Neda Rostami, Mohammad Mahmoudi Gomari, Edris Choupani, Shadi Abkhiz, Mahmood Fadaie, Seyed Sadegh Eslami, Zahra Mahmoudi, Yapei Zhang, Madhu Puri, Fatemeh Nafe Monfared, Elena Demireva, Vladimir N. Uversky, Bryan Ronain Smith, Sidi A. Bencherif
{"title":"Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing","authors":"Neda Rostami, Mohammad Mahmoudi Gomari, Edris Choupani, Shadi Abkhiz, Mahmood Fadaie, Seyed Sadegh Eslami, Zahra Mahmoudi, Yapei Zhang, Madhu Puri, Fatemeh Nafe Monfared, Elena Demireva, Vladimir N. Uversky, Bryan Ronain Smith, Sidi A. Bencherif","doi":"10.1002/smsc.202400192","DOIUrl":null,"url":null,"abstract":"The genetic material within cells plays a pivotal role in shaping the structure and function of living organisms. Manipulating an organism's genome to correct inherited abnormalities or introduce new traits holds great promise. Genetic engineering techniques offers promising pathways for precisely altering cellular genetics. Among these methodologies, clustered regularly interspaced short palindromic repeat (CRISPR), honored with the 2020 Nobel Prize in Chemistry, has garnered significant attention for its precision in editing genomes. However, the CRISPR system faces challenges when applied in vivo, including low delivery efficiency, off-target effects, and instability. To address these challenges, innovative technologies for targeted and precise delivery of CRISPR have emerged. Engineered carrier platforms represent a substantial advancement, improving stability, precision, and reducing the side effects associated with genome editing. These platforms facilitate efficient local and systemic genome engineering of various tissues and cells, including immune cells. This review explores recent advances, benefits, and challenges of CRISPR-based genome editing delivery. It examines various carriers including nanocarriers (polymeric, lipid-derived, metallic, and bionanoparticles), viral particles, virus-like particles, and exosomes, providing insights into their clinical utility and future prospects.","PeriodicalId":29791,"journal":{"name":"Small Science","volume":"50 1","pages":""},"PeriodicalIF":11.1000,"publicationDate":"2024-07-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Small Science","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1002/smsc.202400192","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MATERIALS SCIENCE, MULTIDISCIPLINARY","Score":null,"Total":0}
引用次数: 0
Abstract
The genetic material within cells plays a pivotal role in shaping the structure and function of living organisms. Manipulating an organism's genome to correct inherited abnormalities or introduce new traits holds great promise. Genetic engineering techniques offers promising pathways for precisely altering cellular genetics. Among these methodologies, clustered regularly interspaced short palindromic repeat (CRISPR), honored with the 2020 Nobel Prize in Chemistry, has garnered significant attention for its precision in editing genomes. However, the CRISPR system faces challenges when applied in vivo, including low delivery efficiency, off-target effects, and instability. To address these challenges, innovative technologies for targeted and precise delivery of CRISPR have emerged. Engineered carrier platforms represent a substantial advancement, improving stability, precision, and reducing the side effects associated with genome editing. These platforms facilitate efficient local and systemic genome engineering of various tissues and cells, including immune cells. This review explores recent advances, benefits, and challenges of CRISPR-based genome editing delivery. It examines various carriers including nanocarriers (polymeric, lipid-derived, metallic, and bionanoparticles), viral particles, virus-like particles, and exosomes, providing insights into their clinical utility and future prospects.
期刊介绍:
Small Science is a premium multidisciplinary open access journal dedicated to publishing impactful research from all areas of nanoscience and nanotechnology. It features interdisciplinary original research and focused review articles on relevant topics. The journal covers design, characterization, mechanism, technology, and application of micro-/nanoscale structures and systems in various fields including physics, chemistry, materials science, engineering, environmental science, life science, biology, and medicine. It welcomes innovative interdisciplinary research and its readership includes professionals from academia and industry in fields such as chemistry, physics, materials science, biology, engineering, and environmental and analytical science. Small Science is indexed and abstracted in CAS, DOAJ, Clarivate Analytics, ProQuest Central, Publicly Available Content Database, Science Database, SCOPUS, and Web of Science.