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Published on: July 22, 2019
Nanotechnology-based delivery of CRISPR/Cas9 for cancer treatment
Xiaoyu Xu1, Chang Liu2, Yonghui Wang2
1ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, State Key Laboratory of Medical Neurobiology, Institutes of Biomedical Sciences, Fudan University, Shanghai 200031, China; Pharmaceutical Sciences Laboratory, Faculty of Science and Engineering, Åbo Akademi University, Turku 20520, Finland.
Abstract:
CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats-associated protein 9) is a potent technology for gene-editing. Owing to its high specificity and efficiency, CRISPR/Cas9 is extensity used for human diseases treatment, especially for cancer, which involves multiple genetic alterations. Different concepts of cancer treatment by CRISPR/Cas9 are established. However, significant challenges remain for its clinical applications. The greatest challenge for CRISPR/Cas9 therapy is how to safely and efficiently deliver it to target sites in vivo. Nanotechnology has greatly contributed to cancer drug delivery. Here, we present the action mechanisms of CRISPR/Cas9, its application in cancer therapy and especially focus on the nanotechnology-based delivery of CRISPR/Cas9 for cancer gene editing and immunotherapy to pave the way for its clinical translation. We detail the difficult barriers for CRISIR/Cas9 delivery in vivo and discuss the relative solutions for encapsulation, target delivery, controlled release, cellular internalization, and endosomal escape.
Insights
CRISPR/Cas9 gene editing offers potent cancer therapy, but safe and efficient in vivo delivery remains a challenge. Nanotechnology-based strategies are crucial for advancing CRISPR/Cas9 cancer treatments and immunotherapy for clinical translation.
Area of Science:
- Biotechnology
- Genetics
- Nanomedicine
Background:
- CRISPR/Cas9 is a powerful gene-editing tool with significant potential for treating genetic diseases, particularly cancer.
- Cancer involves complex genetic alterations, making it a prime target for CRISPR/Cas9-based therapies.
- Current clinical applications of CRISPR/Cas9 face challenges, primarily concerning safe and efficient in vivo delivery.
Purpose of the Study:
- To review the mechanisms of CRISPR/Cas9 action in cancer therapy.
- To highlight nanotechnology-based delivery systems for CRISPR/Cas9 in cancer gene editing and immunotherapy.
- To address the barriers and solutions for in vivo delivery of CRISPR/Cas9 for clinical translation.
Main Methods:
- Review of CRISPR/Cas9 mechanisms and applications in cancer.
- Focus on nanotechnology-based delivery systems for CRISPR/Cas9.
- Discussion of in vivo delivery challenges including encapsulation, targeting, controlled release, cellular uptake, and endosomal escape.
Main Results:
- CRISPR/Cas9 demonstrates high specificity and efficiency for potential cancer treatments.
- Nanotechnology offers promising solutions for overcoming in vivo delivery hurdles of CRISPR/Cas9.
- Strategies for encapsulation, targeted delivery, controlled release, cellular internalization, and endosomal escape are detailed.
Conclusions:
- Nanotechnology-based delivery is essential for the clinical translation of CRISPR/Cas9 cancer gene editing and immunotherapy.
- Addressing in vivo delivery challenges is critical for the successful application of CRISPR/Cas9 therapies.
- Further development in nanodelivery systems will pave the way for advanced cancer treatments using CRISPR/Cas9.
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