Luk Vandenberghe, PhD

Associate Professor of Ophthalmology, Massachusetts Eye and Ear Infirmary
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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Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection.
Authors: Authors: Wassmer SJ, Carvalho LS, György B, Vandenberghe LH, Maguire CA.
Sci Rep
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Progress in gene therapy for neurological disorders.
Authors: Authors: Simonato M, Bennett J, Boulis NM, Castro MG, Fink DJ, Goins WF, Gray SJ, Lowenstein PR, Vandenberghe LH, Wilson TJ, Wolfe JH, Glorioso JC.
Nat Rev Neurol
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