liver Fibrosis in Mice

by Ye, Lei, Yu, Haisheng, Li, Chengwen, Hirsch, Matthew L., Zhang, Liguo, Samulski, R. Jude, Li, Wuping, Liu, Zhong
Published 2015

Prediction of Adeno-Associated Virus Neutralizing Antibody Activity for Clinical Application

by Wang, Mei, Crosby, Ashley, Hastie, Eric, Samulski, Jade J., McPhee, Scott, Joshua, Grieger, Samulski, R. Jude, Li, Chengwen
Published 2015

Viral Single-Strand DNA Induces p53-Dependent Apoptosis in Human Embryonic Stem Cells

by Hirsch, Matthew L., Fagan, B. Matthew, Dumitru, Raluca, Bower, Jacquelyn J., Yadav, Swati, Porteus, Matthew H., Pevny, Larysa H., Samulski, R. Jude
Published 2011

Optimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis

by Goodrich, Laurie R, Phillips, Jennifer N, McIlwraith, C Wayne, Foti, Stacey B, Grieger, Joshua C, Gray, Steven J, Samulski, R Jude
Published 2013

Retinal pigment epithelial cell expression of active Rap 1a by scAAV2 inhibits choroidal neovascularization

by Wang, Haibo, Han, Xiaokun, Bretz, Colin A, Becker, Silke, Gambhir, Deeksha, Smith, George W, Samulski, R Jude, Wittchen, Erika S, Quilliam, Lawrence A, Chrzanowska-Wodnicka, Magdalena, Hartnett, M Elizabeth
Published 2016

Safety and biodistribution assessment of sc-rAAV2.5IL-1Ra administered via intra-articular injection in a mono-iodoacetate-induced osteoarthritis rat model

by Wang, Gensheng, Evans, Christopher H, Benson, Janet M, Hutt, Julie A, Seagrave, JeanClare, Wilder, Julie A, Grieger, Joshua C, Samulski, R Jude, Terse, Pramod S
Published 2016

AAV Gene Therapy for MPS1-associated Corneal Blindness

by Vance, Melisa, Llanga, Telmo, Bennett, Will, Woodard, Kenton, Murlidharan, Giridhar, Chungfat, Neil, Asokan, Aravind, Gilger, Brian, Kurtzberg, Joanne, Samulski, R. Jude, Hirsch, Matthew L.
Published 2016

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-Assoc...

by Monahan, Paul E., Sun, Junjiang, Gui, Tong, Hu, Genlin, Hannah, William B., Wichlan, David G., Wu, Zhijian, Grieger, Joshua C., Li, Chengwen, Suwanmanee, Thipparat, Stafford, Darrel W., Booth, Carmen J., Samulski, Jade J., Kafri, Tal, McPhee, Scott W.J., Samulski, R. Jude
Published 2015