Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2016 Aug 9;7(32):52541-52552.
doi: 10.18632/oncotarget.9646.

CRISPR/Cas9 therapeutics: a cure for cancer and other genetic diseases

Faheem Ahmed Khan  1 , Nuruliarizki Shinta Pandupuspitasari  1 , Huang Chun-Jie  1 , Zhou Ao  1 , Muhammad Jamal  2 , Ali Zohaib  3 , Farhan Ahmed Khan  4 , Muthia Raihana Hakim  5 , Zhang ShuJun  1
Affiliations
Review

CRISPR/Cas9 therapeutics: a cure for cancer and other genetic diseases

Faheem Ahmed Khan et al. Oncotarget. .

Abstract

Cancer is caused by a series of alterations in genome and epigenome mostly resulting in activation of oncogenes or inactivation of cancer suppressor genes. Genetic engineering has become pivotal in the treatment of cancer and other genetic diseases, especially the formerly-niche use of clustered regularly interspaced short palindromic repeats (CRISPR) associated with Cas9. In defining its superior use, we have followed the recent advances that have been made in producing CRISPR/Cas9 as a therapy of choice. We also provide important genetic mutations where CRISPRs can be repurposed to create adaptive immunity to fight carcinomas and edit genetic mutations causing it. Meanwhile, challenges to CRISPR technology are also discussed with emphasis on ability of pathogens to evolve against CRISPRs. We follow the recent developments on the function of CRISPRs with different carriers which can efficiently deliver it to target cells; furthermore, analogous technologies are also discussed along CRISPRs, including zinc-finger nuclease (ZFN) and transcription activator-like effector nucleases (TALENs). Moreover, progress in clinical applications of CRISPR therapeutics is reviewed; in effect, patients can have lower morbidity and/or mortality from the therapeutic method with least possible side-effects.

Keywords: CRISPR/Cas9; cancer; genetic diseases; genome editing; next generation sequencing.

PubMed Disclaimer

Conflict of interest statement

The authors disclose no conflict of interests.

Figures

Figure 1
Figure 1. The CRISPR/Cas9 mechanism
Figure 2
Figure 2. Methods for delivery of Cas9-sgRNA complex to cell
A. Microinjection based delivery of Cas9-sgRNA B. viral vector (AAV) based delivery C. Lipofection D. Cell penetrating peptides (CPP) based delivery of Cas9-sgRNA complex into mammalian cells have shown successful genome editing with high efficiency.
Figure 3
Figure 3. CRISPR/Cas9 can be redesigned to cure mutations causing cancers and genetic diseases
Figure 4
Figure 4. The two DNA repair pathways
NHEJ is naturally favored while HDR pathway has therapeutic application in correcting several mutated genes.
See this image and copyright information in PMC

References

    1. Stupp R, Hegi ME, Mason WP, van den Bent MJ, Taphoorn MJ, Janzer RC, Ludwin SK, Allgeier A, Fisher B, Belanger K, Hau P. Effects of radiotherapy with concomitant and adjuvant temozolomide versus radiotherapy alone on survival in glioblastoma in a randomised phase III study: 5-year analysis of the EORTC-NCIC trial. The lancet oncology. 2009;10:459–466. - PubMed
    1. Roychowdhury S, Chinnaiyan AM. Translating cancer genomes and transcriptomes for precision oncology. CA: a cancer journal for clinicians. 2016;66:75–88. - PMC - PubMed
    1. Maresso KC, Tsai KY, Brown PH, Szabo E, Lippman S, Hawk ET. Molecular cancer prevention: Current status and future directions. CA: a cancer journal for clinicians. 2015;65:345–383. - PMC - PubMed
    1. International Human Genome Sequencing Consortium Finishing the euchromatic sequence of the human genome. Nature. 2004;431:931–945. - PubMed
    1. Lander ES, Linton LM, Birren B, Nusbaum C, Zody MC, Baldwin J, Devon K, Dewar K, Doyle M, FitzHugh W, Funke R. Initial sequencing and analysis of the human genome. Nature. 2001;409:860–921. - PubMed

MeSH terms