4.7 Review

Approach for in vivo delivery of CRISPR/Cas system: a recent update and future prospect

期刊

CELLULAR AND MOLECULAR LIFE SCIENCES
卷 78, 期 6, 页码 2683-2708

出版社

SPRINGER BASEL AG
DOI: 10.1007/s00018-020-03725-2

关键词

CRISPR; Cas; Gene editing; Gene delivery; Gene therapy

资金

  1. National Health and Medical Research Council of Australia [1185600, 1123329]
  2. Ophthalmic Research Institute of Australia
  3. Shenzhen Key Laboratory of Biomimetic Materials and Cellular Immunomodulation [ZDSYS20190902093409851]
  4. National Health and Medical Research Council of Australia [1123329, 1185600] Funding Source: NHMRC

向作者/读者索取更多资源

The CRISPR/Cas system is a groundbreaking genetic technology that allows gene modification by targeting specific genomic sites. Despite its versatility, the translation of CRISPR/Cas system still faces hurdles that need to be overcome, especially in improving delivery efficiency for potential applications in various fields.
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system provides a groundbreaking genetic technology that allows scientists to modify genes by targeting specific genomic sites. Due to the relative simplicity and versatility of the CRISPR/Cas system, it has been extensively applied in human genetic research as well as in agricultural applications, such as improving crops. Since the gene editing activity of the CRISPR/Cas system largely depends on the efficiency of introducing the system into cells or tissues, an efficient and specific delivery system is critical for applying CRISPR/Cas technology. However, there are still some hurdles remaining for the translatability of CRISPR/Cas system. In this review, we summarized the approaches used for the delivery of the CRISPR/Cas system in mammals, plants, and aquacultures. We further discussed the aspects of delivery that can be improved to elevate the potential for CRISPR/Cas translatability

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