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