Transgenic Res. 2021 Apr;30(2):129-141. doi: 10.1007/s11248-020-00232-9. Epub 2021 Feb 20.

Mini review: genome and transcriptome editing using CRISPR-cas systems for haematological malignancy gene therapy.

Transgenic research

Esther K Elliott, Larisa M Haupt, Lyn R Griffiths

PMID: 33609253 DOI: 10.1007/s11248-020-00232-9

Abstract

The recent introduction of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR associated protein (Cas) systems, offer an array of genome and transcriptome editing tools for clinical repair strategies. These include Cas9, Cas12a, dCas9 and more recently Cas13 effectors. RNA targeting CRISPR-Cas13 complexes show unique characteristics with the capability to engineer transcriptomes and modify gene expression, providing a potential clinical cancer therapy tool across various tissue types. Cas13 effectors such as RNA base editing for A to I replacement allows for precise transcript modification. Further applications of Cas13a highlights its capability of producing rapid diagnostic results in a mobile platform. This review will focus on the adaptions of existing CRISPR-Cas systems, along with new Cas effectors for transcriptome or RNA modifications used in disease modelling and gene therapy for haematological malignancy. We also address the current diagnostic and therapeutic potential of CRISPR-Cas systems for personalised haematological malignancy.

Keywords: CRISPR1; Cas4; Gene-therapy3; Genome-editing2; Malignancy6; Transcriptome5

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