The power and the promise of CRISPR/Cas9 genome editing for clinical application with gene therapy

Background: Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is derived from the bac-terial innate immune system and engineered as a robust gene-editing tool. Due to the higher specificity and efficiency of CRISPR/Cas9, it has been widely applied to many genetic and non-genetic disease, including cancers, genetic hemolytic diseases, acquired immunodeficiency syndrome, cardiovascular dis-eases, ocular diseases, and neurodegenerative diseases, and some X-linked diseases. Furthermore, in terms of the therapeutic strategy of cancers, many researchers used the CRISPR/Cas9 technique to cure or alleviate cancers through different approaches, such as gene therapy and immune therapy. Aim of Review: Here, we conclude the recent application and clinical trials of CRISPR/Cas9 in non -cancer-ous diseases and cancers and pointed out some of the problems to be solved. Key Scientific Concepts of Review: CRISPR/Cas9, derived from the microbial innate immune system, is developed as a robust gene-editing tool and has been applied widely. Due to its high accuracy and effi-ciency, CRISPR/Cas9 techniques may provide a great chance to treat some gene-related diseases by dis-rupting, inserting, correcting, replacing, or blocking genes for clinical application with gene therapy. (c) 2022 The Authors. Published by Elsevier B.V. on behalf of Cairo University. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).