CRISPR/Cas system: A game changing genome editing technology, to treat human genetic diseases

• Published in: Gene 2019-02, Vol.685, p.70-75
• Main Authors: Hussain, Wajid, Mahmood, Tariq, Hussain, Jawad, Ali, Niyaz, Shah, Tariq, Qayyum, Sadia, Khan, Ibrar
• Format: Article
• Language: English
• Subjects: Animals; Clustered Regularly Interspaced Short Palindromic Repeats; CRISPR-Associated Protein 9 - metabolism; CRISPR-Cas Systems; Gene; Gene Editing; Gene Expression Regulation; Genetic Therapy; Genome editing; Genomics - methods; HDR or NHEJ; Homologous Recombination; Humans; Molecular scissors and CRISPR/Cas9 system; RNA, Guide, CRISPR-Cas Systems
• ISSN: 0378-1119; 1879-0038
• DOI: 10.1016/j.gene.2018.10.072

Genes, are the functional units of heredity that used as an instructors to make proteins either to become the functional or structural part of the cell. Hence, the proteins get more attention because most of the life functions depends on it. Any mutation or alteration in genome sequences results in complete loss of function or formation of abnormal protein which leads to hereditary disorder. Gene therapy on the other hand, used as a remedy, a process that make correction in the gene which is responsible for genomic disorders. The treatment of disease state depends on the understanding of their genetic basis. While, numerous molecular genome editing tools have been developed and are being utilized to translate the abstract of gene therapy into reality, but the problem is still a mystery. The genome editing molecular scissors can be applied to dictate the selected genetic products that can have the therapeutic power. Thus, editing the specific sequences depends on the type of strategies being used by a molecule such is HDR or NHEJ. CRISPR/Cas9 editing technology can use in disease model to study the genitival disorders. One side the CRISPR technology seemed to be extremely accurate but on the other side it has some harmful effects i.e. Cas9 proteins sometimes cuts the similar sequences other than the specific targeted and Off-targeting Sequences etc. Urgent attention and improvement are needed for various implication of CRISPR/Cas9 technology, including the delivery, precision and control over the mention system. This review presents the current scenario of genome editing in vivo and its implications for the future of human genetic disease treatment as well as genome throughput potency. •Gene therapy (replacing the faulty gene), holds promise for treating a wide range of diseases.•CRISPR is a family of DNA sequences found within the genomes of prokaryotic organisms such as bacteria and archaea.•CRISPR, and associated proteins (Cas) comprise the CRISPR-Cas system, which confers adaptive immunity in many microbes.•CRISPR associated Cas9 (endonuclease) editing technology can use in disease model to study the human genitival disorders.•CRISPR/Cas9 proteins also have harmful effects i.e. sometimes also cuts the sequences other than the specific targeted.