SoylentNews

Arthur T Knackerbracket has processed the following story:

Mutant DNA sequences inside cellular mitochondria can be eliminated using a bespoke chemical compound. The approach, developed by scientists at Kyoto University's Institute for Integrated Cell-Material Science (iCeMS) in Japan, could lead to better treatments for mitochondrial diseases. The researchers published their findings in the journal Cell Chemical Biology.

[...] In some mitochondrial diseases, mutated DNA and normal DNA co-exist. "This state is called heteroplasmy," explains Kyoto University's Takuya Hidaka, the first author of the study. "Mitochondrial function can be maintained by normal mitochondrial DNA when the heteroplasmy level is low. But it is impaired when mutated DNA exceeds a critical threshold. To cure mitochondrial diseases, we need to be able to remove mutant mitochondrial DNA from cells."

Current approaches for such mitochondrial diseases are problematic, explains iCeMS bioengineer Ganesh Namasivayam Pandian, who led the study. Some involve injecting genetic material into cells, which could lead to unwanted alterations. In others, antioxidant drugs are administered to reduce the impacts of the mutant DNA, without addressing the core mutation.

Pandian worked with iCeMS chemical biologist Hiroshi Sugiyama, Takuya Hidaka and colleagues to develop a chemical-based approach that overcomes these issues.

[...] "Our proof-of-concept study can be extended to mitochondrial mutations that cause diseases like Leber's hereditary optic neuropathy, an inherited form of vision loss that currently has no proven treatment," says Pandian.

Journal Reference:
Takuya Hidaka. Targeted elimination of mutated mitochondrial DNA by a multi-functional conjugate capable of sequence-specific adenine alkylation, Cell Chemical Biology (DOI: 10.1016/j.chembiol.2021.08.003)

Original Submission