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Delete DNA to treat mitochondrial diseases |
New compounds can find and help remove mutated mitochondrial DNA in cells
.
Photo Credit: Kyoto University/Mindy TakamiyaNew compounds can find and help remove mutated mitochondrial DNA in cells
Mitochondria perform many cellular functions, including energy production, biomolecule synthesis, and calcium homeostasis
.
Mitochondria have their own DNA, which is different from the DNA in the nucleus
In some mitochondrial diseases, mutant DNA coexists with normal DNA
.
"This state is called heterogeneity
Ganesh Namasivayam Pandian, the bioengineer who led the study, explained that the current methods for treating this type of mitochondrial disease are problematic
.
Some of them involve injecting genetic material into cells, which can lead to unnecessary changes
As a result, the research team developed a chemical-based method to overcome these problems
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They designed a compound consisting of mitochondrial penetrating peptide (MPP) and a polymer called pyridine-imidazole polyamide (PIP), which can be modified for specific DNA sequences
MPP brings the compound into the mitochondria, and PIP binds to the target DNA sequence in the mitochondria
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This allows chlorobutyrate to destroy the target DNA for elimination
"Our proof-of-concept research can be extended to mitochondrial mutations that cause diseases such as Leber's hereditary optic neuropathy
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This is an inherited vision loss that has not yet been proven treatment
Related paper information: https://doi.
https://doi.
org/10.
1016/j.
chembiol.
2021.
08.
003