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Luk Hugo Vandenberghe, Ph.D.

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The Vandenberghe laboratory deconstructs viral evolution to improve viral vector design and use in gene therapy. We aim to more profoundly understand capsid structure-function relationships and use this information to select and engineer gene therapy vectors for clinical indications.

For many of these studies, we use the ssDNA mammalian adeno-associated virus (AAV) as a model. AAV is not only one of the smallest and simplest mammalian viruses, it has also captivated the world of gene therapy as AAV, with remarkable efficiency and safety, is able to deliver and transduce genetic cargo into therapeutic target tissues such as the retina, cochlea, liver, and CNS. AAVs come in many flavors that were originally distinguished based on serology, but now are more and more phylogenetically and structurally defined. Different AAVs demonstrate very distinct phenotypes (tropism, receptor use, host response, assembly, etc.) as a wild type virus or replication-defective vector. We are interested in functionally and structurally understanding the mechanism of these stark differences from the perspective of virus as well as host. One avenue towards this goal is to study the evolutionary biology of this virus, and use mathematical, statistical, and systems approaches in combination with empirical and molecular methods to shed light on the structure-function relationship of these small, yet intricately complex infectious protein assemblies.

We aim at integrating these findings into improved technologies for therapeutic gene transfer, and use these findings and novel reagents toward building innovative therapies for diseases with unmet need, with a particular focus on blinding and hearing disorders.

lab webpage: http://www.vdb-lab.org/

Gene Transfer Vector Core: http://vector.meei.harvard.edu/

Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
  1. Naninck T, Kahlaoui N, Lemaitre J, Maisonnasse P, De Mori A, Pascal Q, Contreras V, Marlin R, Relouzat F, Delache B, Hérate C, Aldon Y, van Gils M, Zabaleta N, Ho Tsong Fang R, Bosquet N, Sanders RW, Vandenberghe LH, Chapon C, Le Grand R. Computed tomography and [18F]-FDG PET imaging provide additional readouts for COVID-19 pathogenesis and therapies evaluation in non-human primates. iScience. 2022 Apr 15; 25(4):104101. PMID: 35313622.
  2. Andres-Mateos E, Landegger LD, Unzu C, Phillips J, Lin BM, Dewyer NA, Sanmiguel J, Nicolaou F, Valero MD, Bourdeu KI, Sewell WF, Beiler RJ, McKenna MJ, Stankovic KM, Vandenberghe LH. Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate. Nat Commun. 2022 Mar 15; 13(1):1359. PMID: 35292639.
    Citations:    Fields:    
  3. Grotz S, Schäfer J, Wunderlich KA, Ellederova Z, Auch H, Bähr A, Runa-Vochozkova P, Fadl J, Arnold V, Ardan T, Veith M, Santamaria G, Dhom G, Hitzl W, Kessler B, Eckardt C, Klein J, Brymova A, Linnert J, Kurome M, Zakharchenko V, Fischer A, Blutke A, Döring A, Suchankova S, Popelar J, Rodríguez-Bocanegra E, Dlugaiczyk J, Straka H, May-Simera H, Wang W, Laugwitz KL, Vandenberghe LH, Wolf E, Nagel-Wolfrum K, Peters T, Motlik J, Fischer MD, Wolfrum U, Klymiuk N. Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes. EMBO Mol Med. 2022 Apr 07; 14(4):e14817. PMID: 35254721.
    Citations:    Fields:    
  4. Wang J, Zhao L, Gu X, Xue Y, Wang S, Xiao R, Vandenberghe L, Peng KA, Shu Y, Li H. Efficient delivery of adeno-associated virus (AAV) into inner ear in vivo via trans-stapes route in adult guinea pig. Hum Gene Ther. 2022 Feb 14. PMID: 35156857.
    Citations:    Fields:    
  5. Zolotukhin S, Vandenberghe LH. AAV capsid design: A Goldilocks challenge. Trends Mol Med. 2022 03; 28(3):183-193. PMID: 35093287.
    Citations:    Fields:    
  6. Chekuri A, Logan EM, Krauson AJ, Salani M, Ackerman S, Kirchner EG, Bolduc JM, Wang X, Dietrich P, Dragatsis I, Vandenberghe LH, Slaugenhaupt SA, Morini E. Selective retinal ganglion cell loss and optic neuropathy in a humanized mouse model of familial dysautonomia. Hum Mol Genet. 2021 Dec 15. PMID: 34908112.
    Citations:    Fields:    
  7. Zabaleta N, Dai W, Bhatt U, Hérate C, Maisonnasse P, Chichester JA, Sanmiguel J, Estelien R, Michalson KT, Diop C, Maciorowski D, Dereuddre-Bosquet N, Cavarelli M, Gallouët AS, Naninck T, Kahlaoui N, Lemaitre J, Qi W, Hudspeth E, Cucalon A, Dyer CD, Pampena MB, Knox JJ, LaRocque RC, Charles RC, Li D, Kim M, Sheridan A, Storm N, Johnson RI, Feldman J, Hauser BM, Contreras V, Marlin R, Tsong Fang RH, Chapon C, van der Werf S, Zinn E, Ryan A, Kobayashi DT, Chauhan R, McGlynn M, Ryan ET, Schmidt AG, Price B, Honko A, Griffiths A, Yaghmour S, Hodge R, Betts MR, Freeman MW, Wilson JM, Le Grand R, Vandenberghe LH. An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates. Cell Host Microbe. 2021 09 08; 29(9):1437-1453.e8. PMID: 34428428.
    Citations: 13     Fields:    Translation:HumansAnimalsCellsPHPublic Health
  8. Ilyinskii PO, Michaud AM, Rizzo GL, Roy CJ, Leung SS, Elkins SL, Capela T, Chowdhury A, Li L, Chandler RJ, Manoli I, Andres-Mateos E, Johnston LPM, Vandenberghe LH, Venditti CP, Kishimoto TK. ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia. Mol Ther Methods Clin Dev. 2021 Sep 10; 22:279-292. PMID: 34485611.
  9. Katz MG, Hadas Y, Bailey RA, Fazal S, Vincek A, Madjarova SJ, Shtraizent N, Vandenberghe LH, Eliyahu E. Efficient cardiac gene transfer and early-onset expression of a synthetic adeno-associated viral vector, Anc80L65, after intramyocardial administration. J Thorac Cardiovasc Surg. 2021 Jun 17. PMID: 34985414.
    Citations:    Fields:    
  10. DeRosa S, Salani M, Smith S, Sangster M, Miller-Browne V, Wassmer S, Xiao R, Vandenberghe L, Slaugenhaupt S, Misko A, Grishchuk Y. MCOLN1 gene therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV. Hum Mol Genet. 2021 05 29; 30(10):908-922. PMID: 33822942.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  11. 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. Voices of biotech research. Nat Biotechnol. 2021 03; 39(3):281-286. PMID: 33692517.
    Citations:    Fields:    Translation:Humans
  12. Zabaleta N, Dai W, Bhatt U, Chichester JA, Sanmiguel J, Estelien R, Michalson KT, Diop C, Maciorowski D, Qi W, Hudspeth E, Cucalon A, Dyer CD, Pampena MB, Knox JJ, LaRocque RC, Charles RC, Li D, Kim M, Sheridan A, Storm N, Johnson RI, Feldman J, Hauser BM, Zinn E, Ryan A, Kobayashi DT, Chauhan R, McGlynn M, Ryan ET, Schmidt AG, Price B, Honko A, Griffiths A, Yaghmour S, Hodge R, Betts MR, Freeman MW, Wilson JM, Vandenberghe LH. Immunogenicity of an AAV-based, room-temperature stable, single dose COVID-19 vaccine in mice and non-human primates. bioRxiv. 2021 Jan 19. PMID: 33442684.
  13. Jacob A, Brun L, Jiménez Gil P, Ménard L, Bouzelha M, Broucque F, Roblin A, Vandenberghe LH, Adjali O, Robin C, François A, Blouin V, Penaud-Budloo M, Ayuso E. Homologous Recombination Offers Advantages over Transposition-Based Systems to Generate Recombinant Baculovirus for Adeno-Associated Viral Vector Production. Biotechnol J. 2021 Jan; 16(1):e2000014. PMID: 33067902.
    Citations: 1     Fields:    Translation:Cells
  14. Greenwald SH, Brown EE, Scandura MJ, Hennessey E, Farmer R, Pawlyk BS, Xiao R, Vandenberghe LH, Pierce EA. Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration. Mol Ther Methods Clin Dev. 2020 Sep 11; 18:582-594. PMID: 32775493.
    Citations: 4     
  15. Hu CJ, Lu YC, Tsai YH, Cheng HY, Takeda H, Huang CY, Xiao R, Hsu CJ, Tsai JW, Vandenberghe LH, Wu CC, Cheng YF. Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65. Mol Ther Methods Clin Dev. 2020 Sep 11; 18:493-500. PMID: 32775487.
    Citations: 6     
  16. Vandenberghe LH. COVID-19: Gene Transfer to the Rescue? Hum Gene Ther. 2020 06; 31(11-12):605-607. PMID: 32469280.
    Citations: 3     Fields:    Translation:HumansAnimals
  17. Omichi R, Yoshimura H, Shibata SB, Vandenberghe LH, Smith RJH. Hair Cell Transduction Efficiency of Single- and Dual-AAV Serotypes in Adult Murine Cochleae. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:1167-1177. PMID: 32518805.
    Citations: 7     
  18. Schmit PF, Pacouret S, Zinn E, Telford E, Nicolaou F, Broucque F, Andres-Mateos E, Xiao R, Penaud-Budloo M, Bouzelha M, Jaulin N, Adjali O, Ayuso E, Vandenberghe LH. Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:107-121. PMID: 31909084.
    Citations: 3     
  19. Subramanian S, Maurer AC, Bator CM, Makhov AM, Conway JF, Turner KB, Marden JH, Vandenberghe LH, Hafenstein SL. Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy. Hum Gene Ther. 2019 12; 30(12):1449-1460. PMID: 31530236.
    Citations: 5     Fields:    Translation:HumansCells
  20. Dudek AM, Zabaleta N, Zinn E, Pillay S, Zengel J, Porter C, Franceschini JS, Estelien R, Carette JE, Zhou GL, Vandenberghe LH. GPR108 Is a Highly Conserved AAV Entry Factor. Mol Ther. 2020 02 05; 28(2):367-381. PMID: 31784416.
    Citations: 22     Fields:    Translation:HumansAnimalsCells
  21. Goldstein JM, Tabebordbar M, Zhu K, Wang LD, Messemer KA, Peacker B, Ashrafi Kakhki S, Gonzalez-Celeiro M, Shwartz Y, Cheng JKW, Xiao R, Barungi T, Albright C, Hsu YC, Vandenberghe LH, Wagers AJ. In Situ Modification of Tissue Stem and Progenitor Cell Genomes. Cell Rep. 2019 04 23; 27(4):1254-1264.e7. PMID: 31018138.
    Citations: 19     Fields:    Translation:HumansAnimalsCells
  22. Vandenberghe LH. AAV Engineering Identifies a Species Barrier That Highlights a Portal to the Brain. Mol Ther. 2019 05 08; 27(5):901-903. PMID: 31000409.
    Citations: 2     Fields:    Translation:Cells
  23. Hudry E, Vandenberghe LH. Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality. Neuron. 2019 Apr 03; 102(1):263. PMID: 30946822.
    Citations: 21     Fields:    
  24. Maurer AC, Cepeda Diaz AK, Vandenberghe LH. Residues on Adeno-associated Virus Capsid Lumen Dictate Interactions and Compatibility with the Assembly-Activating Protein. J Virol. 2019 04 01; 93(7). PMID: 30651367.
    Citations: 5     Fields:    Translation:HumansCells
  25. Hudry E, Vandenberghe LH. Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality. Neuron. 2019 03 06; 101(5):839-862. PMID: 30844402.
    Citations: 76     Fields:    Translation:HumansAnimalsCells
  26. Sanmiguel J, Gao G, Vandenberghe LH. Quantitative and Digital Droplet-Based AAV Genome Titration. Methods Mol Biol. 2019; 1950:51-83. PMID: 30783968.
    Citations: 10     Fields:    Translation:HumansCells
  27. 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. FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia. JCI Insight. 2018 12 06; 3(23). PMID: 30518688.
    Citations: 17     Fields:    Translation:HumansAnimalsCells
  28. Shen J, Xiao R, Bair J, Wang F, Vandenberghe LH, Dartt D, Baranov P, Ng YSE. Novel engineered, membrane-localized variants of vascular endothelial growth factor (VEGF) protect retinal ganglion cells: a proof-of-concept study. Cell Death Dis. 2018 10 03; 9(10):1018. PMID: 30282966.
    Citations: 4     Fields:    Translation:HumansAnimalsCells
  29. Miller JW, Vandenberghe LH. Breaking and Sealing Barriers in Retinal Gene Therapy. Mol Ther. 2018 09 05; 26(9):2081-2082. PMID: 30107998.
    Citations: 6     Fields:    Translation:Humans
  30. Hudry E, Andres-Mateos E, Lerner EP, Volak A, Cohen O, Hyman BT, Maguire CA, Vandenberghe LH. Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65. Mol Ther Methods Clin Dev. 2018 Sep 21; 10:197-209. PMID: 30109242.
    Citations: 27     
  31. Ikeda Y, Sun Z, Ru X, Vandenberghe LH, Humphreys BD. Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector. J Am Soc Nephrol. 2018 09; 29(9):2287-2297. PMID: 29976586.
    Citations: 13     Fields:    Translation:HumansAnimalsCells
  32. Maurer AC, Pacouret S, Cepeda Diaz AK, Blake J, Andres-Mateos E, Vandenberghe LH. The Assembly-Activating Protein Promotes Stability and Interactions between AAV's Viral Proteins to Nucleate Capsid Assembly. Cell Rep. 2018 05 08; 23(6):1817-1830. PMID: 29742436.
    Citations: 12     Fields:    Translation:HumansCells
  33. Carvalho LS, Xiao R, Wassmer SJ, Langsdorf A, Zinn E, Pacouret S, Shah S, Comander JI, Kim LA, Lim L, Vandenberghe LH. Synthetic Adeno-Associated Viral Vector Efficiently Targets Mouse and Nonhuman Primate Retina In Vivo. Hum Gene Ther. 2018 07; 29(7):771-784. PMID: 29325457.
    Citations: 16     Fields:    Translation:HumansAnimalsCells
  34. Dudek AM, Pillay S, Puschnik AS, Nagamine CM, Cheng F, Qiu J, Carette JE, Vandenberghe LH. An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor. J Virol. 2018 04 01; 92(7). PMID: 29343568.
    Citations: 39     Fields:    Translation:AnimalsCells
  35. Tao Y, Huang M, Shu Y, Ruprecht A, Wang H, Tang Y, Vandenberghe LH, Wang Q, Gao G, Kong WJ, Chen ZY. Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction. Hum Gene Ther. 2018 04; 29(4):492-506. PMID: 29130354.
    Citations: 26     Fields:    Translation:HumansAnimalsCells
  36. Carvalho LS, Turunen HT, Wassmer SJ, Luna-Velez MV, Xiao R, Bennett J, Vandenberghe LH. Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting. Front Neurosci. 2017; 11:503. PMID: 28943836.
    Citations: 23     
  37. Wang L, Xiao R, Andres-Mateos E, Vandenberghe LH. Single stranded adeno-associated virus achieves efficient gene transfer to anterior segment in the mouse eye. PLoS One. 2017; 12(8):e0182473. PMID: 28763501.
    Citations: 14     Fields:    Translation:HumansAnimalsCells
  38. Huang X, Zhou G, Wu W, Duan Y, Ma G, Song J, Xiao R, Vandenberghe L, Zhang F, D'Amore PA, Lei H. Genome editing abrogates angiogenesis in vivo. Nat Commun. 2017 07 24; 8(1):112. PMID: 28740073.
    Citations: 43     Fields:    Translation:HumansAnimalsCells
  39. Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC. Corrigendum: Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction. Sci Rep. 2017 05 22; 7:46827. PMID: 28530226.
    Citations: 4     Fields:    
  40. Pacouret S, Bouzelha M, Shelke R, Andres-Mateos E, Xiao R, Maurer A, Mevel M, Turunen H, Barungi T, Penaud-Budloo M, Broucque F, Blouin V, Moullier P, Ayuso E, Vandenberghe LH. AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations. Mol Ther. 2017 06 07; 25(6):1375-1386. PMID: 28427840.
    Citations: 22     Fields:    Translation:HumansCells
  41. Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC. Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction. Sci Rep. 2017 04 03; 7:45524. PMID: 28367981.
    Citations: 66     Fields:    Translation:AnimalsCells
  42. Wassmer SJ, Carvalho LS, György B, Vandenberghe LH, Maguire CA. Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection. Sci Rep. 2017 03 31; 7:45329. PMID: 28361998.
    Citations: 45     Fields:    Translation:AnimalsCells
  43. Landegger LD, Pan B, Askew C, Wassmer SJ, Gluck SD, Galvin A, Taylor R, Forge A, Stankovic KM, Holt JR, Vandenberghe LH. A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear. Nat Biotechnol. 2017 03; 35(3):280-284. PMID: 28165475.
    Citations: 111     Fields:    Translation:AnimalsCells
  44. Pan B, Askew C, Galvin A, Heman-Ackah S, Asai Y, Indzhykulian AA, Jodelka FM, Hastings ML, Lentz JJ, Vandenberghe LH, Holt JR, Géléoc GS. Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c. Nat Biotechnol. 2017 03; 35(3):264-272. PMID: 28165476.
    Citations: 113     Fields:    Translation:Animals
  45. Garanto A, Chung DC, Duijkers L, Corral-Serrano JC, Messchaert M, Xiao R, Bennett J, Vandenberghe LH, Collin RW. In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery. Hum Mol Genet. 2016 06 15; 25(12):2552-2563. PMID: 27106101.
    Citations: 57     Fields:    Translation:HumansAnimalsCells
  46. Carvalho LS, Vandenberghe LH. Understanding Cone Photoreceptor Cell Death in Achromatopsia. Adv Exp Med Biol. 2016; 854:231-6. PMID: 26427416.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  47. Castle MJ, Turunen HT, Vandenberghe LH, Wolfe JH. Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids. Methods Mol Biol. 2016; 1382:133-49. PMID: 26611584.
    Citations: 50     Fields:    Translation:HumansAnimalsCells
  48. Tabebordbar M, Zhu K, Cheng JKW, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM, Wagers AJ. In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science. 2016 Jan 22; 351(6271):407-411. PMID: 26721686.
    Citations: 431     Fields:    Translation:AnimalsCells
  49. Zinn E, Pacouret S, Khaychuk V, Turunen HT, Carvalho LS, Andres-Mateos E, Shah S, Shelke R, Maurer AC, Plovie E, Xiao R, Vandenberghe LH. In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector. Cell Rep. 2015 Aug 11; 12(6):1056-68. PMID: 26235624.
    Citations: 120     Fields:    Translation:Cells
  50. Vandenberghe LH. What Is Next for Retinal Gene Therapy? Cold Spring Harb Perspect Med. 2015 Apr 15; 5(10). PMID: 25877395.
    Citations: 6     Fields:    Translation:Humans
  51. Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK. Perspectives on best practices for gene therapy programs. Hum Gene Ther. 2015 Mar; 26(3):127-33. PMID: 25654329.
    Citations: 6     Fields:    Translation:Humans
  52. Cronin T, Vandenberghe LH, Hantz P, Juttner J, Reimann A, Kacsó AE, Huckfeldt RM, Busskamp V, Kohler H, Lagali PS, Roska B, Bennett J. Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter. EMBO Mol Med. 2014 Sep; 6(9):1175-90. PMID: 25092770.
    Citations: 64     Fields:    Translation:HumansAnimalsCells
  53. Zinn E, Vandenberghe LH. Adeno-associated virus: fit to serve. Curr Opin Virol. 2014 Oct; 8:90-7. PMID: 25128609.
    Citations: 41     Fields:    Translation:HumansCells
  54. Carvalho LS, Vandenberghe LH. Promising and delivering gene therapies for vision loss. Vision Res. 2015 Jun; 111(Pt B):124-33. PMID: 25094052.
    Citations: 16     Fields:    Translation:HumansCells
  55. Bell P, Vandenberghe LH, Wilson JM. Formation of newly synthesized adeno-associated virus capsids in the cell nucleus. Hum Gene Ther Methods. 2014 Jun; 25(3):179-80. PMID: 24933465.
    Citations:    Fields:    Translation:HumansCells
  56. 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. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. Hum Gene Ther. 2014 Jun; 25(6):488-97. PMID: 24773122.
    Citations: 5     Fields:    Translation:HumansAnimals
  57. Mikals K, Nam HJ, Van Vliet K, Vandenberghe LH, Mays LE, McKenna R, Wilson JM, Agbandje-McKenna M. The structure of AAVrh32.33, a novel gene delivery vector. J Struct Biol. 2014 May; 186(2):308-17. PMID: 24704217.
    Citations: 17     Fields:    Translation:HumansCells
  58. Simonato M, Bennett J, Boulis NM, Castro MG, Fink DJ, Goins WF, Gray SJ, Lowenstein PR, Vandenberghe LH, Wilson TJ, Wolfe JH, Glorioso JC. Progress in gene therapy for neurological disorders. Nat Rev Neurol. 2013 May 14. PMID: 23670108.
  59. Simonato M, Bennett J, Boulis NM, Castro MG, Fink DJ, Goins WF, Gray SJ, Lowenstein PR, Vandenberghe LH, Wilson TJ, Wolfe JH, Glorioso JC. Progress in gene therapy for neurological disorders. Nat Rev Neurol. 2013 May; 9(5):277-91. PMID: 23609618.
    Citations: 81     Fields:    Translation:Humans
  60. Cepko CL, Vandenberghe LH. Retinal gene therapy coming of age. Hum Gene Ther. 2013 Mar; 24(3):242-4. PMID: 23458444.
    Citations: 24     Fields:    
  61. Vandenberghe LH, Bell P, Maguire AM, Xiao R, Hopkins TB, Grant R, Bennett J, Wilson JM. AAV9 targets cone photoreceptors in the nonhuman primate retina. PLoS One. 2013; 8(1):e53463. PMID: 23382846.
    Citations: 45     Fields:    Translation:AnimalsCells
  62. Lagor WR, Johnston JC, Lock M, Vandenberghe LH, Rader DJ. Adeno-associated viruses as liver-directed gene delivery vehicles: focus on lipoprotein metabolism. Methods Mol Biol. 2013; 1027:273-307. PMID: 23912992.
    Citations: 13     Fields:    Translation:AnimalsCells
  63. Holt JR, Vandenberghe LH. Gene therapy for deaf mice goes viral. Mol Ther. 2012 Oct; 20(10):1836-7. PMID: 23023054.
    Citations: 4     Fields:    Translation:Animals
  64. Mason JB, Vandenberghe LH, Xiao R, Wilson JM, Richardson DW. Influence of serotype, cell type, tissue composition, and time after inoculation on gene expression in recombinant adeno-associated viral vector-transduced equine joint tissues. Am J Vet Res. 2012 Aug; 73(8):1178-85. PMID: 22849678.
    Citations: 5     Fields:    Translation:AnimalsCells
  65. Vandenberghe LH, Auricchio A. Novel adeno-associated viral vectors for retinal gene therapy. Gene Ther. 2012 Feb; 19(2):162-8. PMID: 21993172.
    Citations: 62     Fields:    Translation:HumansAnimalsCells
  66. Aleman TS, Cideciyan AV, Aguirre GK, Huang WC, Mullins CL, Roman AJ, Sumaroka A, Olivares MB, Tsai FF, Schwartz SB, Vandenberghe LH, Limberis MP, Stone EM, Bell P, Wilson JM, Jacobson SG. Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model. Invest Ophthalmol Vis Sci. 2011 Aug 29; 52(9):6898-910. PMID: 21757580.
    Citations: 53     Fields:    Translation:HumansAnimals
  67. Vandenberghe LH, Bell P, Maguire AM, Cearley CN, Xiao R, Calcedo R, Wang L, Castle MJ, Maguire AC, Grant R, Wolfe JH, Wilson JM, Bennett J. Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey. Sci Transl Med. 2011 Jun 22; 3(88):88ra54. PMID: 21697530.
    Citations: 97     Fields:    Translation:HumansAnimalsCells
  68. Kassim SH, Vandenberghe LH, Hovhannisyan R, Wilson JM, Rader DJ. Identification and functional characterization in vivo of a novel splice variant of LDLR in rhesus macaques. Physiol Genomics. 2011 Aug 16; 43(15):911-6. PMID: 21628398.
    Citations: 3     Fields:    Translation:HumansAnimalsCells
  69. Bell CL, Vandenberghe LH, Bell P, Limberis MP, Gao GP, Van Vliet K, Agbandje-McKenna M, Wilson JM. The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice. J Clin Invest. 2011 Jun; 121(6):2427-35. PMID: 21576824.
    Citations: 89     Fields:    Translation:AnimalsCells
  70. Bell P, Gao G, Haskins ME, Wang L, Sleeper M, Wang H, Calcedo R, Vandenberghe LH, Chen SJ, Weisse C, Withnall E, Wilson JM. Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs. Hum Gene Ther. 2011 Aug; 22(8):985-97. PMID: 21204705.
    Citations: 26     Fields:    Translation:AnimalsCells
  71. Van der Perren A, Toelen J, Carlon M, Van den Haute C, Coun F, Heeman B, Reumers V, Vandenberghe LH, Wilson JM, Debyser Z, Baekelandt V. Efficient and stable transduction of dopaminergic neurons in rat substantia nigra by rAAV 2/1, 2/2, 2/5, 2/6.2, 2/7, 2/8 and 2/9. Gene Ther. 2011 May; 18(5):517-27. PMID: 21326331.
    Citations: 38     Fields:    Translation:AnimalsCells
  72. Kassim SH, Li H, Vandenberghe LH, Hinderer C, Bell P, Marchadier D, Wilson A, Cromley D, Redon V, Yu H, Wilson JM, Rader DJ. Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis. PLoS One. 2010 Oct 19; 5(10):e13424. PMID: 20976059.
    Citations: 33     Fields:    Translation:HumansAnimalsCells
  73. Vandenberghe LH, Xiao R, Lock M, Lin J, Korn M, Wilson JM. Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. Hum Gene Ther. 2010 Oct; 21(10):1251-7. PMID: 20649475.
    Citations: 52     Fields:    Translation:HumansAnimalsCells
  74. Lock M, Alvira M, Vandenberghe LH, Samanta A, Toelen J, Debyser Z, Wilson JM. Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther. 2010 Oct; 21(10):1259-71. PMID: 20497038.
    Citations: 154     Fields:    Translation:HumansCells
  75. Carlon M, Toelen J, Van der Perren A, Vandenberghe LH, Reumers V, Sbragia L, Gijsbers R, Baekelandt V, Himmelreich U, Wilson JM, Deprest J, Debyser Z. Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2. Mol Ther. 2010 Dec; 18(12):2130-8. PMID: 20664525.
    Citations: 12     Fields:    Translation:AnimalsCells
  76. Vandenberghe LH, Breous E, Nam HJ, Gao G, Xiao R, Sandhu A, Johnston J, Debyser Z, Agbandje-McKenna M, Wilson JM. Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints. Gene Ther. 2009 Dec; 16(12):1416-28. PMID: 19727141.
    Citations: 37     Fields:    Translation:AnimalsCells
  77. Wang L, Calcedo R, Wang H, Bell P, Grant R, Vandenberghe LH, Sanmiguel J, Morizono H, Batshaw ML, Wilson JM. The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther. 2010 Jan; 18(1):126-34. PMID: 19888196.
    Citations: 70     Fields:    Translation:AnimalsCells
  78. Wang L, Wang H, Bell P, McCarter RJ, He J, Calcedo R, Vandenberghe LH, Morizono H, Batshaw ML, Wilson JM. Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol Ther. 2010 Jan; 18(1):118-25. PMID: 19861950.
    Citations: 76     Fields:    Translation:AnimalsCells
  79. Lin J, Calcedo R, Vandenberghe LH, Bell P, Somanathan S, Wilson JM. A new genetic vaccine platform based on an adeno-associated virus isolated from a rhesus macaque. J Virol. 2009 Dec; 83(24):12738-50. PMID: 19812149.
    Citations: 31     Fields:    Translation:HumansAnimalsCells
  80. Gao G, Wang Q, Calcedo R, Mays L, Bell P, Wang L, Vandenberghe LH, Grant R, Sanmiguel J, Furth EE, Wilson JM. Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Hum Gene Ther. 2009 Sep; 20(9):930-42. PMID: 19441963.
    Citations: 56     Fields:    Translation:AnimalsCells
  81. Breous E, Somanathan S, Vandenberghe LH, Wilson JM. Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver. Hepatology. 2009 Aug; 50(2):612-21. PMID: 19575456.
    Citations: 98     Fields:    Translation:HumansAnimalsCells
  82. Roy S, Vandenberghe LH, Kryazhimskiy S, Grant R, Calcedo R, Yuan X, Keough M, Sandhu A, Wang Q, Medina-Jaszek CA, Plotkin JB, Wilson JM. Isolation and characterization of adenoviruses persistently shed from the gastrointestinal tract of non-human primates. PLoS Pathog. 2009 Jul; 5(7):e1000503. PMID: 19578438.
    Citations: 73     Fields:    Translation:AnimalsCells
  83. Mays LE, Vandenberghe LH, Xiao R, Bell P, Nam HJ, Agbandje-McKenna M, Wilson JM. Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins. J Immunol. 2009 May 15; 182(10):6051-60. PMID: 19414756.
    Citations: 50     Fields:    Translation:AnimalsCells
  84. Song Y, Lou HH, Boyer JL, Limberis MP, Vandenberghe LH, Hackett NR, Leopold PL, Wilson JM, Crystal RG. Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. Hum Gene Ther. 2009 Mar; 20(3):267-81. PMID: 19257851.
    Citations: 25     Fields:    Translation:HumansAnimalsCells
  85. Calcedo R, Vandenberghe LH, Gao G, Lin J, Wilson JM. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis. 2009 Feb 01; 199(3):381-90. PMID: 19133809.
    Citations: 328     Fields:    Translation:HumansCells
  86. Calcedo R, Vandenberghe LH, Roy S, Somanathan S, Wang L, Wilson JM. Host immune responses to chronic adenovirus infections in human and nonhuman primates. J Virol. 2009 Mar; 83(6):2623-31. PMID: 19116257.
    Citations: 35     Fields:    Translation:HumansAnimalsCells
  87. Limberis MP, Vandenberghe LH, Zhang L, Pickles RJ, Wilson JM. Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol Ther. 2009 Feb; 17(2):294-301. PMID: 19066597.
    Citations: 80     Fields:    Translation:HumansAnimalsCells
  88. Vandenberghe LH, Wilson JM, Gao G. Tailoring the AAV vector capsid for gene therapy. Gene Ther. 2009 Mar; 16(3):311-9. PMID: 19052631.
    Citations: 74     Fields:    Translation:HumansAnimalsCells
  89. Cearley CN, Vandenberghe LH, Parente MK, Carnish ER, Wilson JM, Wolfe JH. Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol Ther. 2008 Oct; 16(10):1710-8. PMID: 18714307.
    Citations: 97     Fields:    Translation:AnimalsCells
  90. Lin J, Calcedo R, Vandenberghe LH, Figueredo JM, Wilson JM. Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines. Hum Gene Ther. 2008 Jul; 19(7):663-9. PMID: 18549307.
    Citations: 24     Fields:    Translation:HumansAnimalsCells
  91. Royo NC, Vandenberghe LH, Ma JY, Hauspurg A, Yu L, Maronski M, Johnston J, Dichter MA, Wilson JM, Watson DJ. Specific AAV serotypes stably transduce primary hippocampal and cortical cultures with high efficiency and low toxicity. Brain Res. 2008 Jan 23; 1190:15-22. PMID: 18054899.
    Citations: 41     Fields:    Translation:AnimalsCells
  92. Vandenberghe LH, Wilson JM. AAV as an immunogen. Curr Gene Ther. 2007 Oct; 7(5):325-33. PMID: 17979679.
    Citations: 30     Fields:    Translation:HumansAnimalsCells
  93. Allocca M, Mussolino C, Garcia-Hoyos M, Sanges D, Iodice C, Petrillo M, Vandenberghe LH, Wilson JM, Marigo V, Surace EM, Auricchio A. Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol. 2007 Oct; 81(20):11372-80. PMID: 17699581.
    Citations: 110     Fields:    Translation:AnimalsCells
  94. Bell P, Vandenberghe LH, Wu D, Johnston J, Limberis M, Wilson JM. A comparative analysis of novel fluorescent proteins as reporters for gene transfer studies. J Histochem Cytochem. 2007 Sep; 55(9):931-9. PMID: 17510373.
    Citations: 11     Fields:    Translation:HumansAnimalsCells
  95. Taymans JM, Vandenberghe LH, Haute CV, Thiry I, Deroose CM, Mortelmans L, Wilson JM, Debyser Z, Baekelandt V. Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum Gene Ther. 2007 Mar; 18(3):195-206. PMID: 17343566.
    Citations: 155     Fields:    Translation:HumansAnimalsCells
  96. Vandenberghe LH, Wang L, Somanathan S, Zhi Y, Figueredo J, Calcedo R, Sanmiguel J, Desai RA, Chen CS, Johnston J, Grant RL, Gao G, Wilson JM. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med. 2006 Aug; 12(8):967-71. PMID: 16845388.
    Citations: 96     Fields:    Translation:HumansAnimalsCells
  97. Gao G, Vandenberghe LH, Wilson JM. New recombinant serotypes of AAV vectors. Curr Gene Ther. 2005 Jun; 5(3):285-97. PMID: 15975006.
    Citations: 213     Fields:    Translation:HumansAnimalsCells
  98. Roy S, Gao G, Clawson DS, Vandenberghe LH, Farina SF, Wilson JM. Complete nucleotide sequences and genome organization of four chimpanzee adenoviruses. Virology. 2004 Jul 01; 324(2):361-72. PMID: 15207622.
    Citations: 22     Fields:    Translation:AnimalsCells
  99. Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X, Wilson JM. Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol. 2004 Jun; 78(12):6381-8. PMID: 15163731.
    Citations: 456     Fields:    Translation:HumansAnimalsCells
  100. Simmons G, Rennekamp AJ, Chai N, Vandenberghe LH, Riley JL, Bates P. Folate receptor alpha and caveolae are not required for Ebola virus glycoprotein-mediated viral infection. J Virol. 2003 Dec; 77(24):13433-8. PMID: 14645601.
    Citations: 65     Fields:    Translation:HumansAnimalsCells
  101. Parry RV, Rumbley CA, Vandenberghe LH, June CH, Riley JL. CD28 and inducible costimulatory protein Src homology 2 binding domains show distinct regulation of phosphatidylinositol 3-kinase, Bcl-xL, and IL-2 expression in primary human CD4 T lymphocytes. J Immunol. 2003 Jul 01; 171(1):166-74. PMID: 12816995.
    Citations: 79     Fields:    Translation:HumansAnimalsCells
  102. Gao G, Alvira MR, Somanathan S, Lu Y, Vandenberghe LH, Rux JJ, Calcedo R, Sanmiguel J, Abbas Z, Wilson JM. Adeno-associated viruses undergo substantial evolution in primates during natural infections. Proc Natl Acad Sci U S A. 2003 May 13; 100(10):6081-6. PMID: 12716974.
    Citations: 144     Fields:    Translation:AnimalsCells
  103. Simmons G, Reeves JD, Grogan CC, Vandenberghe LH, Baribaud F, Whitbeck JC, Burke E, Buchmeier MJ, Soilleux EJ, Riley JL, Doms RW, Bates P, Pöhlmann S. DC-SIGN and DC-SIGNR bind ebola glycoproteins and enhance infection of macrophages and endothelial cells. Virology. 2003 Jan 05; 305(1):115-23. PMID: 12504546.
    Citations: 186     Fields:    Translation:HumansCells
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Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.