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Peer-reviewedCC BYTheranostics

Recent advances in CRISPR technologies for biomedical applications and disease treatment.

  1. 1Department of Ophthalmology, West China Hospital, Sichuan University, Chengdu, 610041, China.
  2. 2Department of Biotherapy, Cancer Center and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu, 610041, China.
  3. 3Department of Otolaryngology-Head &Neck Surgery, West China Hospital, Sichuan University, Chengdu, 610041, China.

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR), a precise genome-editing platform, has revolutionized genetic engineering. The highly selective and programmable characteristics of CRISPR have enabled its broad application. Recent advances in CRISPR biotechnology have promoted its widespread use in various biomedical fields, such as CRISPR screening. Ongoing preclinical and clinical trials will help determine the full capabilities of CRISPR in disease therapy. The efficacy of CRISPR-based applications is fundamentally dependent on optimized delivery strategies. Multiple approaches for delivering the CRISPR components have been investigated, primarily relying on physical, viral and non-viral delivery methods. Each method has specific strengths and limitations. Notably, non-viral delivery systems hold significant promise for CRISPR applications, offering distinct advantages such as transient expression, low immunogenicity, and fewer size constraints for cargo delivery. Various biomaterials demonstrate strong potential for delivering CRISPR components to achieve gene editing, including inorganic nanoparticles, polymeric nanoparticles, lipid nanoparticles, metal-organic frameworks, DNA origami, and virus-like particles. Therefore, this review provides an overview of recent research on the molecular principles of CRISPR and its biomedical applications. Recent advances in CRISPR-based treatments and their delivery vectors are also reviewed. Finally, the challenges faced by CRISPR technology are discussed.

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BibTeX
@article{wu2026recent,
  title = {{Recent advances in CRISPR technologies for biomedical applications and disease treatment.}},
  author = {Xue Wu and Xiaorong Kou and Liping Bai and Chao Liu and Qinjie Wu and Ming Zhang and Ning Wang and Changyang Gong},
  journal = {Theranostics},
  year = {2026},
  month = sep,
  doi = {10.7150/thno.136062},
  url = {https://europepmc.org/article/MED/42909932},
  note = {CC BY},
}
RIS
TY  - JOUR
TI  - Recent advances in CRISPR technologies for biomedical applications and disease treatment.
AU  - Xue Wu
AU  - Xiaorong Kou
AU  - Liping Bai
AU  - Chao Liu
AU  - Qinjie Wu
AU  - Ming Zhang
AU  - Ning Wang
AU  - Changyang Gong
PY  - 2026
DA  - 2026/09/24
JO  - Theranostics
DO  - 10.7150/thno.136062
UR  - https://europepmc.org/article/MED/42909932
AB  - Clustered regularly interspaced short palindromic repeats (CRISPR), a precise genome-editing platform, has revolutionized genetic engineering. The highly selective and programmable characteristics of CRISPR have enabled its broad application. Recent advances in CRISPR biotechnology have promoted its widespread use in various biomedical fields, such as CRISPR screening. Ongoing preclinical and clinical trials will help determine the full capabilities of CRISPR in disease therapy. The efficacy of CRISPR-based applications is fundamentally dependent on optimized delivery strategies. Multiple approaches for delivering the CRISPR components have been investigated, primarily relying on physical, viral and non-viral delivery methods. Each method has specific strengths and limitations. Notably, non-viral delivery systems hold significant promise for CRISPR applications, offering distinct advantages such as transient expression, low immunogenicity, and fewer size constraints for cargo delivery. Various biomaterials demonstrate strong potential for delivering CRISPR components to achieve gene editing, including inorganic nanoparticles, polymeric nanoparticles, lipid nanoparticles, metal-organic frameworks, DNA origami, and virus-like particles. Therefore, this review provides an overview of recent research on the molecular principles of CRISPR and its biomedical applications. Recent advances in CRISPR-based treatments and their delivery vectors are also reviewed. Finally, the challenges faced by CRISPR technology are discussed.
SN  - 1838-7640
ER  -