Singapore Scientists Develop Novel CRISPR-Cas9-Based Gene Editor to Correct Disease-Causing Mutations; Advance May Open Up Possible Treatment for Up to 40% of Genetic Disorders Caused by Single-Nucleotide Mutations

A team of researchers from the Agency for Science, Technology, and Research's (A*STAR) Genome Institute of Singapore (GIS) has developed a CRISPR-based gene editor, C-to-G Base Editor (CGBE), to correct mutations that cause genetic disorders. Their research was published online on March 2, 2021 in Nature Communications. The open-access article is titled “Programmable C:G to G:C Genome Editing with CRISPR-Cas9-Directed Base Excision Repair Proteins” ( ). One in seventeen people in the world suffers from some type of genetic disorder. Chances are, you or someone you know--a relative, friend, or colleague--is one of approximately 450 million people affected worldwide. Mutations responsible for these disorders can be caused by multiple mutagens--from sunlight to spontaneous errors in your cells. The most common mutation by far is the single-based substitution, in which a single-base in the DNA (such as G) is replaced by another base (such as C). For example, countless cystic fibrosis patients worldwide have C instead of G, leading to defective proteins that cause the genetic disease. In another case, replacing A with T in hemoglobin causes sickle cell anemia. To fix these substitutions, the team invented a CRISPR-based gene editor that precisely changes the defective C within the genome to the desired G. This C-to-G base editor (CGBE) invention opens up treatment options for approximately 40 per cent of the single-base substitutions that are associated with human diseases such as the aforementioned cystic fibrosis, cardiovascular diseases, musculoskeletal diseases, and neurological disorders.
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