Current issues in molecular biology · 2017
Treating Genetic Disorders Using State-Of-The-Art Technology.
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Jamal Muhammad, Ullah Arif, Ahsan Muhammad, Tyagi Rohit, Habib Zeshan, Khan Faheem Ahmad, Rehman Khaista
Аннотация
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.
Предметные рубрики 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, CRISPR-Cas Systems