Chapter 6 —— 159 —— 14. Wada M, Uchida N, Posadas-Herrera G, Hayashita-Kinoh H, Tsunekawa Y, Hirai Y, Okada T. Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor. Gene Ther. 2023 30:641648. 15. McColl-Carboni A, Dollive S, Laughlin S, Lushi R, MacArthur M, Zhou S, Gagnon J, Smith CA, Burnham B, Horton R, Lata D, Uga B, Natu K, Michel E, Slater C, DaSilva E, Bruccoleri R, Kelly T, McGivney JB 4th. Analytical characterization of full, intermediate, and empty AAV capsids. Gene Ther. 2024 31:285-294. 16. Miller DG, Petek LM, Russell DW. Adeno-associated virus vectors integrate at chromosome breakage sites. Nat Genet. 2004 36:767-773. 17. Ferrari S, Jacob A, Cesana D, Laugel M, Beretta S, Varesi A, Unali G, Conti A, Canarutto D, Albano L, Calabria A, Vavassori V, Cipriani C, Castiello MC, Esposito S, Brombin C, Cugnata F, Adjali O, Ayuso E, Merelli I, Villa A, Di Micco R, KajasteRudnitski A, Montini E, Penaud-Budloo M, Naldini L. Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells. Cell Stem Cell 2022 29:1428-1444. 18. Suchy FP, Karigane D, Nakauchi Y, Higuchi M, Zhang J, Pekrun K, Hsu I, Fan AC, Nishimura T, Charlesworth CT, Bhadury J, Nishimura T, Wilkinson AC, Kay MA, Majeti R, Nakauchi H. Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectors. Nat Biotechnol. 2025 43:204-213. 19. Schiroli G, Conti A, Ferrari S, Della Volpe L, Jacob A, Albano L, Beretta S, Calabria A, Vavassori V, Gasparini P, Salataj E, Ndiaye-Lobry D, Brombin C, Chaumeil J, Montini E, Merelli I, Genovese P, Naldini L, Di Micco R. Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response. Cell Stem Cell. 2019 24:551-565. 20. Allen D, Weiss LE, Saguy A, Rosenberg M, Iancu O, Matalon O, Lee C, Beider K, Nagler A, Shechtman Y, Hendel A. High-Throughput Imaging of CRISPR- and Recombinant Adeno-Associated Virus-Induced DNA Damage Response in Human Hematopoietic Stem and Progenitor Cells. CRISPR J. 2022 5:80-94. 21. Agudelo D, Duringer A, Bozoyan L, Huard CC, Carter S, Loehr J, Synodinou D, Drouin M, Salsman J, Dellaire G, Laganière J, Doyon Y. Marker-free coselection for CRISPRdriven genome editing in human cells. Nat Methods. 2017 :615-620. 22. Frock RL, Hu J, Meyers RM, Ho YJ, Kii E, Alt FW. Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases. Nat Biotechnol. 2015 33:179186.
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