Cutting-edge technologies can repair the underlying mutations that cause Rett syndrome.
How Gene Editing Works
Genes are made up of specific nucleotide bases (A, T, C and G), which encode for amino acids. Every three bases code for a specific amino acid. The MECP2 gene has 1,497 nucleotide bases that code for the 498 amino acids in the MECP2 protein. Alterations in the MECP2 gene range from a single letter to large sections of the gene being deleted or inserted. To learn more about the genetics of Rett syndrome please visit our Genetics Primer.
The CRISPR-Cas system uses two key enzymes to target and repair the mutated portion of genes. The first enzyme is a guide RNA that brings the Cas9 enzyme to the proper location in the genome. The second enzyme, called Cas9, which acts as a pair of molecular scissors that can cut DNA at specific locations so that bits of DNA can then be added or removed.
RSRT has invested almost $2 million in gene editing. RSRT-driven collaborations, initiatives, and investments have helped to generate all of the following gene editing programs.