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Magnetic Resonance Imaging–Guided Phase 1 Trial of Putaminal AADC Gene Therapy for Parkinson’s Disease

Chadwick C, Bankiewicz K, Van Laar A, Richardson R, Ravina B, Kells A, Boot B, Martin A, Nutt J, Thompson M, Larson P. Ann Neurol. 2019 Feb 25. doi: 10.1002/ana.25450.

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,

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.

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.


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