The Ly Group

Bioorganic Chemistry  •  Combinatorial Chemistry  •  Chemical Genetics  •  Functional Genomics


Gene Correction

Many genetic diseases occur as the result of single-base mutations of key, protein-encoding genes. In principle, it should be possible to treat (or perhaps cure) such genetic diseases by correcting the mutations—i.e. changing the mutated base-pairs to the original sets. Earnest attempts have been made to try to tackle this problem; however, there appears to be no solution insight. Our work in this area explores the possibility of using especially designed Janus-based γ-peptide nucleic acids (J-γPNAs) to invade genomic DNA, the endogenous repair enzymes to excise the mutant base-pairs, and the innate recombination process to correct the mutations.