Abstract
Abstract
Background
CRISPR/Cas9 system to treat human-related diseases has achieved significant results and, even if its potential application in cancer research is improving, the application of this approach in clinical practice is still a nascent technology.
Main body
CRISPR/Cas9 technology is not yet used as a single therapy to treat tumors but it can be combined with traditional treatment strategies to provide personalized gene therapy for patients. The combination with chemotherapy, radiation and immunotherapy has been proven to be a powerful means of screening, identifying, validating and correcting tumor targets. Recently, CRISPR/Cas9 technology and CAR T-cell therapies have been integrated to open novel opportunities for the production of more efficient CAR T-cells for all patients. GMP-compatible equipment and reagents are already available for several clinical-grade systems at present, creating the basis and framework for the accelerated development of novel treatment methods.
Conclusion
Here we will provide a comprehensive collection of the actual GMP-grade CRISPR/Cas9-mediated approaches used to support cancer therapy highlighting how this technology is opening new opportunities for treating tumors.
Publisher
Springer Science and Business Media LLC
Reference88 articles.
1. European commision. EudraLex - Volume 4 - Good Manufacturing Practice (GMP) guidelines. 2003. https://health.ec.europa.eu/medicinal-products/eudralex/eudralex-volume-4_en.
2. European medicine agency. Good manufacturing practice. https://www.ema.europa.eu/en/human-regulatory-overview/research-and-development/compliance-research-and-development/good-manufacturing-practice.
3. Palmer DC, Webber BR, Patel Y, Johnson MJ, Kariya CM, Lahr WS, et al. Internal checkpoint regulates T cell neoantigen reactivity and susceptibility to PD1 blockade. Med. 2022. https://doi.org/10.1016/j.medj.2022.07.008.
4. Shy BR, Vykunta VS, Ha A, Talbot A, Roth TL, Nguyen DN, et al. High-yield genome engineering in primary cells using a hybrid ssDNA repair template and small-molecule cocktails. Nat Biotechnol. 2023. https://doi.org/10.1038/s41587-022-01418-8.
5. Basar R, Daher M, Uprety N, Gokdemir E, Alsuliman A, Ensley E, et al. Large-scale GMP-compliant CRISPR-Cas9-mediated deletion of the glucocorticoid receptor in multivirus-specific T cells. Blood Adv. 2020. https://doi.org/10.1182/bloodadvances.2020001977.