• ISSN 1674-8301
  • CN 32-1810/R
Volume 35 Issue 2
Mar.  2021
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Article Contents
Colin T. Konishi, Chengzu Long. Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases[J]. The Journal of Biomedical Research, 2021, 35(2): 148-162. doi: 10.7555/JBR.34.20200105
Citation: Colin T. Konishi, Chengzu Long. Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases[J]. The Journal of Biomedical Research, 2021, 35(2): 148-162. doi: 10.7555/JBR.34.20200105

Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases

doi: 10.7555/JBR.34.20200105
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  • Corresponding author: Chengzu Long, Leon H. Charney Division ofCardiology, Helen and Martin Kimmel Center forStem Cell Biology, Department of Neurology, Department of Neuroscience andPhysiology, New York UniversityGrossman School of Medicine, 435 East30th Street, New York, NY 10016, USA. Tel/Fax:+1-212-263-9100/+1-212-263-9115. E-mail: chengzu.long@nyumc.org
  • Received: 2020-07-10
  • Accepted: 2020-10-15
  • Published: 2020-11-27
  • Issue Date: 2021-03-26
  • There are an estimated 10 000 monogenic diseases affecting tens of millions of individuals worldwide. The application of CRISPR/Cas genome editing tools to treat monogenic diseases is an emerging strategy with the potential to generate personalized treatment approaches for these patients. CRISPR/Cas-based systems are programmable and sequence-specific genome editing tools with the capacity to generate base pair resolution manipulations to DNA or RNA. The complexity of genomic insults resulting in heritable disease requires patient-specific genome editing strategies with consideration of DNA repair pathways, and CRISPR/Cas systems of different types, species, and those with additional enzymatic capacity and/or delivery methods. In this review we aim to discuss broad and multifaceted therapeutic applications of CRISPR/Cas gene editing systems including in harnessing of homology directed repair, non-homologous end joining, microhomology-mediated end joining, and base editing to permanently correct diverse monogenic diseases.


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