{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T06:47:01Z","timestamp":1790318821219,"version":"4.1.0"},"reference-count":329,"publisher":"MDPI AG","issue":"6","license":[{"start":{"date-parts":[[2024,6,7]],"date-time":"2024-06-07T00:00:00Z","timestamp":1717718400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"National Institutes of Health","award":["AI174301"],"award-info":[{"award-number":["AI174301"]}]},{"name":"National Institutes of Health","award":["DA056876"],"award-info":[{"award-number":["DA056876"]}]},{"name":"National Institutes of Health","award":["MH130193"],"award-info":[{"award-number":["MH130193"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Vaccines"],"abstract":"<jats:p>Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated enzyme-CAS holds great promise for treating many uncured human diseases and illnesses by precisely correcting harmful point mutations and disrupting disease-causing genes. The recent Food and Drug Association (FDA) approval of the first CRISPR-based gene therapy for sickle cell anemia marks the beginning of a new era in gene editing. However, delivering CRISPR specifically into diseased cells in vivo is a significant challenge and an area of intense research. The identification of new CRISPR\/Cas variants, particularly ultra-compact CAS systems with robust gene editing activities, paves the way for the low-capacity delivery vectors to be used in gene therapies. CRISPR\/Cas technology has evolved beyond editing DNA to cover a wide spectrum of functionalities, including RNA targeting, disease diagnosis, transcriptional\/epigenetic regulation, chromatin imaging, high-throughput screening, and new disease modeling. CRISPR\/Cas can be used to engineer B-cells to produce potent antibodies for more effective vaccines and enhance CAR T-cells for the more precise and efficient targeting of tumor cells. However, CRISPR\/Cas technology has challenges, including off-target effects, toxicity, immune responses, and inadequate tissue-specific delivery. Overcoming these challenges necessitates the development of a more effective and specific CRISPR\/Cas delivery system. This entails strategically utilizing specific gRNAs in conjunction with robust CRISPR\/Cas variants to mitigate off-target effects. This review seeks to delve into the intricacies of the CRISPR\/Cas mechanism, explore progress in gene therapies, evaluate gene delivery systems, highlight limitations, outline necessary precautions, and scrutinize the ethical considerations associated with its application.<\/jats:p>","DOI":"10.3390\/vaccines12060636","type":"journal-article","created":{"date-parts":[[2024,6,7]],"date-time":"2024-06-07T08:05:17Z","timestamp":1717747517000},"page":"636","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats\/Cas in Gene Therapies"],"prefix":"10.3390","volume":"12","author":[{"given":"Amrutha","family":"Banda","sequence":"first","affiliation":[{"name":"Department of Biology, The College of New Jersey, Ewing Township, NJ 08618, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivia","family":"Impomeni","sequence":"additional","affiliation":[{"name":"Department of Biology, The College of New Jersey, Ewing Township, NJ 08618, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aparana","family":"Singh","sequence":"additional","affiliation":[{"name":"Department of Chemistry, National Institute of Technology Agartala, Agartala 799046, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Abdul Rasheed","family":"Baloch","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Neurobiology, School of Medicine, Virginia Commonwealth University, Richmond, VA 23284, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8152-6116","authenticated-orcid":false,"given":"Wenhui","family":"Hu","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Neurobiology, School of Medicine, Virginia Commonwealth University, Richmond, VA 23284, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dabbu Kumar","family":"Jaijyan","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Neurobiology, School of Medicine, Virginia Commonwealth University, Richmond, VA 23284, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,6,7]]},"reference":[{"key":"ref_1","first-page":"353","article-title":"Mechanism and Applications of CRISPR\/Cas-9-Mediated Genome Editing","volume":"15","author":"Asmamaw","year":"2021","journal-title":"Biologics"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"e00580-17","DOI":"10.1128\/JB.00580-17","article-title":"History of CRISPR-Cas from Encounter with a Mysterious Repeated Sequence to Genome Editing Technology","volume":"200","author":"Ishino","year":"2018","journal-title":"J. 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