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Treating Genetic Disorders Using State-Of-The-Art Technology.
Curr Issues Mol Biol. 2018; 26:33-46.CI

Abstract

CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-CRISPR associated Protein 9), basically a bacterial immune system is now widely applicable to engineer genomes of a number of cells and organisms because of its simplicity and robustness. In research avenue the system has been optimized to regulate gene expression, modify epigenome and edit target locus. These applications make CRISPR/Cas9, a technology of choice to edit disease causing mutations as well as the epigenome more efficiently than ever before. Meanwhile its application in in vivo and ex vivo cells is encouraging the scientific community for more vigorous gene therapy and in clinical setups for therapeutic genome editing. Here we review the recent advances that CRISPR-Cas9 mediated genome editing has achieved and is reported in previous studies and address the challenges associated with it.

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Authors+Show Affiliations

Jamal M
State Key Laboratory of Agriculture Microbiology, Huazhong Agricultural University, Wuhan, The People's Republic of China, and College of Animal Science and Medicine, Huazhong Agricultural University, Wuhan, The People's Republic of China.
Ullah A
Institute of Biochemistry and Biotechnology, University of Veterinary and Animal Sciences, Lahore, Pakistan.
Ahsan M
State Key Laboratory of Agriculture Microbiology, Huazhong Agricultural University, Wuhan, The People's Republic of China, and College of Animal Science and Medicine, Huazhong Agricultural University, Wuhan, The People's Republic of China.
Tyagi R
State Key Laboratory of Agriculture Microbiology, Huazhong Agricultural University, Wuhan, The People's Republic of China, and College of Animal Science and Medicine, Huazhong Agricultural University, Wuhan, The People's Republic of China.
Habib Z
State Key Laboratory of Agriculture Microbiology, Huazhong Agricultural University, Wuhan, The People's Republic of China, and College of Animal Science and Medicine, Huazhong Agricultural University, Wuhan, The People's Republic of China.
Khan FA
Key Laboratory of Agricultural Animal Genetics, Breeding and Reproduction of the Ministry of Education, College of Animal Science and Technology, Huazhong Agricultural University, Wuhan, The People's Republic of China.
Rehman K
State Key Laboratory of Agriculture Microbiology, Huazhong Agricultural University, Wuhan, The People's Republic of China, and College of Animal Science and Medicine, Huazhong Agricultural University, Wuhan, The People's Republic of China.

MeSH

Anemia, Sickle CellBacterial ProteinsCRISPR-Associated Protein 9CRISPR-Cas SystemsClustered Regularly Interspaced Short Palindromic RepeatsCystic FibrosisEndonucleasesFanconi AnemiaGene EditingGenetic TherapyHumansLeukemia, Myeloid, AcuteMuscular Dystrophy, DuchenneNeurodegenerative DiseasesPolycythemia VeraRNA, Guide

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

28879854

Citation

Jamal, Muhammad, et al. "Treating Genetic Disorders Using State-Of-The-Art Technology." Current Issues in Molecular Biology, vol. 26, 2018, pp. 33-46.
Jamal M, Ullah A, Ahsan M, et al. Treating Genetic Disorders Using State-Of-The-Art Technology. Curr Issues Mol Biol. 2018;26:33-46.
Jamal, M., Ullah, A., Ahsan, M., Tyagi, R., Habib, Z., Khan, F. A., & Rehman, K. (2018). Treating Genetic Disorders Using State-Of-The-Art Technology. Current Issues in Molecular Biology, 26, 33-46. https://doi.org/10.21775/cimb.026.033
Jamal M, et al. Treating Genetic Disorders Using State-Of-The-Art Technology. Curr Issues Mol Biol. 2018;26:33-46. PubMed PMID: 28879854.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Treating Genetic Disorders Using State-Of-The-Art Technology. AU - Jamal,Muhammad, AU - Ullah,Arif, AU - Ahsan,Muhammad, AU - Tyagi,Rohit, AU - Habib,Zeshan, AU - Khan,Faheem Ahmad, AU - Rehman,Khaista, Y1 - 2017/09/07/ PY - 2017/9/8/pubmed PY - 2018/8/9/medline PY - 2017/9/8/entrez SP - 33 EP - 46 JF - Current issues in molecular biology JO - Curr Issues Mol Biol VL - 26 N2 - CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-CRISPR associated Protein 9), basically a bacterial immune system is now widely applicable to engineer genomes of a number of cells and organisms because of its simplicity and robustness. In research avenue the system has been optimized to regulate gene expression, modify epigenome and edit target locus. These applications make CRISPR/Cas9, a technology of choice to edit disease causing mutations as well as the epigenome more efficiently than ever before. Meanwhile its application in in vivo and ex vivo cells is encouraging the scientific community for more vigorous gene therapy and in clinical setups for therapeutic genome editing. Here we review the recent advances that CRISPR-Cas9 mediated genome editing has achieved and is reported in previous studies and address the challenges associated with it. SN - 1467-3045 UR - https://www.unboundmedicine.com/medline/citation/28879854/Treating_Genetic_Disorders_Using_State_Of_The_Art_Technology_ L2 - https://www.caister.com/cimb/vb/v26/33.pdf DB - PRIME DP - Unbound Medicine ER -