Table 3 Nucleases, proposed for elimination of mutant mtDNA
From: Current advances in gene therapy of mitochondrial diseases
Nuclease | Brief description | Target | Reference |
|---|---|---|---|
mitoTev‐TALE | Monomeric nuclease derived from T4 phage (I‐TevI). Smaller than TALEN and hence more suitable for package its coding gene into viral vectors | Programmable | [184] |
PstI | Bacterial PstI endonuclease was recoded to optimize expression in mammalian cells and cloned downstream to MTS. Human mtDNA harbours two restriction sites for PstI (at positions 6914 and 9024) | Positions 6914 and 9024 of human mtDNA | [173] |
Smal | Smal gene from Serratia marcescens appended by MTS sequence cloned from pCoxIV of Saccharomyces cerevisiae | T8399G mutation in NARP | [197] |
Mito-ApaLI-HA | Synthetic ApaLI endonuclease from Acetobacter pasterianus added by MTS coding sequence. For immunological detection of ApaLI, a hemagglutinin epitope tag was added to its C terminus | Specific site in mtDNA of BALB mice | [198] |