# CRISPR–Cas13: Pioneering RNA Editing for Nucleic Acid Therapeutics

**Authors:** Guanglin Zhu, Xinzhi Zhou, Mingzhang Wen, Jianjun Qiao, Guo Li, Yuan Yao

PMC · DOI: 10.34133/bdr.0041 · 2024-09-06

## TL;DR

CRISPR–Cas13 is a new tool for editing RNA, which can help treat diseases by targeting RNA without changing DNA.

## Contribution

This review highlights the novel use of CRISPR–Cas13 for RNA editing and its potential in developing nucleic acid therapeutics.

## Key findings

- CRISPR–Cas13 targets RNA, avoiding genomic instability seen with DNA-targeting CRISPR.
- It can suppress disease-causing genes and correct splicing errors.
- Challenges include improving specificity and delivery methods for clinical use.

## Abstract

The CRISPR–Cas13 system has emerged as a revolutionary tool for RNA editing, offering new opportunities for the development of nucleic acid therapeutics. Unlike DNA-targeting CRISPR–Cas9, Cas13 targets and cleaves RNA, enabling gene silencing and preventing genomic instability. Its applications include suppressing disease-causing genes, correcting splicing errors, and modulating immune responses. Despite these advances, challenges persist, such as the need to refine specificity, mitigate off-target impacts, and ensure effective delivery. This review provides an overview of the CRISPR–Cas13 mechanism, elucidating its role in RNA-targeted therapies and its transformative potential for disease treatment. Furthermore, it addresses the ongoing challenges that the scientific community is striving to overcome.

## Linked entities

- **Proteins:** cas9 (type II CRISPR RNA-guided endonuclease Cas9)

## Full-text entities

- **Genes:** ALKBH5 (alkB homolog 5, RNA demethylase) [NCBI Gene 54890] {aka ABH5, OFOXD, OFOXD1}, RBMS3 (RNA binding motif single stranded interacting protein 3) [NCBI Gene 27303], PCSK9 (proprotein convertase subtilisin/kexin type 9) [NCBI Gene 255738] {aka FH3, FHCL3, HCHOLA3, LDLCQ1, NARC-1, NARC1}, TMC1 (transmembrane channel like 1) [NCBI Gene 117531] {aka DFNA36, DFNB11, DFNB7}, SUGP1 (SURP and G-patch domain containing 1) [NCBI Gene 57794] {aka F23858, RBP, SF4}, VEGFA (vascular endothelial growth factor A) [NCBI Gene 7422] {aka L-VEGF, MVCD1, VEGF, VPF}, ADARB1 (adenosine deaminase RNA specific B1) [NCBI Gene 104] {aka ADAR2, DRABA2, DRADA2, NEDHYMS, RED1}, METTL3 (methyltransferase 3, N6-adenosine-methyltransferase complex catalytic subunit) [NCBI Gene 56339] {aka IME4, M6A, MT-A70, Spo8, hMETTL3}, OTOF (otoferlin) [NCBI Gene 9381] {aka AUNB1, DFNB6, DFNB9, FER1L2, NSRD9}, DMD (dystrophin) [NCBI Gene 1756] {aka BMD, CMD3B, DXS142, DXS164, DXS206, DXS230}
- **Diseases:** Duchenne muscular dystrophy (MESH:D020388), age-related macular degeneration (MESH:D008268), autosomal-dominant hearing loss (MESH:D034381), choroidal neovascularization (MESH:D020256), viral diseases (MESH:D014777), severe acute respiratory syndrome coronavirus 2 infection (MESH:D000086382), genetic diseases (MESH:D030342), metabolic diseases (MESH:D008659), cancer (MESH:D009369)
- **Chemicals:** m6A (-), rapamycin (MESH:D020123), 5-methylcytosine (MESH:D044503), cholesterol (MESH:D002784), N6-methyladenosine (MESH:C010223), 1-methyladenosine (MESH:C002230)
- **Species:** Homo sapiens (human, species) [taxon 9606]
- **Mutations:** A-to-G, A > I, A-to-I

## Figures

1 figure with captions in the complete paper: https://tomesphere.com/paper/PMC11371277/full.md

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Source: https://tomesphere.com/paper/PMC11371277