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CRISPR-based therapeutics: current challenges and future applications.

ABSTRACT: The discovery, only a decade ago, of the genome editing power of clustered regularly interspaced short palindromic repeats (CRISPR)-associated nucleases is already reinventing the therapeutic process, from how new drugs are discovered to novel ways to treat diseases. CRISPR-based screens can aid therapeutic development by quickly identifying a drug's mechanism of action and escape mutants. Additionally, CRISPR-Cas has advanced emerging ex vivo therapeutics, such as cell replacement therapies. However, Cas9 is limited as an in vivo therapeutic due to ineffective delivery, unwanted immune responses, off-target effects, unpredictable repair outcomes, and cellular stress. To address these limitations, controls that inhibit or degrade Cas9, biomolecule-Cas9 conjugates, and base editors have been developed. Herein, we discuss CRISPR-Cas systems that advance both conventional and emerging therapeutics.

SUBMITTER: Modell AE

PROVIDER: S-EPMC9726229 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature

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CRISPR-based therapeutics: current challenges and future applications.

Modell Ashley E AE Lim Donghyun D Nguyen Tuan M TM Sreekanth Vedagopuram V Choudhary Amit A

Trends in pharmacological sciences 20211221 2

The discovery, only a decade ago, of the genome editing power of clustered regularly interspaced short palindromic repeats (CRISPR)-associated nucleases is already reinventing the therapeutic process, from how new drugs are discovered to novel ways to treat diseases. CRISPR-based screens can aid therapeutic development by quickly identifying a drug's mechanism of action and escape mutants. Additionally, CRISPR-Cas has advanced emerging ex vivo therapeutics, such as cell replacement therapies. Ho ...[more]

PMID: 34952739

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