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Treating Genetic Disorders Using State-Of-The-Art Technology.
- Source :
-
Current issues in molecular biology [Curr Issues Mol Biol] 2018; Vol. 26, pp. 33-46. Date of Electronic Publication: 2017 Sep 07. - Publication Year :
- 2018
-
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.
- Subjects :
- Anemia, Sickle Cell genetics
Anemia, Sickle Cell pathology
Anemia, Sickle Cell therapy
Bacterial Proteins metabolism
CRISPR-Associated Protein 9
Cystic Fibrosis genetics
Cystic Fibrosis pathology
Cystic Fibrosis therapy
Endonucleases metabolism
Fanconi Anemia genetics
Fanconi Anemia pathology
Fanconi Anemia therapy
Humans
Leukemia, Myeloid, Acute genetics
Leukemia, Myeloid, Acute pathology
Leukemia, Myeloid, Acute therapy
Muscular Dystrophy, Duchenne genetics
Muscular Dystrophy, Duchenne pathology
Muscular Dystrophy, Duchenne therapy
Neurodegenerative Diseases genetics
Neurodegenerative Diseases pathology
Neurodegenerative Diseases therapy
Polycythemia Vera genetics
Polycythemia Vera pathology
Polycythemia Vera therapy
RNA, Guide, CRISPR-Cas Systems metabolism
Bacterial Proteins genetics
CRISPR-Cas Systems
Clustered Regularly Interspaced Short Palindromic Repeats
Endonucleases genetics
Gene Editing methods
Genetic Therapy methods
RNA, Guide, CRISPR-Cas Systems genetics
Subjects
Details
- Language :
- English
- ISSN :
- 1467-3045
- Volume :
- 26
- Database :
- MEDLINE
- Journal :
- Current issues in molecular biology
- Publication Type :
- Academic Journal
- Accession number :
- 28879854
- Full Text :
- https://doi.org/10.21775/cimb.026.033