Programs

INTRAPUTAMINAL AADC GENE THERAPY (VY-AADC01) FOR ADVANCED PARKINSON’S DISEASE: INTERIM RESULTS OF A PHASE 1B TRIAL

Chadwick C, Bankiewicz K, Van Laar A, Richardson R, Ravina B, Kells A, Boot B, Martin A, Thompson M, Nutt J,Larson P. Presented at the 2017 American Academy of Neurology Annual Meeting, April 25, 2017.

Merging DBS with viral vector or stem cell implantation: “hybrid” stereotactic surgery as an evolution in the surgical treatment of Parkinson’s disease.

Rowland NC, Kalia SK, Kalia LV, Larson PS, Lim DA, Bankiewicz KS. Mol Ther Methods Clin Dev. 2016 Jan 13;3:15051.

Safety and tolerability of MRI-guided infusion of AAV2-HAADC into the mid-brain of non-human primate.

San Sebastian W, Kells AP, Bringas J, Samaranch L, Hadaczek P, Ciesielska A, Macayan M, Pivirotto PJ, Forsayeth J, Osborne S, Wright JF, Green F, Heller G, Bankiewicz KS. Mol Ther Methods Clin Dev. 2014 Oct 15;3.

Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson’s disease.

Mittermeyer G, Christine CW, Rosenbluth KH, Baker SL, Starr P, Larson P, Kaplan PL, Forsayeth J, Aminoff MJ, Bankiewicz KS. Hum Gene Ther. 2012 Apr;23(4):377-81.

Safety and tolerability of magnetic resonance imaging-guided convection-enhanced delivery of AAV2-hAADC with a novel delivery platform in nonhuman primate striatum.

San Sebastian W, Richardson RM, Kells AP, Lamarre C, Bringas J, Pivirotto P, Salegio EA, Dearmond SJ, Forsayeth J, Bankiewicz KS. Hum Gene Ther. 2012 Feb;23(2):210-7.

Real-time MR imaging with Gadoteridol predicts distribution of transgenes after convection-enhanced delivery of AAV2 vectors.

Su X, Kells AP, Salegio EA, Richardson RM, Hadaczek P, Beyer J, Bringas J, Pivirotto P, Forsayeth J, Bankiewicz KS. Mol Ther. 2010 Aug;18(8):1490-5.

Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC.

Hadaczek P, Eberling JL, Pivirotto P, Bringas J, Forsayeth J, Bankiewicz KS. Mol Ther. 2010 Aug;18(8):1458-61.

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease.

Christine CW, Starr PA, Larson PS, Eberling JL, Jagust WJ, Hawkins RA, VanBrocklin HF, Wright JF, Bankiewicz KS, Aminoff MJ. Neurology. 2009 Nov 17;73(20):1662-9.

Results from a phase I safety trial of hAADC gene therapy for Parkinson disease.

Eberling JL, Jagust WJ, Christine CW, Starr P, Larson P, Bankiewicz KS, Aminoff MJ. Neurology. 2008 May 20;70(21):1980-3.

Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain.

Cunningham J, Pivirotto P, Bringas J, Suzuki B, Vijay S, Sanftner L, Kitamura M, Chan C, Bankiewicz KS. Mol Ther. 2008 Jul;16(7):1267-75.

A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys.

Forsayeth JR, Eberling JL, Sanftner LM, Zhen Z, Pivirotto P, Bringas J, Cunningham J, Bankiewicz KS. Mol Ther. 2006 Oct;14(4):571-7.

Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

Bankiewicz KS, Forsayeth J, Eberling JL, Sanchez-Pernaute R, Pivirotto P, Bringas J, Herscovitch P, Carson RE, Eckelman W, Reutter B, Cunningham J. Mol Ther. 2006 Oct;14(4):564-70.

Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach.

Bankiewicz KS, Eberling JL, Kohutnicka M, Jagust W, Pivirotto P, Bringas J, Cunningham J, Budinger TF, Harvey-White J. Exp Neurol. 2000 Jul;164(1):2-14.

Product Engine

Empty Virions In AAV8 Vector Preparations Reduce Transduction Efficiency And May Cause Total Viral Particle Dose-Limiting Side-Effects.

Gao K, Li M, Zhong L, Su Q, Li J, Li S, He R, Zhang Y, Hendricks G, Wang J, Gao G. Mol Ther Methods Clin Dev. 2014;1(9):20139.

Production and discovery of novel recombinant adeno-associated viral vectors.

Mueller C, Ratner D, Zhong L, Esteves-Sena M, Gao G. Curr Protoc Microbiol. 2012 Aug;Chapter 14:Unit14D.1.

Reproducible high yields of recombinant adeno-associated virus produced using invertebrate cells in 0.02- to 200-liter cultures.

Cecchini S, Virag T, Kotin RM. Hum Gene Ther. 2011 Aug;22(8):1021-30.

Large-scale recombinant adeno-associated virus production.

Kotin RM. Hum Mol Genet. 2011 Apr 15;20(R1):R2-6.

A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells.

Smith RH, Levy JR, Kotin RM. Mol Ther. 2009 Nov;17(11):1888-96.

Producing recombinant adeno-associated virus in foster cells: overcoming production limitations using a baculovirus-insect cell expression strategy.

Virag T, Cecchini S, Kotin RM. Hum Gene Ther. 2009 Aug;20(8):807-17.

Strategies for manufacturing recombinant adeno-associated virus vectors for gene therapy applications exploiting baculovirus technology.

Negrete A, Kotin RM. Brief Funct Genomic Proteomic. 2008 Jul;7(4):303-11.

Large-scale production of recombinant adeno-associated viral vectors.

Negrete A1, Kotin RM. Methods Mol Biol. 2008;433:79-96.

Chromatography-based purification of adeno-associated virus.

Smith RH, Yang L, Kotin RM. Methods Mol Biol. 2008;434:37-54.

Economized large-scale production of high yield of rAAV for gene therapy applications exploiting baculovirus expression system.

Negrete A, Yang LC, Mendez AF, Levy JR, Kotin RM. J Gene Med. 2007 Nov;9(11):938-48.

Production of recombinant adeno-associated vectors using two bioreactor configurations at different scales.

Negrete A, Kotin RM. J Virol Methods. 2007 Nov;145(2):155-61.

Process optimization of large-scale production of recombinant adeno-associated vectors using dielectric spectroscopy.

Negrete A, Esteban G, Kotin RM. Appl Microbiol Biotechnol. 2007 Sep;76(4):761-72.

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems.

Ken Y Chan, Min J Jang, Bryan B Yoo, Alon Greenbaum, Namita Ravi, Wei-Li Wu, Luis Sánchez-Guardado, Carlos Lois, Sarkis K Mazmanian, Benjamin E Deverman, Viviana Gradinaru. Nat Neurosci. 2017, June. 10.1038/nn.4593.

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain.

Deverman BE, Pravdo PL, Simpson BP, Kumar SR, Chan KY, Banerjee A, Wu WL, Yang B, Huber N, Pasca SP, Gradinaru V. Nat Biotechnol. 2016 Feb;34(2):204-9.

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy.

Choudhury SR, Fitzpatrick Z, Harris AF, Maitland SA, Ferreira JS, Zhang Y, Ma S, Sharma RB, Gray-Edwards HL, Johnson JA, Johnson AK, Alonso LC, Punzo C, Wagner KR, Maguire CA, Kotin RM, Martin DR, Sena-Esteves M. Mol Ther. 2016 Aug;24(7):1247-57.

Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10.

Yang B, Li S, Wang H, Guo Y, Gessler DJ, Cao C, Su Q, Kramer J, Zhong L, Ahmed SS, Zhang H, He R, Desrosiers RC, Brown R, Xu Z, Gao G. Mol Ther. 2014 Jul;22(7):1299-309.

Recombinant AAV as a platform for translating the therapeutic potential of RNA interference.

Borel F, Kay MA, Mueller C. Mol Ther. 2014 Apr;22(4):692-701.

Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing.

Adachi K, Enoki T, Kawano Y, Veraz M, Nakai H. Nat Commun. 2014;5:3075.

Gene transfer to the CNS using recombinant adeno-associated virus.

Stoica L, Ahmed SS, Gao G, Sena-Esteves M. Curr Protoc Microbiol. 2013;Chapter 14:Unit14D.5.

Exploiting natural diversity of AAV for the design of vectors with novel properties.

Gao G, Zhong L, Danos O. Methods Mol Biol. 2011;807:93-118.

Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system.

Zhang H, Yang B, Mu X, Ahmed SS, Su Q, He R, Wang H, Mueller C, Sena-Esteves M, Brown R, Xu Z, Gao G. Mol Ther. 2011 Aug;19(8):1440-8.

A New Recombinant Adeno-Associated Virus (AAV)-Based Random Peptide Display Library System: Infection-Defective Aav1.9-3 as a Novel Detargeted Platform for Vector Evolution

Adachi K, Nakai H. Gene Ther Regul. 2010 Oct;5(1):31-55.

A Single Injection of Recombinant Adeno-Associated Virus into the Lumbar Cistern Delivers Transgene Expression Throughout the Whole Spinal Cord.

Guo Y, Wang D, Qiao T, Yang C, Su Q, Gao G, Xu Z. Mol Neurobiol. 2016 Jul;53(5):3235-48.

Axonal transport of AAV9 in nonhuman primate brain.

Green F, Samaranch L, Zhang HS, Manning-Bog A, Meyer K, Forsayeth J, Bankiewicz KS. Gene Ther. 2016 Jun;23(6):520-6.

AAV viral vector delivery to the brain by shape-conforming MR-guided infusions.

Bankiewicz KS, Sudhakar V, Samaranch L, San Sebastian W, Bringas J, Forsayeth J. J Control Release. 2016 Feb 27.

MRI-Guided Delivery of Viral Vectors.

Salegio EA, Bringas J, Bankiewicz KS. Methods Mol Biol. 2016;1382:217-30.

State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.

Wang D, Gao G. Discov Med. 2014 Sep;18(98):151-61.

State-of-the-art human gene therapy: part I. Gene delivery technologies.

Wang D, Gao G. Discov Med. 2014 Jul-Aug;18(97):67-77.

The potential of adeno-associated viral vectors for gene delivery to muscle tissue.

Wang D, Zhong L, Nahid MA, Gao G. Expert Opin Drug Deliv. 2014 Mar;11(3):345-64.

AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction.

Samaranch L, San Sebastian W, Kells AP, Salegio EA, Heller G, Bringas JR, Pivirotto P, DeArmond S, Forsayeth J, Bankiewicz KS. Mol Ther. 2014 Feb;22(2):329-37.

Adeno-associated virus type 6 is retrogradely transported in the non-human primate brain.

San Sebastian W, Samaranch L, Heller G, Kells AP, Bringas J, Pivirotto P, Forsayeth J, Bankiewicz KS. Gene Ther. 2013 Dec;20(12):1178-83.

Gene therapy for misfolding protein diseases of the central nervous system.

San Sebastian W, Samaranch L, Kells AP, Forsayeth J, Bankiewicz KS. Neurotherapeutics. 2013 Jul;10(3):498-510.

Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates.

Samaranch L, Salegio EA, San Sebastian W, Kells AP, Bringas JR, Forsayeth J, Bankiewicz KS. Hum Gene Ther. 2013 May;24(5):526-32.

Axonal transport of adeno-associated viral vectors is serotype-dependent.

Salegio EA1, Samaranch L, Kells AP, Mittermeyer G, San Sebastian W, Zhou S, Beyer J, Forsayeth J, Bankiewicz KS. Gene Ther. 2013 Mar;20(3):348-52.

Interventional MRI-guided putaminal delivery of AAV2-GDNF for a planned clinical trial in Parkinson’s disease.

Richardson RM, Kells AP, Rosenbluth KH, Salegio EA, Fiandaca MS, Larson PS, Starr PA, Martin AJ, Lonser RR, Federoff HJ, Forsayeth JR, Bankiewicz KS. Mol Ther. 2011 Jun;19(6):1048-57.

Novel platform for MRI-guided convection-enhanced delivery of therapeutics: preclinical validation in nonhuman primate brain.

Richardson RM, Kells AP, Martin AJ, Larson PS, Starr PA, Piferi PG, Bates G, Tansey L, Rosenbluth KH, Bringas JR, Berger MS, Bankiewicz KS. Stereotact Funct Neurosurg. 2011;89(3):141-51.

Design of an in-dwelling cannula for convection-enhanced delivery.

Rosenbluth KH, Luz M, Mohr E, Mittermeyer S, Bringas J, Bankiewicz KS. J Neurosci Methods. 2011 Mar 15;196(1):118-23.

MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression.

Xie J, Xie Q, Zhang H, Ameres SL, Hung JH, Su Q, He R, Mu X, Seher Ahmed S, Park S, Kato H, Li C, Mueller C, Mello CC, Weng Z, Flotte TR, Zamore PD, Gao G. Mol Ther. 2011 Mar;19(3):526-35.

Scientific Conferences

2017 European Society of Gene and Cell Therapy