Vandenberghe Lab Studies

1.    Dudek, AM, Pillay, S, Puschnik, AS, Nagamine, CM, Cheng, F, Qiu, J, Carette, JE, and Vandenberghe, LH (2018). An alternate route for adeno-associated virus entry independent of AAVR. J Virol.

2.    Carvalho, LS, Xiao, R, Wassmer, S, Langsdorf, A, Zinn, E, Pacouret, S, Shah, S, Comander, JI, Kim, L, Lim, L, and Vandenberghe, LH (2018). Synthetic adeno-associated viral vector efficiently targets mouse and non-human primate retina in vivo. Hum Gene Ther.

3.    Tao, Y, Huang, M, Shu, Y, Ruprecht, A, Wang, H, Tang, Y, Vandenberghe, LH, Wang, Q, Gao, G, Kong, WJ, and Chen, ZY (2018). Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction. Hum Gene Ther.

4.    Carvalho, LS, Turunen, HT, Wassmer, SJ, Luna-Velez, MV, Xiao, R, Bennett, J, and Vandenberghe, LH (2017). Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting. Front Neurosci 11: 503.

5.    Wang, L, Xiao, R, Andres-Mateos, E, and Vandenberghe, LH (2017). Single stranded adeno-associated virus achieves efficient gene transfer to anterior segment in the mouse eye. PLoS One 12: e0182473.

6.    Suzuki, J, Hashimoto, K, Xiao, R, Vandenberghe, LH, and Liberman, MC (2017). Corrigendum: Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction. Sci Rep 7: 46827.

7.    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, and Vandenberghe, LH (2017). AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations. Mol Ther 25: 1375-1386.

8.    Suzuki, J, Hashimoto, K, Xiao, R, Vandenberghe, LH, and Liberman, MC (2017). Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction. Sci Rep 7: 45524.

9.    Wassmer, SJ, Carvalho, LS, Gyorgy, B, Vandenberghe, LH, and Maguire, CA (2017). Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection. Sci Rep 7: 45329.

10.    Pan, B, Askew, C, Galvin, A, Heman-Ackah, S, Asai, Y, Indzhykulian, AA, Jodelka, FM, Hastings, ML, Lentz, JJ, Vandenberghe, LH, Holt, JR, and Geleoc, GS (2017). Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c. Nat Biotechnol 35: 264-272.

11.    Landegger, LD, Pan, B, Askew, C, Wassmer, SJ, Gluck, SD, Galvin, A, Taylor, R, Forge, A, Stankovic, KM, Holt, JR, and Vandenberghe, LH (2017). A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear. Nat Biotechnol 35: 280-284.

12.    Garanto, A, Chung, DC, Duijkers, L, Corral-Serrano, JC, Messchaert, M, Xiao, R, Bennett, J, Vandenberghe, LH, and Collin, RW (2016). In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery. Hum Mol Genet 25: 2552-2563.

13.    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, and Wagers, AJ (2016). In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351: 407-411.

14.    Castle, MJ, Turunen, HT, Vandenberghe, LH, and Wolfe, JH (2016). Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids. Methods Mol Biol 1382: 133-149.

15.    Carvalho, LS, and Vandenberghe, LH (2016). Understanding Cone Photoreceptor Cell Death in Achromatopsia. Adv Exp Med Biol 854: 231-236.

16.    Zinn, E, Pacouret, S, Khaychuk, V, Turunen, HT, Carvalho, LS, Andres-Mateos, E, Shah, S, Shelke, R, Maurer, AC, Plovie, E, Xiao, R, and Vandenberghe, LH (2015). In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector. Cell Rep 12: 1056-1068.

17.    Vandenberghe, LH (2015). What Is Next for Retinal Gene Therapy? Cold Spring Harb Perspect Med 5.

18.    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, and Gubitz, AK (2015). Perspectives on best practices for gene therapy programs. Hum Gene Ther 26: 127-133.

19.    Zinn, E, and Vandenberghe, LH (2014). Adeno-associated virus: fit to serve. Curr Opin Virol 8: 90-97.

20.    Carvalho, LS, and Vandenberghe, LH (2015). Promising and delivering gene therapies for vision loss. Vision Res 111: 124-133.


Prior work

21.    Cronin, T, Vandenberghe, LH, Hantz, P, Juttner, J, Reimann, A, Kacso, AE, Huckfeldt, RM, Busskamp, V, Kohler, H, Lagali, PS, Roska, B, and Bennett, J (2014). Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter. EMBO Mol Med 6: 1175-1190.
22.    Bell, P, Vandenberghe, LH, and Wilson, JM (2014). Formation of newly synthesized adeno-associated virus capsids in the cell nucleus. Hum Gene Ther Methods 25: 179-180.
23.    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, and Corrigan-Curay, J (2014). Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. Hum Gene Ther 25: 488-497.
24.    Mikals, K, Nam, HJ, Van Vliet, K, Vandenberghe, LH, Mays, LE, McKenna, R, Wilson, JM, and Agbandje-McKenna, M (2014). The structure of AAVrh32.33, a novel gene delivery vector. J Struct Biol 186: 308-317.
25.    Lagor, WR, Johnston, JC, Lock, M, Vandenberghe, LH, and Rader, DJ (2013). Adeno-associated viruses as liver-directed gene delivery vehicles: focus on lipoprotein metabolism. Methods Mol Biol 1027: 273-307.
26.    Simonato, M, Bennett, J, Boulis, NM, Castro, MG, Fink, DJ, Goins, WF, Gray, SJ, Lowenstein, PR, Vandenberghe, LH, Wilson, TJ, Wolfe, JH, and Glorioso, JC (2013). Progress in gene therapy for neurological disorders. Nat Rev Neurol 9: 277-291.
27.    Cepko, CL, and Vandenberghe, LH (2013). Retinal gene therapy coming of age. Hum Gene Ther 24: 242-244.
28.    Vandenberghe, LH, Bell, P, Maguire, AM, Xiao, R, Hopkins, TB, Grant, R, Bennett, J, and Wilson, JM (2013). AAV9 targets cone photoreceptors in the nonhuman primate retina. PLoS One 8: e53463.
29.    Holt, JR, and Vandenberghe, LH (2012). Gene therapy for deaf mice goes viral. Mol Ther 20: 1836-1837.
30.    Mason, JB, Vandenberghe, LH, Xiao, R, Wilson, JM, and Richardson, DW (2012). 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 73: 1178-1185.
31.    Vandenberghe, LH, and Auricchio, A (2012). Novel adeno-associated viral vectors for retinal gene therapy. Gene Ther 19: 162-168.
32.    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, and Jacobson, SG (2011). Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model. Invest Ophthalmol Vis Sci 52: 6898-6910.
33.    Vandenberghe, LH, Bell, P, Maguire, AM, Cearley, CN, Xiao, R, Calcedo, R, Wang, L, Castle, MJ, Maguire, AC, Grant, R, Wolfe, JH, Wilson, JM, and Bennett, J (2011). Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey. Sci Transl Med 3: 88ra54.
34.    Kassim, SH, Vandenberghe, LH, Hovhannisyan, R, Wilson, JM, and Rader, DJ (2011). Identification and functional characterization in vivo of a novel splice variant of LDLR in rhesus macaques. Physiol Genomics 43: 911-916.
35.    Bell, CL, Vandenberghe, LH, Bell, P, Limberis, MP, Gao, GP, Van Vliet, K, Agbandje-McKenna, M, and Wilson, JM (2011). The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice. J Clin Invest 121: 2427-2435.
36.    Van der Perren, A, Toelen, J, Carlon, M, Van den Haute, C, Coun, F, Heeman, B, Reumers, V, Vandenberghe, LH, Wilson, JM, Debyser, Z, and Baekelandt, V (2011). 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 18: 517-527.
37.    Bell, P, Gao, G, Haskins, ME, Wang, L, Sleeper, M, Wang, H, Calcedo, R, Vandenberghe, LH, Chen, SJ, Weisse, C, Withnall, E, and Wilson, JM (2011). Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs. Hum Gene Ther 22: 985-997.
38.    Kassim, SH, Li, H, Vandenberghe, LH, Hinderer, C, Bell, P, Marchadier, D, Wilson, A, Cromley, D, Redon, V, Yu, H, Wilson, JM, and Rader, DJ (2010). Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis. PLoS One 5: e13424.
39.    Carlon, M, Toelen, J, Van der Perren, A, Vandenberghe, LH, Reumers, V, Sbragia, L, Gijsbers, R, Baekelandt, V, Himmelreich, U, Wilson, JM, Deprest, J, and Debyser, Z (2010). Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2. Mol Ther 18: 2130-2138.
40.    Vandenberghe, LH, Xiao, R, Lock, M, Lin, J, Korn, M, and Wilson, JM (2010). Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. Hum Gene Ther 21: 1251-1257.
41.    Lock, M, Alvira, M, Vandenberghe, LH, Samanta, A, Toelen, J, Debyser, Z, and Wilson, JM (2010). Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther 21: 1259-1271.
42.    Wang, L, Calcedo, R, Wang, H, Bell, P, Grant, R, Vandenberghe, LH, Sanmiguel, J, Morizono, H, Batshaw, ML, and Wilson, JM (2010). The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther 18: 126-134.
43.    Wang, L, Wang, H, Bell, P, McCarter, RJ, He, J, Calcedo, R, Vandenberghe, LH, Morizono, H, Batshaw, ML, and Wilson, JM (2010). Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol Ther 18: 118-125.
44.    Lin, J, Calcedo, R, Vandenberghe, LH, Bell, P, Somanathan, S, and Wilson, JM (2009). A new genetic vaccine platform based on an adeno-associated virus isolated from a rhesus macaque. J Virol 83: 12738-12750.
45.    Vandenberghe, LH, Breous, E, Nam, HJ, Gao, G, Xiao, R, Sandhu, A, Johnston, J, Debyser, Z, Agbandje-McKenna, M, and Wilson, JM (2009). Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints. Gene Ther 16: 1416-1428.
46.    Roy, S, Vandenberghe, LH, Kryazhimskiy, S, Grant, R, Calcedo, R, Yuan, X, Keough, M, Sandhu, A, Wang, Q, Medina-Jaszek, CA, Plotkin, JB, and Wilson, JM (2009). Isolation and characterization of adenoviruses persistently shed from the gastrointestinal tract of non-human primates. PLoS Pathog 5: e1000503.
47.    Breous, E, Somanathan, S, Vandenberghe, LH, and Wilson, JM (2009). Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver. Hepatology 50: 612-621.
48.    Gao, G, Wang, Q, Calcedo, R, Mays, L, Bell, P, Wang, L, Vandenberghe, LH, Grant, R, Sanmiguel, J, Furth, EE, and Wilson, JM (2009). Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Hum Gene Ther 20: 930-942.
49.    Mays, LE, Vandenberghe, LH, Xiao, R, Bell, P, Nam, HJ, Agbandje-McKenna, M, and Wilson, JM (2009). Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins. J Immunol 182: 6051-6060.
50.    Song, Y, Lou, HH, Boyer, JL, Limberis, MP, Vandenberghe, LH, Hackett, NR, Leopold, PL, Wilson, JM, and Crystal, RG (2009). Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. Hum Gene Ther 20: 267-281.
51.    Calcedo, R, Vandenberghe, LH, Gao, G, Lin, J, and Wilson, JM (2009). Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis 199: 381-390.
52.    Calcedo, R, Vandenberghe, LH, Roy, S, Somanathan, S, Wang, L, and Wilson, JM (2009). Host immune responses to chronic adenovirus infections in human and nonhuman primates. J Virol 83: 2623-2631.
53.    Limberis, MP, Vandenberghe, LH, Zhang, L, Pickles, RJ, and Wilson, JM (2009). Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol Ther 17: 294-301.
54.    Vandenberghe, LH, Wilson, JM, and Gao, G (2009). Tailoring the AAV vector capsid for gene therapy. Gene Ther 16: 311-319.
55.    Cearley, CN, Vandenberghe, LH, Parente, MK, Carnish, ER, Wilson, JM, and Wolfe, JH (2008). Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol Ther 16: 1710-1718.
56.    Lin, J, Calcedo, R, Vandenberghe, LH, Figueredo, JM, and Wilson, JM (2008). Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines. Hum Gene Ther 19: 663-669.
57.    Royo, NC, Vandenberghe, LH, Ma, JY, Hauspurg, A, Yu, L, Maronski, M, Johnston, J, Dichter, MA, Wilson, JM, and Watson, DJ (2008). Specific AAV serotypes stably transduce primary hippocampal and cortical cultures with high efficiency and low toxicity. Brain Res 1190: 15-22.
58.    Vandenberghe, LH, and Wilson, JM (2007). AAV as an immunogen. Curr Gene Ther 7: 325-333.
59.    Allocca, M, Mussolino, C, Garcia-Hoyos, M, Sanges, D, Iodice, C, Petrillo, M, Vandenberghe, LH, Wilson, JM, Marigo, V, Surace, EM, and Auricchio, A (2007). Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol 81: 11372-11380.
60.    Bell, P, Vandenberghe, LH, Wu, D, Johnston, J, Limberis, M, and Wilson, JM (2007). A comparative analysis of novel fluorescent proteins as reporters for gene transfer studies. J Histochem Cytochem 55: 931-939.
61.    Taymans, JM, Vandenberghe, LH, Haute, CV, Thiry, I, Deroose, CM, Mortelmans, L, Wilson, JM, Debyser, Z, and Baekelandt, V (2007). Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum Gene Ther 18: 195-206.
62.    Vandenberghe, LH, Wang, L, Somanathan, S, Zhi, Y, Figueredo, J, Calcedo, R, Sanmiguel, J, Desai, RA, Chen, CS, Johnston, J, Grant, RL, Gao, G, and Wilson, JM (2006). Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med 12: 967-971.
63.    Gao, G, Vandenberghe, LH, and Wilson, JM (2005). New recombinant serotypes of AAV vectors. Curr Gene Ther 5: 285-297.
64.    Roy, S, Gao, G, Clawson, DS, Vandenberghe, LH, Farina, SF, and Wilson, JM (2004). Complete nucleotide sequences and genome organization of four chimpanzee adenoviruses. Virology 324: 361-372.
65.    Gao, G, Vandenberghe, LH, Alvira, MR, Lu, Y, Calcedo, R, Zhou, X, and Wilson, JM (2004). Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol 78: 6381-6388.
66.    Simmons, G, Rennekamp, AJ, Chai, N, Vandenberghe, LH, Riley, JL, and Bates, P (2003). Folate receptor alpha and caveolae are not required for Ebola virus glycoprotein-mediated viral infection. J Virol 77: 13433-13438.
67.    Parry, RV, Rumbley, CA, Vandenberghe, LH, June, CH, and Riley, JL (2003). 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 171: 166-174.
68.    Gao, G, Alvira, MR, Somanathan, S, Lu, Y, Vandenberghe, LH, Rux, JJ, Calcedo, R, Sanmiguel, J, Abbas, Z, and Wilson, JM (2003). Adeno-associated viruses undergo substantial evolution in primates during natural infections. Proceedings of the National Academy of Sciences of the United States of America 100: 6081-6086.
69.    Simmons, G, Reeves, JD, Grogan, CC, Vandenberghe, LH, Baribaud, F, Whitbeck, JC, Burke, E, Buchmeier, MJ, Soilleux, EJ, Riley, JL, Doms, RW, Bates, P, and Pohlmann, S (2003). DC-SIGN and DC-SIGNR bind ebola glycoproteins and enhance infection of macrophages and endothelial cells. Virology 305: 115-123.