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. 2016 Aug 9;7(32):52541-52552.
doi: 10.18632/oncotarget.9646.

CRISPR/Cas9 Therapeutics: A Cure for Cancer and Other Genetic Diseases

Free PMC article

CRISPR/Cas9 Therapeutics: A Cure for Cancer and Other Genetic Diseases

Faheem Ahmed Khan et al. Oncotarget. .
Free PMC article


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.

Conflict of interest statement

The authors disclose no conflict of interests.


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.

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