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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. 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.
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.
Corti M, Liberati C, Smith BK, Lawson LA, Tuna IS, Conlon TJ, Coleman KE, Islam S, Herzog RW, Fuller DD, Collins SW, Byrne BJ. Corti M, et al. Among authors: coleman ke. Hum Gene Ther Clin Dev. 2017 Dec;28(4):208-218. doi: 10.1089/humc.2017.146. Hum Gene Ther Clin Dev. 2017. PMID: 29160099 Free PMC article. Clinical Trial.
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Song C, Conlon TJ, Deng WT, Coleman KE, Zhu P, Plummer C, Mandapati S, Van Hoosear M, Green KB, Sonnentag P, Sharma AK, Timmers A, Robinson PM, Knop DR, Hauswirth WW, Chulay JD, Shearman MS, Ye GJ. Song C, et al. Among authors: coleman ke. Hum Gene Ther Clin Dev. 2018 Dec;29(4):188-197. doi: 10.1089/humc.2018.168. Hum Gene Ther Clin Dev. 2018. PMID: 30280954 Free PMC article.
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice.
Hamm SE, Fathalikhani DD, Bukovec KE, Addington AK, Zhang H, Perry JB, McMillan RP, Lawlor MW, Prom MJ, Vanden Avond MA, Kumar SN, Coleman KE, Dupont JB, Mack DL, Brown DA, Morris CA, Gonzalez JP, Grange RW. Hamm SE, et al. Among authors: coleman ke. Mol Ther Methods Clin Dev. 2021 Mar 3;21:144-160. doi: 10.1016/j.omtm.2021.02.024. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33850950 Free PMC article.
861 results