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| Autore principale: | |
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| Natura: | Recurso digital |
| Lingua: | inglese |
| Pubblicazione: |
Zenodo
2026
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| Soggetti: | |
| Accesso online: | https://doi.org/10.5281/zenodo.19658912 |
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Sommario:
- This article provides a comprehensive overview of CRISPR technology, tracing its history from the 1987 discovery of unusual bacterial DNA repeats to its establishment as a programmable gene-editing tool. The core mechanism involves the Cas9 nuclease, guided by a synthetic RNA (gRNA), which recognizes a specific DNA target adjacent to a Protospacer Adjacent Motif (PAM) to create a double-strand break. This RNA-guided system is significantly more efficient, cost-effective, and easier to program than its protein-based predecessors, ZFNs and TALENs, democratizing genome engineering. The article details the critical challenges that have shaped CRISPR's therapeutic development, including off-target effects, which have been mitigated by engineering high-fidelity Cas9 variants, and immunogenicity against the bacterial Cas9 protein. A primary hurdle is the delivery of CRISPR components, with viral vectors (AAVs) and non-viral lipid nanoparticles (LNPs) emerging as leading platforms for in vivo and ex vivo applications. The technology has evolved beyond simple gene knockouts with the advent of base and prime editors, which allow for precise single-nucleotide changes without creating double-strand breaks, enhancing safety. These advancements have culminated in major clinical successes, such as the FDA-approved ex vivo therapy Casgevy for sickle cell disease and beta-thalassemia, and pioneering in vivo LNP-delivered therapies for genetic liver disorders, demonstrating CRISPR's transformative potential in medicine. Source: https://www.geneeditsci.com/posts/from-bacterial-immunity-to-biotech-revolution-the-comprehensive-history-and-discovery-of-crispr-technology