CRISPR-based gene and cell therapy designs to improve treatment efficacy and safety for patients with genetic disorders, infectious diseases and cancer

“Clustered Regularly Interspaced Short Palindromic Repeats” (CRISPR) genome editing technology has revolutionized molecular biology and therapeutic development since its adaptation from a bacterial immune defense mechanism. This technological innovation has opened new avenues for treating previously intractable diseases, from correcting monogenic disorders like sickle cell disease to engineering immune cells for cancer immunotherapy and targeting persistent viral infections. The objective of this review was to study the role of CRISPR-based gene and cell therapy in the treatment of cancer, genetic and infectious disorders and its implementations to improve the treatment efficacy. A comprehensive search was carried out using data bases such as Cochrane, PubMed and Google Scholar which identified relevant studies published between 2014 and 2024. After screening and applying inclusion-exclusion criteria,18 articles were selected. CRISPR/Cas9, a genome editing tool from bacterial immune systems, shows promise for treating cancer, genetic and infectious diseases. It holds potential for enhancing T-cell therapies and fighting viruses like HPV, hepatitis B and C, and EBV. Challenges include cell toxicity, immune responses, off-target impacts and delivery methods. CRISPR/Cas9 is transforming the way genome is edited and is critical in the progress of cancer research, therapy, and management of infectious diseases. Novel applications include universal CAR T-cells precision gene correction and the elimination of HIV reservoirs. While it holds great promise, many issues still need to be addressed, such as off-target effects and how therapeutic genes are delivered into cells.

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Publication Details

Journal
Journal of Rehman Medical Institute
Published
2025-06-16
DOI
https://doi.org/10.52442/jrmi.v11i2.949
Primary Topic
Biomedical Ethics and Regulation
Type
article
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article

CRISPR-based gene and cell therapy designs to improve treatment efficacy and safety for patients with genetic disorders, infectious diseases and cancer

Dania Aziz
1 links
Journal of Rehman Medical Institute
Biomedical Ethics and Regulation
article

CRISPR-based gene and cell therapy designs to improve treatment efficacy and safety for patients with genetic disorders, infectious diseases and cancer

Dania Aziz
article en

Abstract

“Clustered Regularly Interspaced Short Palindromic Repeats” (CRISPR) genome editing technology has revolutionized molecular biology and therapeutic development since its adaptation from a bacterial immune defense mechanism. This technological innovation has opened new avenues for treating previously intractable diseases, from correcting monogenic disorders like sickle cell disease to engineering immune cells for cancer immunotherapy and targeting persistent viral infections. The objective of this review was to study the role of CRISPR-based gene and cell therapy in the treatment of cancer, genetic and infectious disorders and its implementations to improve the treatment efficacy. A comprehensive search was carried out using data bases such as Cochrane, PubMed and Google Scholar which identified relevant studies published between 2014 and 2024. After screening and applying inclusion-exclusion criteria,18 articles were selected. CRISPR/Cas9, a genome editing tool from bacterial immune systems, shows promise for treating cancer, genetic and infectious diseases. It holds potential for enhancing T-cell therapies and fighting viruses like HPV, hepatitis B and C, and EBV. Challenges include cell toxicity, immune responses, off-target impacts and delivery methods. CRISPR/Cas9 is transforming the way genome is edited and is critical in the progress of cancer research, therapy, and management of infectious diseases. Novel applications include universal CAR T-cells precision gene correction and the elimination of HIV reservoirs. While it holds great promise, many issues still need to be addressed, such as off-target effects and how therapeutic genes are delivered into cells.

Journal of Rehman Medical InstituteVol. 11(2)
Marymount University (US)
Openalex Percentile: Top 82%
Biomedical Ethics and Regulation
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