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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy.
Passini MA, Bu J, Richards AM, Treleaven CM, Sullivan JA, O'Riordan CR, Scaria A, Kells AP, Samaranch L, San Sebastian W, Federici T, Fiandaca MS, Boulis NM, Bankiewicz KS, Shihabuddin LS, Cheng SH. Passini MA, et al. Hum Gene Ther. 2014 Jul;25(7):619-30. doi: 10.1089/hum.2014.011. Epub 2014 Apr 28. Hum Gene Ther. 2014. PMID: 24617515
Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection.
Nambiar B, Cornell Sookdeo C, Berthelette P, Jackson R, Piraino S, Burnham B, Nass S, Souza D, O'Riordan CR, Vincent KA, Cheng SH, Armentano D, Kyostio-Moore S. Nambiar B, et al. Hum Gene Ther Methods. 2017 Feb;28(1):23-38. doi: 10.1089/hgtb.2016.124. Hum Gene Ther Methods. 2017. PMID: 28166648 Free PMC article.
89 results