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CRISPR Genome Editing Technology and its Application in Genetic Diseases: A Review.
Curr Pharm Biotechnol. 2020 Jun 21 [Online ahead of print]CP

Abstract

Gene therapy has been a long-lasting goal of scientists and there are many ideal methods and tools that have been developed to correct disease-causing mutations in human. Recently, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology has been progressively used for the assessment or treatment of human diseases, including Thalassemia, Parkinson disease, Cystic Fibrosis, Glaucoma, Huntington's disease and Human Immunodeficiency Virus/Acquired Immunodeficiency Syndrome (HIV/AIDS). CRISPR sequences is a part of the bacterial immune system, which includes nuclease Cas enzyme and a RNA sequence. The RNA sequence is unique and pathogen-specific, which identifies and binds to the DNA of invasive viruses, and allows the nuclease Cas enzyme to cut identified DNA and destroy the invasive viruses. This feature provides a possibility to edit mutations in DNA sequence of live cells through replacement of a specific targeted RNA sequence with the RNA sequence in the CRISPR system. Previous studies showed the improvement steps in confrontation to human diseases caused by single nucleotide mutations using this system. In this review, we first concisely introduce CRISPR and its functions, and next study the use of CRISPR in the treatment of human diseases.

Authors+Show Affiliations

Department of Medical Biotechnology, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad. Iran.Halal Research Center of IRI, FDA, Tehran. Iran.Department of Biotechnology, Mashhad University of Medical Sciences, Vakilabad blvd.. Iran.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

32564746

Citation

Khatibi, Sepideh, et al. "CRISPR Genome Editing Technology and Its Application in Genetic Diseases: a Review." Current Pharmaceutical Biotechnology, 2020.
Khatibi S, Sahebkar A, Bakhtiari SHA. CRISPR Genome Editing Technology and its Application in Genetic Diseases: A Review. Curr Pharm Biotechnol. 2020.
Khatibi, S., Sahebkar, A., & Bakhtiari, S. H. A. (2020). CRISPR Genome Editing Technology and its Application in Genetic Diseases: A Review. Current Pharmaceutical Biotechnology. https://doi.org/10.2174/1389201021666200621161610
Khatibi S, Sahebkar A, Bakhtiari SHA. CRISPR Genome Editing Technology and Its Application in Genetic Diseases: a Review. Curr Pharm Biotechnol. 2020 Jun 21; PubMed PMID: 32564746.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - CRISPR Genome Editing Technology and its Application in Genetic Diseases: A Review. AU - Khatibi,Sepideh, AU - Sahebkar,Amirhossein, AU - Bakhtiari,Seyed Hamid Aghaee, Y1 - 2020/06/21/ PY - 2020/02/14/received PY - 2020/04/29/revised PY - 2020/05/18/accepted PY - 2020/6/23/entrez KW - AIDS KW - CRISPR technology KW - genome editing KW - human genetic disease KW - mutations. KW - nuclease cas enzyme JF - Current pharmaceutical biotechnology JO - Curr Pharm Biotechnol N2 - Gene therapy has been a long-lasting goal of scientists and there are many ideal methods and tools that have been developed to correct disease-causing mutations in human. Recently, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology has been progressively used for the assessment or treatment of human diseases, including Thalassemia, Parkinson disease, Cystic Fibrosis, Glaucoma, Huntington's disease and Human Immunodeficiency Virus/Acquired Immunodeficiency Syndrome (HIV/AIDS). CRISPR sequences is a part of the bacterial immune system, which includes nuclease Cas enzyme and a RNA sequence. The RNA sequence is unique and pathogen-specific, which identifies and binds to the DNA of invasive viruses, and allows the nuclease Cas enzyme to cut identified DNA and destroy the invasive viruses. This feature provides a possibility to edit mutations in DNA sequence of live cells through replacement of a specific targeted RNA sequence with the RNA sequence in the CRISPR system. Previous studies showed the improvement steps in confrontation to human diseases caused by single nucleotide mutations using this system. In this review, we first concisely introduce CRISPR and its functions, and next study the use of CRISPR in the treatment of human diseases. SN - 1873-4316 UR - https://www.unboundmedicine.com/medline/citation/32564746/CRISPR_Genome_Editing_Technology_and_its_Application_in_Genetic_Diseases:_A_Review L2 - http://www.eurekaselect.com/182996/article DB - PRIME DP - Unbound Medicine ER -
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