Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment

Abstract: Human genetic disorders, arising from a range of genetic irregularities, can significantly affect human physiology, often with limited available treatment options. The development of the CRISPR system, facilitating precise editing of the genome, has opened new avenues for addressing a range of mutations found in various genetic disorders. However, there is currently a lack of comprehensive reviews that specifically address the application of CRISPR in genetic diseases. To bridge this gap, this review focuses on exploring the advancements in CRISPR technology and their utility in therapeutic approaches for various genetic disorders. This review introduces human genetic disorders, explains the fundamental mechanisms of CRISPR editing, and highlights the latest advancements in CRISPR technology. Additionally, it examines three CRISPR delivery techniques, including physical delivery, viral vectors, and nanocarriers. It further reviews CRISPR's applications in therapeutic approaches for genetic disorders. Finally, it identifies the primary hurdles associated with industrial development and ethics considerations that should be addressed before the application of CRISPR in a medical context.

Location
Deutsche Nationalbibliothek Frankfurt am Main
Extent
Online-Ressource
Language
Englisch

Bibliographic citation
Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment ; day:27 ; month:05 ; year:2024 ; extent:19
Advanced functional materials ; (27.05.2024) (gesamt 19)

Creator
Yang, Xinpu
Bui, Thuy Anh
Mei, Haoqi
Aksoy, Yagiz Alp
Deng, Fei
Hutvagner, Gyorgy
Deng, Wei

DOI
10.1002/adfm.202402630
URN
urn:nbn:de:101:1-2405281405392.288833851064
Rights
Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
Last update
14.08.2025, 11:02 AM CEST

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Associated

  • Yang, Xinpu
  • Bui, Thuy Anh
  • Mei, Haoqi
  • Aksoy, Yagiz Alp
  • Deng, Fei
  • Hutvagner, Gyorgy
  • Deng, Wei

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