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Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.
Corti M, Byrne BJ, Gessler DJ, Thompson G, Norman S, Lammers J, Coleman KE, Liberati C, Elder ME, Escolar ML, Tuna IS, Mesaros C, Kleiner GI, Barbouth DS, Gray-Edwards HL, Clement N, Cleaver BD, Gao G. Corti M, et al. Among authors: gessler dj. Mol Ther Methods Clin Dev. 2023 Jun 19;30:303-314. doi: 10.1016/j.omtm.2023.06.001. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37601414 Free PMC article.
A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates.
Wang D, Li S, Gessler DJ, Xie J, Zhong L, Li J, Tran K, Van Vliet K, Ren L, Su Q, He R, Goetzmann JE, Flotte TR, Agbandje-McKenna M, Gao G. Wang D, et al. Among authors: gessler dj. Mol Ther Methods Clin Dev. 2018 Mar 16;9:234-246. doi: 10.1016/j.omtm.2018.03.004. eCollection 2018 Jun 15. Mol Ther Methods Clin Dev. 2018. PMID: 29766031 Free PMC article.
23 results