Как подружить гены в клетках. Коктейль молодости, светящиеся котики, напечатанные органы и другие прелести науки

33. Hoang T., Wang J., Boud P. et al. (2020, October 1). Gene regulatory networks controlling vertebrate retinal regeneration. Science, 370(6519). DOI: 10.1126/science.abb8598.

34. Dong, Q., Gentry N.W., McMahon Th. et al. (2022, April 15). Familial natural short sleep mutations reduce Alzheimer pathology in mice. Cell 25(4), 103964. DOI: https://doi.Org/10.1016/j. isci.2022.103964.

Глава 3

1. Guell, М. Gene editing in translational research (2019). Rev. 47. Bioetica & Derecho 5. Retrieved from https:// heinonline.org/HOL/LandingPage?handle=hein. journals/ rebiod47÷=4&id=&page.

2. Fikes, B.J. (2017, October 17). Yescarta, second blood cancer gene therapy, approved by FDA. The San Diego Union-Tribune. Retrieved from https://www.sandiegouniontribune.com/business/biotech/ sd-me-yescarta-cancer-20171018-story.html.

3. Mullin, E. (2017, August 30). FDA Approves Groundbreaking Gene Therapy for Cancer. MIT Technology Review. Retrieved from https://www.technologyreview.com/2017/08/30/149399/the-fda-has-approved-the-first-gene-therapy-for-cancer/.

4. High, K. A., George, L., Sullivan, S. et al. (2016, June 11). AAV-mediated gene therapy for hemophilia B-expression at therapeutic levels with low vector doses. EHA. Retrieved from https://library.ehaweb.org/eha/2016/21st/135342/spencer. sullivan.aav-mediated.gene.therapy.for.hemophilia.b-expression. at.html?f=pl6m3111619..

5. George, L.A., Sullivan, S.K. et al. (2017, December 7). Hemophilia В Gene Therapy with a High-Specifi c-Activity Factor IX Variant N Engl J Med; 377:2215-27. DOI: 10.1056/NEJMoal708538. Retrieved from https://www.nejm.org/doi/full/10.1056/ NEJMoal708538.

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.

13. Oregon Health and Science University Voyager Therapeutics (2013–2020). Retrieved from https://clinicaltrials.gov/ct2/show/study/ NCT01973543? term=AADC&rank=2.

14. Cachôn-Gonzalez M.B., Wang S.Z., Lynch A., Ziegler R., Cheng

S.H., Cox T.M. (2006 Jul 5). Effective gene therapy in an authentic model of Tay-Sachs-related diseases. Proc Natl Acad Sci USA. 103(27): 10373-10378. DOI: 10.1073/pnas.0603765103. Epub 2006 Jun 26. PMID: 16801539; PMCID: PMC1482797. Retrieved from https://pubmed.ncbi.nlm.nih.gov/16801539/.

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.