Luk Vandenberghe, PhD

Associate Professor of Ophthalmology, Massachusetts Eye and Ear Infirmary
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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Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing.
Authors: Authors: Vandenberghe LH, Xiao R, Lock M, Lin J, Korn M, Wilson JM.
Hum Gene Ther
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Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines.
Authors: Authors: Lin J, Calcedo R, Vandenberghe LH, Figueredo JM, Wilson JM.
Hum Gene Ther
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Voices of biotech research.
Authors: Authors: Annabi N, Baker M, Boettiger A, Chakraborty D, Chen Y, Corbett KS, Correia B, Dahlman J, de Oliveira T, Ertuerk A, Yanik MF, Henaff E, Huch M, Iliev ID, Jacobs T, Junca H, Keung A, Kolodkin-Gal I, Krishnaswamy S, Lancaster M, Macosko E, Martínez-Núñez MA, Miura K, Molloy J, Cruz AO, Platt RJ, Posey AD, Shao H, Simunovic M, Slavov N, Takebe T, Vandenberghe LH, Varshney RK, Wang J.
Nat Biotechnol
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FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.
Authors: Authors: Manoli I, Sysol JR, Epping MW, Li L, Wang C, Sloan JL, Pass A, Gagné J, Ktena YP, Li L, Trivedi NS, Ouattara B, Zerfas PM, Hoffmann V, Abu-Asab M, Tsokos MG, Kleiner DE, Garone C, Cusmano-Ozog K, Enns GM, Vernon HJ, Andersson HC, Grunewald S, Elkahloun AG, Girard CL, Schnermann J, DiMauro S, Andres-Mateos E, Vandenberghe LH, Chandler RJ, Venditti CP.
JCI Insight
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A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear.
Authors: Authors: Landegger LD, Pan B, Askew C, Wassmer SJ, Gluck SD, Galvin A, Taylor R, Forge A, Stankovic KM, Holt JR, Vandenberghe LH.
Nat Biotechnol
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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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