Archives

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.

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich’s ataxia.

Perdomini M, Belbellaa B, Monassier L, Reutenauer L, Messaddeq N, Cartier N, Crystal RG, Aubourg P, Puccio H. Nat Med. 2014 May;20(5):542-7.

Microglial internalization and degradation of pathological tau is enhanced by an anti-tau monoclonal antibody.

Luo W, Liu W, Hu X, Hanna M, Caravaca A, Paul SM. Sci Rep. 2015 Jun 9;5:11161.

Therapeutic antibodies for brain disorders.

Paul SM. Sci Transl Med. 2011 May 25;3(84):84ps20.

Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington’s disease.

Hadaczek P, Stanek L, Ciesielska A, Sudhakar V, Samaranch L, Pivirotto P, Bringas J, O’Riordan C, Mastis B, San Sebastian W, Forsayeth J, Cheng SH, Bankiewicz KS, Shihabuddin LS. Mol Ther Methods Clin Dev.

Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington’s disease.

Stanek LM, Sardi SP, Mastis B, Richards AR, Treleaven CM, Taksir T, Misra K, Cheng SH, Shihabuddin LS. Hum Gene Ther. 2014 May;25(5):461-74. (Sanofi-Genzyme)

Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis.

Wang H, Yang B, Qiu L, Yang C, Kramer J, Su Q, Guo Y, Brown RH Jr, Gao G, Xu Z. Hum Mol Genet. 2014 Feb 1;23(3):668-81.

Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.

Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabreja GC, Song L, Su Q, Gao GP, Elmallah MK, Brown RH Jr, Mueller C. Hum Gene Ther. 2016 Jan;27(1):19-31. (UMMS)


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