CRISPR/CAS9 gene editing in diabetes mellitus: a future cure
Palabras clave:
Diabetes mellitus, CRISPR-Cas9, gene therapy, gene editing, diabetes therapyResumen
Current advances in gene therapy have shown great potential in experimental studies for several disorders, including diabetes mellitus (DM). Site-specific gene editing has been enabled by clustered regularly interspaced short palindromic repeats (CRISPR) and its CRISPR-associated protein 9 (Cas9), a third-generation nuclease that causes double-strand DNA to break. This process allows the insertion, deletion or silencing of any desired gene. Studies in mice have demonstrated that diabetic status can be reversed by specific gene targeting. Other studies have targeted obesity and obesity-related genes as an alternative to improve insulin resistance and provide better metabolic homeostasis. There is ongoing investigation to identify the best possible strategy to treat DM with the CRISPR system. This review aims to analyze different experimental gene therapy studies mediated by CRISPR-Cas9 to determine their efficacy, safety profile and reliability as a possible future cure for DM. Likewise, basic concepts regarding CRISPR/cas9 will be reviewed.
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American Diabetes Association. 2. Classification and Diagnosis of Diabetes: Standards of Medical Care in Diabetes—2021. Diabetes Care. 2020 Dec 4;44(Supplement_1):S15–33.
Forouzanfar MH, Afshin A, Alexander LT, Anderson HR, Bhutta ZA, Biryukov S, et al. Global, regional, and national comparative risk assessment of 79 behavioural, environmental and occupational, and metabolic risks or clusters of risks, 1990–2015: a systematic analysis for the Global Burden of Disease Study 2015. The Lancet. 2016 Oct 8;388(10053):1659–724.
Bommer C, Sagalova V, Heesemann E, Manne-Goehler J, Atun R, Bärnighausen T, et al. Global Economic Burden of Diabetes in Adults: Projections From 2015 to 2030. Diabetes Care. 2018 May;41(5):963–70.
Bragg F, Holmes MV, Iona A, Guo Y, Du H, Chen Y, et al. Association Between Diabetes and Cause-Specific Mortality in Rural and Urban Areas of China. JAMA. 2017 Jan 17;317(3):280–9.
Yang JJ, Yu D, Wen W, Saito E, Rahman S, Shu XO, et al. Association of Diabetes With All-Cause and Cause-Specific Mortality in Asia: A Pooled Analysis of More Than 1 Million Participants. JAMA Netw Open. 2019 Apr 19;2(4):e192696.
Chen H, Chen G, Zheng X, Guo Y. Contribution of specific diseases and injuries to changes in health adjusted life expectancy in 187 countries from 1990 to 2013: retrospective observational study. BMJ. 2019 Mar 27;364:l969.
American Diabetes Association. 9. Pharmacologic Approaches to Glycemic Treatment: Standards of Medical Care in Diabetes—2021. Diabetes Care. 2020 Dec 4;44(Supplement_1):S111–24.
Landman GW, van Hateren KJ, Kleefstra N, Groenier KH, Gans RO, Bilo HJ. The relationship between glycaemic control and mortality in patients with type 2 diabetes in general practice (ZODIAC-11). Br J Gen Pract. 2010 Mar 1;60(572):172–5.
Alqarni AM, Alrahbeni T, Qarni AA, Qarni HMA. Adherence to diabetes medication among diabetic patients in the Bisha governorate of Saudi Arabia – a cross-sectional survey. Patient Prefer Adherence. 2018 Dec 24;13:63–71.
Roy P, Saha S, Chakraborty J. Looking into the possibilities of cure of the type 2 diabetes mellitus by nanoparticle-based RNAi and CRISPR-Cas9 system: A review. J Drug Deliv Sci Technol. 2021 Dec 1;66:102830.
Amitai G, Sorek R. CRISPR-Cas adaptation: insights into the mechanism of action. Nat Rev Microbiol. 2016 Feb;14(2):67–76.
Barrangou R, Fremaux C, Deveau H, Richards M, Boyaval P, Moineau S, et al. CRISPR provides acquired resistance against viruses in prokaryotes. Science. 2007 Mar 23;315(5819):1709–12.
Thurtle‐Schmidt DM, Lo T. Molecular biology at the cutting edge: A review on CRISPR/CAS9 gene editing for undergraduates. Biochem Mol Biol Educ. 2018;46(2):195–205.
Makarova KS, Wolf YI, Alkhnbashi OS, Costa F, Shah SA, Saunders SJ, et al. An updated evolutionary classification of CRISPR–Cas systems. Nat Rev Microbiol. 2015 Nov;13(11):722–36.
Mir A, Edraki A, Lee J, Sontheimer EJ. Type II-C CRISPR-Cas9 Biology, Mechanism and Application. ACS Chem Biol. 2018 Feb 16;13(2):357–65.
Liao C, Beisel CL. The tracrRNA in CRISPR Biology and Technologies. Annu Rev Genet. 2021;55(1):161–81.
Luo M, Wang J, Dong Z, Wang C, Lu G. CRISPR-Cas9 sgRNA design and outcome assessment: Bioinformatics tools and aquaculture applications. Aquac Fish. 2022 Mar 1;7(2):121–30.
Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012 Aug 17;337(6096):816–21.
Lieber MR. The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway. Annu Rev Biochem. 2010;79:181–211.
Gleditzsch D, Pausch P, Müller-Esparza H, Özcan A, Guo X, Bange G, et al. PAM identification by CRISPR-Cas effector complexes: diversified mechanisms and structures. RNA Biol. 2018 Sep 18;16(4):504–17.
Doudna JA, Charpentier E. The new frontier of genome engineering with CRISPR-Cas9. Science. 2014 Nov 28;346(6213):1258096.
Morwessel NJ. The genetic basis of diabetes mellitus. AACN Clin Issues. 1998 Nov;9(4):539–54.
Adli M. The CRISPR tool kit for genome editing and beyond. Nat Commun. 2018 May 15;9(1):1911.
Wang CH, Lundh M, Fu A, Kriszt R, Huang TL, Lynes MD, et al. CRISPR-engineered human brown-like adipocytes prevent diet-induced obesity and ameliorate metabolic syndrome in mice. Sci Transl Med. 2020 Aug 26;12(558):eaaz8664.
Brondani L de A, Assmann TS, Duarte GCK, Gross JL, Canani LH, Crispim D. The role of the uncoupling protein 1 (UCP1) on the development of obesity and type 2 diabetes mellitus. Arq Bras Endocrinol Metabol. 2012 Jun;56(4):215–25.
Chung JY, Ain QU, Song Y, Yong SB, Kim YH. Targeted delivery of CRISPR interference system against Fabp4 to white adipocytes ameliorates obesity, inflammation, hepatic steatosis, and insulin resistance. Genome Res [Internet]. 2019 Aug 29 [cited 2022 Jul 20]; Available from: https://genome.cshlp.org/content/early/2019/08/17/gr.246900.118
Won YW, Adhikary PP, Lim KS, Kim HJ, Kim JK, Kim YH. Oligopeptide complex for targeted non-viral gene delivery to adipocytes. Nat Mater. 2014 Dec;13(12):1157–64.
Nandal A, Mallon B, Telugu BP. Efficient Generation and Editing of Feeder-free IPSCs from Human Pancreatic Cells Using the CRISPR-Cas9 System. J Vis Exp JoVE. 2017 Nov 8;(129):56260.
Coombe L, Kadri A, Martinez JF, Tatachar V, Gallicano GI. Current approaches in regenerative medicine for the treatment of diabetes: introducing CRISPR/CAS9 technology and the case for non-embryonic stem cell therapy. Am J Stem Cells. 2018 Dec 1;7(5):104–13.
Rezania A, Bruin JE, Arora P, Rubin A, Batushansky I, Asadi A, et al. Reversal of diabetes with insulin-producing cells derived in vitro from human pluripotent stem cells. Nat Biotechnol. 2014 Nov;32(11):1121–33.
Bevacqua RJ, Dai X, Lam JY, Gu X, Friedlander MSH, Tellez K, et al. CRISPR-based genome editing in primary human pancreatic islet cells. Nat Commun. 2021 Apr 23;12:2397.
Ahlgren U, Jonsson J, Jonsson L, Simu K, Edlund H. β-Cell-specific inactivation of the mouse Ipf1/Pdx1 gene results in loss of the β-cell phenotype and maturity onset diabetes. Genes Dev. 1998 Jun 15;12(12):1763–8.
van Haasteren J, Li J, Scheideler OJ, Murthy N, Schaffer DV. The delivery challenge: fulfilling the promise of therapeutic genome editing. Nat Biotechnol. 2020 Jul;38(7):845–55.
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Derechos de autor 2023 Diabetes Internacional y Endocrinologia
Esta obra está bajo una licencia internacional Creative Commons Atribución-NoComercial-SinDerivadas 4.0.
