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CRISPR/Cas9 therapeutics: a cure for cancer and other genetic diseases
Faheem Ahmed Khan1, Nuruliarizki Shinta Pandupuspitasari1, Huang Chun-Jie1
1Key Laboratory of Agricultural Animal Genetics, Breeding and Reproduction, Education Ministry of China, Huazhong Agricultural University, Wuhan, People's Republic of China.
Abstract:
Cancer is caused by a series of alterations in genome and epigenome mostly resulting in activation of oncogenes or inactivation of cancer suppressor genes. Genetic engineering has become pivotal in the treatment of cancer and other genetic diseases, especially the formerly-niche use of clustered regularly interspaced short palindromic repeats (CRISPR) associated with Cas9. In defining its superior use, we have followed the recent advances that have been made in producing CRISPR/Cas9 as a therapy of choice. We also provide important genetic mutations where CRISPRs can be repurposed to create adaptive immunity to fight carcinomas and edit genetic mutations causing it. Meanwhile, challenges to CRISPR technology are also discussed with emphasis on ability of pathogens to evolve against CRISPRs. We follow the recent developments on the function of CRISPRs with different carriers which can efficiently deliver it to target cells; furthermore, analogous technologies are also discussed along CRISPRs, including zinc-finger nuclease (ZFN) and transcription activator-like effector nucleases (TALENs). Moreover, progress in clinical applications of CRISPR therapeutics is reviewed; in effect, patients can have lower morbidity and/or mortality from the therapeutic method with least possible side-effects.
Insights
CRISPR-Cas9 gene editing offers a promising new therapy for cancer by correcting genetic mutations and enhancing immunity. While challenges exist, ongoing research shows potential for reduced patient side effects and improved outcomes.
Area of Science:
- Genetics
- Molecular Biology
- Oncology
Background:
- Cancer arises from genomic and epigenomic alterations, often activating oncogenes or inactivating tumor suppressor genes.
- Genetic engineering, particularly CRISPR-Cas9 technology, is increasingly vital for treating genetic diseases and cancers.
Purpose of the Study:
- To review recent advancements in CRISPR/Cas9 as a cancer therapy.
- To explore the repurposing of CRISPR for adaptive immunity against carcinomas and editing cancer-causing mutations.
- To discuss challenges and analogous technologies in gene editing for cancer treatment.
Main Methods:
- Review of recent scientific literature on CRISPR/Cas9 technology and its applications in cancer.
- Analysis of genetic mutations amenable to CRISPR-based editing.
- Comparison of CRISPR with other gene editing tools like ZFN and TALENs.
- Examination of delivery methods and clinical progress of CRISPR therapeutics.
Main Results:
- CRISPR/Cas9 demonstrates potential in editing cancer-causing mutations and developing adaptive immunity.
- Various delivery systems are being developed for efficient CRISPR targeting.
- Analogous technologies like ZFN and TALENs are also advancing.
- Clinical trials indicate reduced morbidity and mortality with minimal side effects.
Conclusions:
- CRISPR/Cas9 is a rapidly advancing and promising therapeutic strategy for cancer treatment.
- Overcoming challenges like pathogen evolution against CRISPR is crucial for its widespread adoption.
- Continued research into delivery systems and clinical applications will further enhance CRISPR's role in oncology.
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