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 and Zhang ShuJun 1 1 Key Laboratory of Agricultural Animal Genetics, Breeding and Reproduction, Education Ministry of China, Huazhong Agricultural University, Wuhan, People’s Republic of China 2 State Key Laboratory of Agriculture Microbiology, Huazhong Agricultural University, Wuhan, People’s Republic of China 3 Key Laboratory of Special Pathogens and Center for Emerging Infectious Diseases, Wuhan Institute of Virology, Chinese Academy of Sciences, Wuhan, China 4 Department of Cardiovascular Medicine, Zulfiqar Ali Bhutto Medical University, Pakistan Institute of Medical Sciences, Islamabad, Pakistan 5 Tongji Medical College, Huazhong University of Science And Technology, Wuhan, China Correspondence to: Zhang ShuJun, email: // Keywords : CRISPR/Cas9, genetic diseases, cancer, genome editing, next generation sequencing Received : March 12, 2016 Accepted : May 19, 2016 Published : May 26, 2016 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.