SelvakumarSC, PreethiKA, RossK, et al.CRISPR/Cas9 and next generation sequencing in the personalized treatment of cancer. Mol Cancer, 2022; 21(1):83; doi: 10.1186/s12943-022-01565-1
2.
BiswashMAR, SiddiqueMAB, ShabujMMH, et al.Advancing personalized cancer care: Integrating CRISPR/Cas9 with next-generation sequencing technologies. J Precis Biosci, 2024; 6(1):1–14.
3.
BadowskaKA. Allele specific genome editing for the treatment of retinitis pigmentosa. Thesis. 2022. Available from: https://iris.unitn.it/handle/11572/355261 doi: 10.15168/11572_355261
4.
ShamsnajafabadiH, KaukonenM, BellingrathJS, et al.In silico CRISPR-Cas-mediated base editing strategies for early-onset, severe cone-rod retinal degeneration in three Crumbs homolog 1 patients, including the novel variant c.2833G>A. Genes (Basel), 2024; 15(5):625; doi: 10.3390/genes15050625
5.
MusunuruK, GrandinetteSA, WangX, et al.Patient-specific in vivo gene editing to treat a rare genetic disease. N Engl J Med, 2025; 392(22):2235–2243; doi: 10.1056/NEJMoa2504747
6.
NorooziA, BarzegaryA, KarimiA, et al.Carbamoyl phosphate synthetase I deficiency. In: Genetic syndromes: A comprehensive reference guide. Cham: Springer International Publishing; 2023. pp. 1–5.
7.
GropmanAL, KomorAC. Personalized gene editing to treat an inborn error of metabolism. N Engl J Med, 2025; 392(22):2273–2276; doi: 10.1056/NEJMe2505721
8.
DilliardSA, SiegwartDJ. Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs. Nat Rev Mater, 2023; 8(4):282–300; doi: 10.1038/s41578-022-00529-7
9.
SuK, ShiL, ShengT, et al.Reformulating lipid nanoparticles for organ-targeted mRNA accumulation and translation. Nat Commun, 2024; 15(1):5659; doi: 10.1038/s41467-024-50093-7
10.
StoneD, TakeuchiR, DulinH, et al.Serum factors create species-specific barriers to hepatic gene transfer by lipid nanoparticles in liver-humanized mice. Mol Ther Methods Clin Dev, 2025; 33(2):101470; doi: 10.1016/j.omtm.2025.101470
11.
BaburMN. Harnessing CRISPR technology for precision gene editing: Innovations and ethical considerations. Multidiscip J Biochem, 2024; 1(1):1–7.
12.
PerillatL, McFadyenA, FurlongP, et al.A conceptual model and practical guidance for the development, administration, and evaluation of individualized therapies. Front Med (Lausanne), 2025; 12:1493832; doi: 10.3389/fmed.2025.1493832