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CRISPR based therapeutics: a new paradigm in cancer precision medicine
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Journal Article
CRISPR based therapeutics: a new paradigm in cancer precision medicine
2022
Overview
Background
Clustered regularly interspaced short palindromic repeat (CRISPR)-CRISPR-associated protein (Cas) systems are the latest addition to the plethora of gene-editing tools. These systems have been repurposed from their natural counterparts by means of both guide RNA and Cas nuclease engineering. These RNA-guided systems offer greater programmability and multiplexing capacity than previous generation gene editing tools based on zinc finger nucleases and transcription activator like effector nucleases. CRISPR-Cas systems show great promise for individualization of cancer precision medicine.
Main body
The biology of Cas nucleases and dead Cas based systems relevant for in vivo gene therapy applications has been discussed. The CRISPR knockout, CRISPR activation and CRISPR interference based genetic screens which offer opportunity to assess functions of thousands of genes in massively parallel assays have been also highlighted. Single and combinatorial gene knockout screens lead to identification of drug targets and synthetic lethal genetic interactions across different cancer phenotypes. There are different viral and non-viral (nanoformulation based) modalities that can carry CRISPR-Cas components to different target organs
in vivo
.
Conclusion
The latest developments in the field in terms of optimization of performance of the CRISPR-Cas elements should fuel greater application of the latter in the realm of precision medicine. Lastly, how the already available knowledge can help in furtherance of use of CRISPR based tools in personalized medicine has been discussed.
Publisher
BioMed Central,BioMed Central Ltd,Springer Nature B.V,BMC
Subject
/ Biology
/ Biomedical and Life Sciences
/ Cancer
/ Cas12a
/ Cas9
/ Clustered regularly interspaced short pallindromic repeat (CRISPR)
/ CRISPR
/ CRISPR-associated protein (Cas)
/ CRISPRa
/ CRISPRi
/ Enzymes
/ Genes
/ Genome Editing and the Future of Personalized and Targeted Cancer Medicine
/ Genomes
/ Humans
/ Materia medica and therapeutics
/ Mutation
/ Nuclease
/ Oncology
/ Proteins
/ Review
/ RNA
/ Transcription Activator-Like Effector Nucleases - genetics
