NemecAA, WallaceSS, SweasyJB. Variant base excision repair proteins: contributors to genomic instability. Semin Cancer Biol, 2010; 20:320–328. DOI: 10.1016/j.semcancer.2010.10.010.
2.
De BontR, van LarebekeN. Endogenous DNA damage in humans: a review of quantitative data. Mutagenesis, 2004; 19:169–185. DOI: 10.1093/mutage/geh025.
3.
Cavazzana-CalvoM, PayenE, NegreO, et al.Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature, 2010; 467:318-322. DOI: 10.1038/nature09328.
4.
TurchianoG, AndrieuxG, KlemundJ, et al.Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq. Cell Stem Cell, 2021; 28:1136–1147. DOI: 10.1016/j.stem.2021.02.002.
5.
LeibowitzML, PapathanasiouS, DoerflerPA, et al.Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing. Nat Genet, 2021; 53:895–905. DOI: 10.1038/s41588-021-00838-7.
6.
DalwadiDA, TorrensL, Abril-FornagueraJ, et al.Liver injury increases the incidence of HCC following AAV gene therapy in mice. Mol Ther, 2021; 29:680–690. DOI: 10.1016/j.ymthe.2020.10.018.
7.
ChenJ, GuoZ, TianH, et al.Production and clinical development of nanoparticles for gene delivery. Mol Ther Methods Clin Dev, 2016; 3:16023. DOI: 10.1038/mtm.2016.23.
FrangoulH, AltshulerD, CappelliniMD, et al.CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. New Engl J Med, 2021; 384:252–260. DOI: 10.1056/NEJMoa2031054.
10.
StadtmauerEA, FraiettaJA, DavisMM, et al.CRISPR-engineered T cells in patients with refractory cancer. Science, 2020; 367. DOI: 10.1126/science.aba7365.
BoutinJ, RosierJ, CappellenD, et al.CRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells. Nat Commun, 2021; 12:4922. DOI: 10.1038/s41467-021-25190-6.
13.
ChuSH, PackerM, ReesH, et al.Rationally designed base editors for precise editing of the sickle cell disease mutation. CRISPR J, 2021; 4:169–177. DOI: 10.1089/crispr.2020.0144.
