Gene Therapy and Cardiovascular Diseases

Overview

Journal Adv Exp Med Biol

Specialties Biology
General Medicine

Date 2022 Dec 1

PMID 36454471

Authors

Dongchao Lu

Sarah Cushman

Thomas Thum

Christian Bar

Affiliations

Soon will be listed here.

Abstract

Cardiovascular diseases (CVDs) are the leading causes of death globally and urgently require new novel therapeutic strategies. Gene therapy is the application of gene modulation technology to treat abnormal gene expression under disease conditions. Viral- and nonviral-based gene delivery systems are the foundation of gene modulation in target cells. Moreover, plasmid- or oligo-based gene modulation tools as well as new advancements in gene editing using CRISPR/Cas technology are currently being tested in a variety of clinical trials. Here, we summarized state-of-the-art gene therapy technologies as well as recent clinical trials and discuss the applications and lessons of gene therapy in CVDs.

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References

Eddy S . Non-coding RNA genes and the modern RNA world. Nat Rev Genet. 2001; 2(12):919-29. DOI: 10.1038/35103511. View

Huszno J, Kolosza Z, Grzybowska E . mutation in breast cancer patients: Analysis of prognostic factors and survival. Oncol Lett. 2019; 17(2):1986-1995. PMC: 6341769. DOI: 10.3892/ol.2018.9770. View

Cavanagh H, Rogers K . The role of BRCA1 and BRCA2 mutations in prostate, pancreatic and stomach cancers. Hered Cancer Clin Pract. 2015; 13(1):16. PMC: 4521499. DOI: 10.1186/s13053-015-0038-x. View

Ford D, Easton D, Bishop D, Narod S, Goldgar D . Risks of cancer in BRCA1-mutation carriers. Breast Cancer Linkage Consortium. Lancet. 1994; 343(8899):692-5. DOI: 10.1016/s0140-6736(94)91578-4. View

HEIDELBERGER C, Chaudhuri N, DANNEBERG P, Mooren D, GRIESBACH L, DUSCHINSKY R . Fluorinated pyrimidines, a new class of tumour-inhibitory compounds. Nature. 1957; 179(4561):663-6. DOI: 10.1038/179663a0. View

Farber S, DIAMOND L . Temporary remissions in acute leukemia in children produced by folic acid antagonist, 4-aminopteroyl-glutamic acid. N Engl J Med. 1948; 238(23):787-93. DOI: 10.1056/NEJM194806032382301. View

Li M, Hertz R, BERGENSTAL D . Therapy of choriocarcinoma and related trophoblastic tumors with folic acid and purine antagonists. N Engl J Med. 1958; 259(2):66-74. DOI: 10.1056/NEJM195807102590204. View

Miles W . A method of performing abdomino-perineal excision for carcinoma of the rectum and of the terminal portion of the pelvic colon (1908). CA Cancer J Clin. 1971; 21(6):361-4. DOI: 10.3322/canjclin.21.6.361. View

Sherwood J, Brock M . Lung cancer: new surgical approaches. Respirology. 2007; 12(3):326-32. DOI: 10.1111/j.1440-1843.2007.01083.x. View

10.

James N, Caty A, Borre M, Zonnenberg B, Beuzeboc P, Morris T . Safety and efficacy of the specific endothelin-A receptor antagonist ZD4054 in patients with hormone-resistant prostate cancer and bone metastases who were pain free or mildly symptomatic: a double-blind, placebo-controlled, randomised, phase 2 trial. Eur Urol. 2008; 55(5):1112-23. DOI: 10.1016/j.eururo.2008.11.002. View

11.

Couzin-Frankel J . Breakthrough of the year 2013. Cancer immunotherapy. Science. 2013; 342(6165):1432-3. DOI: 10.1126/science.342.6165.1432. View

12.

Bulaklak K, Gersbach C . The once and future gene therapy. Nat Commun. 2020; 11(1):5820. PMC: 7670458. DOI: 10.1038/s41467-020-19505-2. View

13.

Friedmann T, Roblin R . Gene therapy for human genetic disease?. Science. 1972; 175(4025):949-55. DOI: 10.1126/science.175.4025.949. View

14.

Cepko C, Roberts B, Mulligan R . Construction and applications of a highly transmissible murine retrovirus shuttle vector. Cell. 1984; 37(3):1053-62. DOI: 10.1016/0092-8674(84)90440-9. View

15.

Blaese R, Culver K, Miller A, Carter C, Fleisher T, Clerici M . T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science. 1995; 270(5235):475-80. DOI: 10.1126/science.270.5235.475. View

16.

Ozcan G, Ozpolat B, Coleman R, Sood A, Lopez-Berestein G . Preclinical and clinical development of siRNA-based therapeutics. Adv Drug Deliv Rev. 2015; 87:108-19. PMC: 4504743. DOI: 10.1016/j.addr.2015.01.007. View

17.

Wittrup A, Lieberman J . Knocking down disease: a progress report on siRNA therapeutics. Nat Rev Genet. 2015; 16(9):543-52. PMC: 4756474. DOI: 10.1038/nrg3978. View

18.

Song E, Lee S, Wang J, Ince N, Ouyang N, Min J . RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med. 2003; 9(3):347-51. DOI: 10.1038/nm828. View

19.

Tabernero J, Shapiro G, LoRusso P, Cervantes A, Schwartz G, Weiss G . First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement. Cancer Discov. 2013; 3(4):406-17. DOI: 10.1158/2159-8290.CD-12-0429. View

20.

Cideciyan A, Aleman T, Boye S, Schwartz S, Kaushal S, Roman A . Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A. 2008; 105(39):15112-7. PMC: 2567501. DOI: 10.1073/pnas.0807027105. View