Luk Vandenberghe, PhD

Associate Professor of Ophthalmology, Massachusetts Eye and Ear Infirmary
Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.
Authors: Authors: O'Reilly M, Federoff HJ, Fong Y, Kohn DB, Patterson AP, Ahmed N, Asokan A, Boye SE, Crystal RG, De Oliveira S, Gargiulo L, Harper SQ, Ikeda Y, Jambou R, Montgomery M, Prograis L, Rosenthal E, Sterman DH, Vandenberghe LH, Zoloth L, Abedi M, Adair J, Adusumilli PS, Goins WF, Gray J, Monahan P, Popplewell L, Sena-Esteves M, Tannous B, Weber T, Wierda W, Gopal-Srivastava R, McDonald CL, Rosenblum D, Corrigan-Curay J.
Hum Gene Ther
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Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis.
Authors: Authors: Kassim SH, Li H, Vandenberghe LH, Hinderer C, Bell P, Marchadier D, Wilson A, Cromley D, Redon V, Yu H, Wilson JM, Rader DJ.
PLoS One
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Tailoring the AAV vector capsid for gene therapy.
Authors: Authors: Vandenberghe LH, Wilson JM, Gao G.
Gene Ther
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Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.
Authors: Authors: Hudry E, Vandenberghe LH.
Neuron
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Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction.
Authors: Authors: Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC.
Sci Rep
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The structure of AAVrh32.33, a novel gene delivery vector.
Authors: Authors: Mikals K, Nam HJ, Van Vliet K, Vandenberghe LH, Mays LE, McKenna R, Wilson JM, Agbandje-McKenna M.
J Struct Biol
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Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale.
Authors: Authors: Lock M, Alvira M, Vandenberghe LH, Samanta A, Toelen J, Debyser Z, Wilson JM.
Hum Gene Ther
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Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain.
Authors: Authors: Cearley CN, Vandenberghe LH, Parente MK, Carnish ER, Wilson JM, Wolfe JH.
Mol Ther
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MCOLN1 gene therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV.
Authors: Authors: DeRosa S, Salani M, Smith S, Sangster M, Miller-Browne V, Wassmer S, Xiao R, Vandenberghe L, Slaugenhaupt S, Misko A, Grishchuk Y.
Hum Mol Genet
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Quantitative and Digital Droplet-Based AAV Genome Titration.
Authors: Authors: Sanmiguel J, Gao G, Vandenberghe LH.
Methods Mol Biol
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