Targeting Cancer with CRISPR/Cas9-Based Therapy

TY - JOUR

T1 - Targeting Cancer with CRISPR/Cas9-Based Therapy

AU - Balon, Katarzyna

AU - Sheriff, Adam

AU - Jacków, Joanna

AU - Łaczmański, Łukasz

N1 - Funding Information: Funding: This study was supported by EB Research partnership, Inc. & EB Medical Research Foundations for project “Investigating the role of JAK/STAT3 signaling in the development of recessive dystrophic epidermolysis bullosa-squamous cell carcinoma (Collectively “EB Charities”) grant, grant numbers RE17721 and RE17763 and by CureEB grant entitled “Testing of Genomic Base Editing in EB Primary Fibroblasts”, grant number RE18567 to JJ. This research was also supported by the National Institute for Health Research (NIHR) Biomedical Research Centre based at Guy’s and St Thomas’ NHS Foundation Trust and King’s College London and/or the NIHR Clinical Research Facility. The views expressed are those of the author(s) and not necessarily those of the NHS, the NIHR or the Department of Health. Funding Information: This study was supported by EB Research partnership, Inc. & EB Medical Research Foundations for project “Investigating the role of JAK/STAT3 signaling in the development of recessive dystrophic epidermolysis bullosa-squamous cell carcinoma (Collectively “EB Charities”) grant, grant numbers RE17721 and RE17763 and by CureEB grant entitled “Testing of Genomic Base Editing in EB Primary Fibroblasts”, grant number RE18567 to JJ. This research was also supported by the National Institute for Health Research (NIHR) Biomedical Research Centre based at Guy’s and St Thomas’ NHS Foundation Trust and King’s College London and/or the NIHR Clinical Research Facility. The views expressed are those of the author(s) and not necessarily those of the NHS, the NIHR or the Department of Health. Publisher Copyright: © 2022 by the authors. Licensee MDPI, Basel, Switzerland.

PY - 2022/1/5

Y1 - 2022/1/5

N2 - Cancer is a devastating condition characterised by the uncontrolled division of cells with many forms remaining resistant to current treatment. A hallmark of cancer is the gradual accumulation of somatic mutations which drive tumorigenesis in cancerous cells, creating a mutation landscape distinctive to a cancer type, an individual patient or even a single tumour lesion. Gene editing with CRISPR/Cas9-based tools now enables the precise and permanent targeting of mutations and offers an opportunity to harness this technology to target oncogenic mutations. However, the development of safe and effective gene editing therapies for cancer relies on careful design to spare normal cells and avoid introducing other mutations. This article aims to describe recent advancements in cancer-selective treatments based on the CRISPR/Cas9 system, especially focusing on strategies for targeted delivery of the CRISPR/Cas9 machinery to affected cells, controlling Cas9 expression in tissues of interest and disrupting cancer-specific genes to result in selective death of malignant cells.

AB - Cancer is a devastating condition characterised by the uncontrolled division of cells with many forms remaining resistant to current treatment. A hallmark of cancer is the gradual accumulation of somatic mutations which drive tumorigenesis in cancerous cells, creating a mutation landscape distinctive to a cancer type, an individual patient or even a single tumour lesion. Gene editing with CRISPR/Cas9-based tools now enables the precise and permanent targeting of mutations and offers an opportunity to harness this technology to target oncogenic mutations. However, the development of safe and effective gene editing therapies for cancer relies on careful design to spare normal cells and avoid introducing other mutations. This article aims to describe recent advancements in cancer-selective treatments based on the CRISPR/Cas9 system, especially focusing on strategies for targeted delivery of the CRISPR/Cas9 machinery to affected cells, controlling Cas9 expression in tissues of interest and disrupting cancer-specific genes to result in selective death of malignant cells.

KW - Cancer

KW - CRISPR/Cas9

KW - Genetic therapy

KW - Targeting

UR - http://www.scopus.com/inward/record.url?scp=85122123748&partnerID=8YFLogxK

U2 - 10.3390/ijms23010573

DO - 10.3390/ijms23010573

M3 - Review article

AN - SCOPUS:85122123748

SN - 1661-6596

VL - 23

JO - International Journal of Molecular Sciences

JF - International Journal of Molecular Sciences

IS - 1

M1 - 573

ER -