Riset Informasi Kesehatan
Vol 14 No 1 (2025): Riset Informasi Kesehatan

The effectiveness of sickle cell anemia treatment using CRISPR

Fahira, Adinda Putri Awalia (Unknown)
Widowati, Retno (Unknown)
Fajri, Hidayatul (Unknown)



Article Info

Publish Date
30 Jan 2025

Abstract

The rapid advancement of medical biotechnology plays a crucial role in healthcare practices, particularly in genetic engineering for sickle cell anemia. Currently, there is no definitive cure for sickle cell anemia, but in recent years, gene therapy has been extensively researched as an alternative solution. Gene therapy trials on sickle cell anemia patients have been conducted using CRISPR technology, and its effectiveness is still under investigation. The principle involves manipulating the abnormal hemoglobin gene in erythrocytes, which causes sickle cell disease, by extracting stem cells from the patient’s bone marrow and replacing the faulty gene with a normal one using CRISPR (Clustered Regulatory Interspaced Short Palindromic Repeats). The critical components required are Cas9, an endonuclease restriction enzyme, and gRNA, which guides Cas9 to the target gene for manipulation. Once the target gene is successfully cut by Cas9, DNA polymerase naturally initiates a gene repair mechanism through the HDR (Homologue-directed repair) pathway, a process of repairing damaged DNA sequences in CRISPR. Based on tests conducted on 44 patients, 28 patients showed promising results, with their bone marrow producing functional, normal erythrocytes.

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Journal Info

Abbrev

rik

Publisher

Subject

Health Professions Medicine & Pharmacology Nursing Public Health

Description

Focus and Scope Riset Informasi Kesehatan publishes review article, original article, as well as short communication in the all scopes of Nursing, Public Health, and Pharmacy Science. The article published on the internal and external academic community STIKES Harapan Ibu Jambi. Riset Informasi ...