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Книга Как подружить гены в клетках. Коктейль молодости, светящиеся котики, напечатанные органы и другие прелести науки - Ангелина Владимировна Потапова

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DOI: 10.1038/s41562-020-00956-y. Epub 2020 Sep 28. PMID: 32989287; PMCID: PMC7116623.

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Глава 3

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6. Cideciyan, A.V., Jacobson, S.G., Ho, A.C. et al. Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report. Nat Med 27, 785–789 (2021). https://doi. org/10.1038/s41591-021-01297-7.

7. Yu-Wai-Man, P., Newman, N.J., Carelli V. et al. (2020, December 9). Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy. Science Translational Medicine 12(573). DOI: 10.1126/scitranslmed.aaz7423.

8. Maloney, D.M., Chadderton, N., Millington-Ward, S. et al. (2020, November 26). Optimized OPA1 Isoforms 1 and 7 Provide Therapeutic Benefit in Models of Mitochondrial Dysfunction. Front. Neurosci., Sec. Neurodegeneration, https://doi.org/10.3389/ fnins.2020.571479.

9. Batabyal, S., Gajjeraman, S., Pradhan, S., Bhattacharya, S., Wright, W., Mohanty, S. (2021 April). Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice. Gene Ther. 28(3–4): 162–176. DOI: 10.1038/s41434-020-00200-2. Epub 2020 Oct 22. PMID: 33087861; PMCID: PMC9191254.

10. Shubina-OleinikO., French C. et al. (2021, Dec 15). Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss. Science Advances. Vol 7, Issue 51. DOI: 10.1126/sciadv.abi7629.

11. Wegmann, S., DeVos, S.L., Zeitler, B. et al. (2021). Persistent repression of tau in the brain using engineered zinc finger protein transcription factors. Sci. Adv. 7, eabel611. DOI: 10.1126/sciadv. abel611.

12. Safety Study ofAADC Gene Therapy (V Y-А ADC01) for Parkinson’s Disease (AADC). University of California, San FranciscoVeristat, Inc., Feinstein Institute for Medical Research. Retrieved from https://clinicaltrials.gov/ct2/show/NCT01973543.

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15. Flotte, T.R., Cataltepe, O., Puri, A. et al. AAV gene therapy for Tay-Sachs disease. Nat Med 28, 251–259 (2022). Retrieved from: https:// doi.org/10.1038/s41591-021-01664-4.

16. Yonekawa T., Rauckhorst A.J., El-Hattab S. et al. (2022). Largel gene transfer in older myd mice with severe muscular dystrophy restores muscle function and greatly improves survival. Sci. Adv., 8 (21), eabn0379. DOI: 10.1126/sciadv.abn0379. Retrieved from https://www.science.org/doi/10.1126/sciadv.abn0379.

Глава 4

1. Ormond, K.E., Mortlock, D.P., Scholes, D.T. et al. Human Germline Genome Editing. Am J Hum Genet. (2017, Aug 3). 101(2): 167–176. PubMed: 28777929. Free full-text available from PubMed Central: PMC5544380.

2. Gupta, R.M., Musunuru, K. Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9. J Clin Invest. (2014, Oct). 124(10): 4154-61. DOI: 10.1172/JCI72992. Epub 2014 Oct 1. PMID: 25271723; PMCID: PMC4191047.

3. Hsu, P.D., Lander, E.S., Zhang, F. (2014 Jun 5). Development and applications of CRISPR-Cas9 for genome engineering. Cell. 157(6): 1262-78.DOI:10.1016/j.celL2014.05.010.Review.PubMed:24906146. Free full-text available from PubMed Central: PMC4343198.

4. Komor, A.C., Badran, A.H., Liu, D.R. (2017 Apr 20). CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes. Cell. 169(3): 559. D01:10.1016/j.cell.2017.04.005. PubMed: 28431253.

5. Lander, E.S. (2016 Jan 14). The Heroes of CRISPR. Cell. 164(1–2): 18–28. D01:10.1016/j.cell.2015.12.041. Review. PubMed: 26771483.

6. Liang, P, Xu, Y, Zhang, X. et al. (2015). CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes Protein. Cell 6,363–372. DOI: 10.1007/S13238-015-0153-5. PMID: 25894090. PMCID: PMC4417674.

7. Kaminski, R., Chen, Y, Fischer, T. et al. (2016). Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR. Cas9 Gene Editing. Sci Rep 6,22555. DOI: 10.1038/srep22555. PMID: 27389633. PMCID: PMC4936507.

8. Marchione,M.(2018).Chineseresearcherclaimsfirstgene-editedbabies. Retrieved from https://apnews.com/4997bb7aa36c45449b488el9 ac83e86d.

9. Kinling Lo. (2019). Chinas gene-editing ‘Frankenstein jailed for three years in modified baby case. South China Morning post. Retrieved from https://www.scmp.com/news/china/science/ article/3043894/chinas-gene-editing-frankenstein-jailed-3-years-modified-baby.

10. Xu, L„Wang, J„Liu, Y. et al. (2019). CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia. N Engl J Med. 381: 1240–1247. DOI: 10.1056/NEJMoal817426. PMID: 31509667. Retrieved from https://www.nejm.org/doi/ full/10.1056/NEJMoal817426?query=TOC#article_citing_articles.

11. Brown, T.R. (2015, Jan). I Am the Berlin Patient: A Personal Reflection. AIDS Research and Human Retroviruses, 31(1), pp. 2–3. DOI: 10.1089/aid.2014.0224

12. Gupta, R.K., Abdul-Jawad, S., McCoy, L.E. et al. (2019). HIV-1 remission following CCR5A32/A32 haematopoietic stem-cell transplantation. Nature 568,244–248. DOI: 10.1038/s41586-019-1027-4.

13. Wilson, C. (2019, March 5). A third person may have become HIV-free after a bone marrow transplant. Newscientist. Retrieved from https://www.newscientist.com/article/2195780-a-third-person-may-have-become-hiv-free-after-a-bone-marrow-transplant/.

14. Gallagher J. Fourth patient seemingly cured of HIV (2022 July 27). BBC News. Retrieved from: https://www.bbc.com/news/health-62312249.

15. McKay B. Woman Appears Cured of HIV After Umbilical-Cord Blood Transplant (2022, February 15). The Wall Street Journal. Retrieved from https://www.wsj.com/articles/woman-appears-cured-of-hiv-after-umbilical-cord-blood-transplant-11644945720-?mod=hp_lead_poslO.

16. CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001® for Severe Hemoglobinopathies. CRISPR Therapeutics. Press Release. Retrieved from http://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-positive-safety-and#.

17. Haridy, R. (2019, November 19). Encouraging early results from first human CRISPR gene therapy trials. New Atlas. Retrieved from https:// newatlas.com/medical/encouraging– early-results-first-human-crispr-gene-therapy-trials/.

18. Revolutionary CRISPR-based genome editing system treatment destroys cancer cells (2020, November 18). News Release. Peer-Reviewed Publication American Friends of

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