Chapter 4 —— 92 —— Precise and broad scope genome editing based on high-specificity Cas9 nickases. Nucleic Acids Res. 2021, 49, 1173–1198. 36. Wang, Q.; Liu, J.; Janssen, J.M.; Gonçalves, M.A.F.V.. Precise homology-directed installation of large genomic edits in human cells with cleaving and nicking highspecificity Cas9 variants. Nucleic Acids Res. 2023, 51, 3465–3484. 37. Chen, X.; Rinsma, M.; Janssen, J.M.; Liu, J.; Maggio, I.; Gonçalves, M.A.. Probing the impact of chromatin conformation on genome editing tools. Nucleic Acids Res. 2016, 44, 6482–6492. 38. Pavani, G.; Amendola, M.. Targeted gene delivery: where to land. Front. Genome Ed. 2021, 3, 682171. 39. Knaän-Shanzer, S.; Van Der Velde, I.; Havenga, M.J.; Lemckert, A.A.; de Vries, A.A.F.; Valerio, D.. Highly efficient targeted transduction of undifferentiated human hematopoietic cells by adenoviral vectors displaying fiber knobs of subgroup B. Hum. Gene Ther. 2001, 12, 1989–2005. 40. Gonçalves, M.A.F.V.; de Vries, A.A.F.; Holkers, M.; van de Watering, M.J.M.; van der Velde, I.; van Nierop, G.P.; Valerio, D.; Knaän-Shanzer, S.. Human mesenchymal stem cells ectopically expressing full-length dystrophin can complement Duchenne muscular dystrophy myotubes by cell fusion. Hum. Mol. Genet. 2006, 15, 213–221. 41. Gonçalves, M.A.F.V.; Holkers, M.; Cudré-Mauroux, C.; van Nierop, G.P.; KnaänShanzer, S.; van der Velde, I.; Valerio, D.; de Vries, A.A.F.. Transduction of myogenic cells by retargeted dual high-capacity hybrid viral vectors: robust dystrophin synthesis in Duchenne muscular dystrophy muscle cells. Mol. Ther. 2006, 13, 976–986. 42. Ellis, B.L.; Hirsch, M.L.; Barker, J.C.; Connelly, J.P.; Steininger, R.J. 3rd; Porteus, M.H.. A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with nine natural adeno-associated virus (AAV1-9) and one engineered adenoassociated virus serotype. Virol. J. 2013, 10, 74. 43. Havenga, M.J.; Holterman, L.; Melis, I.; Smits, S.; Kaspers, J.; Heemskerk, E.; van der Vlugt, R.; Koldijk, M.; Schouten, G.J.; Hateboer, G.; et al. Serum-free transient protein production system based on adenoviral vector and PER.C6 technology: high yield and preserved bioactivity. Biotechnol. Bioeng. 2008, 100, 273–283. 44. Allen, J.M.; Halbert, C.L.; Miller, A.D.. Improved adeno-associated virus vector production with transfection of a single helper adenovirus gene, E4orf6. Mol. Ther. 2000, 1, 88–95. 45. Ferrari, F.K.; Samulski, T.; Shenk, T.; Samulski, R.J.. Second-strand synthesis is a ratelimiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 1996, 70, 3227–3234.
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