Zhen Li

Chapter 4 —— 93 —— 46. Qing, K.; Wang, X.S.; Kube, D.M.; Ponnazhagan, S.; Bajpai, A.; Srivastava, A.. Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression. Proc. Natl Acad. Sci. U.S.A. 1997, 94, 10879–10884. 47. Cervelli, T.; Palacios, J.A.; Zentilin, L.; Mano, M.; Schwartz, R.A.; Weitzman, M.D.; Giacca, M.. Processing of recombinant AAV genomes occurs in specific nuclear structures that overlap with foci of DNA-damage-response proteins. J. Cell Sci. 2008, 121, 349–357. 48. Lovric, J.; Mano, M.; Zentilin, L.; Eulalio, A.; Zacchigna, S.; Giacca, M.. Terminal differentiation of cardiac and skeletal myocytes induces permissivity to AAV transduction by relieving inhibition imposed by DNA damage response proteins. Mol. Ther. 2012, 20, 2087–2097. 49. Russell, D.W.; Alexander, I.E.; Miller, A.D.. DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc. Natl Acad. Sci. U.S.A. 1995, 92, 5719–5723. 50. Mano, M.; Ippodrino, R.; Zentilin, L.; Zacchigna, S.; Giacca, M.. Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction. Proc. Natl Acad. Sci. U.S.A. 2015, 112, 11276–11281. 51. Ihry, R.J.; Worringer, K.A.; Salick, M.R.; Frias, E.; Ho, D.; Theriault, K.; Kommineni, S.; Chen, J.; Sondey, M.; Ye, C.; et al. p53 inhibits CRISPR-Cas9 engineering in human pluripotent stem cells. Nat. Med. 2018, 24, 939–946. 52. McCarty, D.M.; Monahan, P.E.; Samulski, R.J.. Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther. 2001, 8, 1248–1254. 53. McCarty, D.M.; Fu, H.; Monahan, P.E.; Toulson, C.E.; Naik,P.; Samulski, R.J.. Adenoassociated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 2003, 10, 2112–2118. 54. Ngo, A.M.; Puschnik, A.S.. Genome-scale analysis of cellular restriction factors that inhibit transgene expression from adeno-associated virus vectors. J. Virol. 2023, 97, e0194822. 55. Duan, D.; Goemans, N.; Takeda, S.; Mercuri, E.; Aartsma-Rus, A.. Duchenne muscular dystrophy. Nat. Rev. Dis. Primers 2021, 7, 13. 56. Roberts, T.C.; Wood, M.J.A.; Davies, K.E.. Therapeutic approaches for Duchenne muscular dystrophy. Nat. Rev. Drug Discov. 2023, 22, 917–934. 57. Maggio, I.; Chen, X.; Gonçalves, M.A.. The emerging role of viral vectors as vehicles for DMD gene editing. Genome Med. 2016, 8, 59. 58. Biressi, S.; Filareto, A.; Rando,T.A.. Stem cell therapy for muscular dystrophies. J. Clin.

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