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The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup.
Brooks PJ, Ottinger EA, Portero D, Lomash RM, Alimardanov A, Terse P, Xu X, Chandler RJ, Geist Hauserman J, Esposito E, Bönnemann CG, Venditti CP, Austin CP, Pariser A, Lo DC. Brooks PJ, et al. Among authors: esposito e. Hum Gene Ther. 2020 Oct;31(19-20):1034-1042. doi: 10.1089/hum.2020.259. Hum Gene Ther. 2020. PMID: 32993373 Free PMC article. No abstract available.
Allele-specific CRISPR/Cas9 editing inactivates a single nucleotide variant associated with collagen VI muscular dystrophy.
Bolduc V, Sizov K, Brull A, Esposito E, Chen GS, Uapinyoying P, Sarathy A, Johnson K, Bönnemann CG. Bolduc V, et al. Among authors: esposito e. bioRxiv [Preprint]. 2024 Mar 22:2024.03.22.586265. doi: 10.1101/2024.03.22.586265. bioRxiv. 2024. Update in: Mol Ther Nucleic Acids. 2024 Jul 16;35(3):102269. doi: 10.1016/j.omtn.2024.102269. PMID: 38585815 Free PMC article. Updated. Preprint.
1,056 results