Pengeditan Genom: Potensi CRISPR untuk Terapi pada Penyakit

Oleh :

dr.Jualita Heidy Saputri, Sp.PD

Penemuan protein clustered regularly interspaced short palindromic repeats (CRISPR) dan CRISPR-associated (Cas) telah memicu perkembangan penelitian terkait pengeditan genom dan potensi penggunaannya dalam dunia medis. CRISPR dan Cas diharapkan dapat digunakan dalam berbagai kondisi genetik, seperti talasemia, penyakit Fanconi, dan penyakit Huntington.[1-3]

Sekilas Tentang Pengeditan Genom

Gen merupakan bagian dari deoxyribonucleic acid (DNA) yang bertanggung jawab sebagai pembuat protein yang menurunkan informasi genetika. Fungsi gen dapat dinonaktifkan dengan rekombinasi homolog atau dengan memblokir ribonucleic acid (RNA) pembawa pesannya melalui intervensi RNA. Hal ini dapat dilakukan dengan transfeksi sel atau transgenesis organisme. Pengeditan genom dapat memanipulasi gen dengan harapan dapat memperbaiki beberapa penyakit bawaan.[3,4]

(Konten ini khusus untuk dokter. Registrasi untuk baca selengkapnya)

Referensi

1. Pacesa M, Pelea O, et al. Past, present, and future of CRISPR genome editing technologies. Cell. 2024 Feb 29;187(5):1076-1100. doi: 10.1016/j.cell.2024.01.042. .
2. Laurent M, Geoffroy M, et al. CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments. Cells. 2024 May 8;13(10):800. doi: 10.3390/cells13100800.
3. Minchin S, Lodge J. Understanding biochemistry: structure and function of nucleic acids. Essays Biochem. 2019;63(4):433-456. doi:10.1042/EBC20180038
4. Hall B, Cho A, et al. Genome Editing in Mice Using CRISPR/Cas9 Technology. Curr Protoc Cell Biol. 2018 Dec;81(1):e57. doi: 10.1002/cpcb.57.
5. Rodríguez-Rodríguez DR, Ramírez-Solís R, et al. Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review). Int J Mol Med. 2019;43(4):1559-1574. doi:10.3892/ijmm.2019.4112
6. Uddin F, Rudin CM, et al. CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future. Front Oncol. 2020 Aug 7;10:1387. doi: 10.3389/fonc.2020.01387.
7. Chen F, Ding X, et al. Targeted activation of diverse CRISPR-Cas systems for mammalian genome editing via proximal CRISPR targeting. Nat Commun. 2017 Apr 7;8:14958. doi: 10.1038/ncomms14958.
8. Canver MC, Bauer DE, et al. Functional interrogation of non-coding DNA through CRISPR genome editing. Methods. 2017 May 15;121-122:118-129. doi: 10.1016/j.ymeth.2017.03.008.
9. Cornu TI, Mussolino C, et al. Refining strategies to translate genome editing to the clinic. Nat Med. 2017 Apr 3;23(4):415-423. doi: 10.1038/nm.4313.
10. Chehelgerdi M, Chehelgerdi M, et al. Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy. Mol Cancer. 2024 Jan 9;23(1):9. doi: 10.1186/s12943-023-01925-5.
11. Zhang H, McCarty N. CRISPR-Cas9 technology and its application in haematological disorders. Br J Haematol. 2016 Oct;175(2):208-225. doi: 10.1111/bjh.14297.
12. Xu Y, Qi Y, Luo J, et al. Hepatitis B Virus X Protein Stimulates Proliferation, Wound Closure and Inhibits Apoptosis of HuH-7 Cells via CDC42. Int J Mol Sci. 2017;18(3):586. Published 2017 Mar 8. doi:10.3390/ijms18030586
13. Hammond A, Galizi R, et al. A CRISPR-Cas9 gene drive system targeting female reproduction in the malaria mosquito vector Anopheles gambiae. Nat Biotechnol. 2016 Jan;34(1):78-83. doi: 10.1038/nbt.3439.
14. Zuckermann M, Hovestadt V, et al. Somatic CRISPR/Cas9-mediated tumour suppressor disruption enables versatile brain tumour modelling. Nat Commun. 2015 Jun 11;6:7391. doi: 10.1038/ncomms8391.
15. Brunger JM, Zutshi A, et al. Genome Engineering of Stem Cells for Autonomously Regulated, Closed-Loop Delivery of Biologic Drugs. Stem Cell Reports. 2017 May 9;8(5):1202-1213. doi: 10.1016/j.stemcr.2017.03.022.
16. Goodman MA, Moradi Manesh D, et al. CRISPR/Cas9 in allergic and immunologic diseases. Expert Rev Clin Immunol. 2017 Jan;13(1):5-9. doi: 10.1080/1744666X.2017.1241711.