期刊
JOURNAL OF CONTROLLED RELEASE
卷 343, 期 -, 页码 703-723出版社
ELSEVIER
DOI: 10.1016/j.jconrel.2022.02.005
关键词
Therapeutics; Neurological disorders; Cancer; Stem cells; Immunotherapy; CRISPR delivery
资金
- Gujarat State Biotechnology Mission, Gujarat, India [5LY45F]
- Indian Council of Medical Research of the Government of India [5/3/8/63/ITR-F/2020]
- Taif University, Saudi Arabia [TURSP-2020/ 128]
- National Research Foundation of Korea [2017M3A9C6061361, 2021M3A9H3015389]
- National Research Foundation of Korea [2021M3A9H3015389] Funding Source: Korea Institute of Science & Technology Information (KISTI), National Science & Technology Information Service (NTIS)
A single gene mutation can cause human diseases. The CRISPR-Cas9 system is a powerful tool for correcting these mutations and has been used in various diseases. Effective delivery of CRISPR components is crucial.
A single gene mutation can cause a number of human diseases that affect the quality of life. Until the development of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas) systems, it was challenging to correct a gene mutation to avoid a disease by reverting phenotypes. The advent of CRISPR technology has changed the field of gene editing, given its simplicity and intrinsic programmability, surpassing the limitations of both zinc-finger nuclease and transcription activator-like effector nuclease and becoming the method of choice for therapeutic gene editing by overcoming the bottlenecks of conventional gene-editing techniques. Currently, there is no commercially available medicinal cure to correct a gene mutation that corrects and reverses the abnormality of a gene's function. Devising reprogramming strategies for faithful recapitulation of normal phenotypes is a crucial aspect for directing the reprogrammed cells toward clinical trials. The CRISPR-Cas9 system has been promising as a tool for correcting gene mutations in maladies including blood disorders and muscular degeneration as well as neurological, cardiovascular, renal, genetic, stem cell, and optical diseases. In this review, we highlight recent developments and utilization of the CRISPR-Cas9 system in correcting or generating gene mutations to create model organisms to develop deeper insights into diseases, rescue normal gene functionality, and curb the progression of a disease. Delivery of CRISPR-components being a pivotal aspect in proving its effectiveness, various proven delivery systems have also been briefly discussed.
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