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- W4310701758 abstract "Genome editing technologies, particularly CRISPR-Cas (clustered regularly interspaced short palindromic repeats (CRISPR) associated nucleases), are redefining the boundaries of therapeutic gene therapy. CRISPR-Cas is a robust, straightforward, and programmable genome editing tool capable of mediating site-specific DNA modifications. The rapid advancements from discovery to clinical adaptation have expanded the therapeutic landscape to treat genetically defined diseases. Together with the technical developments in human DNA and RNA sequencing, CRISPR-directed gene therapy enables a new era to realize precision medicine where pathogenic mutations underlying monogenic disorders can potentially be corrected. Also, protective or therapeutic genomic alterations can be introduced as preventative or curative therapy. Despite its high therapeutic potential, CRISPR-Cas´ clinical translation is still in its infancy and is highly dependent on its efficiency, specificity in gene corrections, and cell-specific delivery. Therefore, this chapter focuses on the challenges and opportunities the CRISPR-Cas toolbox offers together with delivery vehicles to realize its use for therapeutic gene editing. Furthermore, we discuss the obstacles the CRISPR-Cas system faces for successful clinical translation and summarize its current clinical progress." @default.
- W4310701758 created "2022-12-16" @default.
- W4310701758 creator A5005834517 @default.
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- W4310701758 creator A5009748610 @default.
- W4310701758 creator A5020613114 @default.
- W4310701758 creator A5079355181 @default.
- W4310701758 date "2022-12-02" @default.
- W4310701758 modified "2023-10-10" @default.
- W4310701758 title "Enabling Precision Medicine with CRISPR-Cas Genome Editing Technology: A Translational Perspective" @default.
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