Nature Podcast Audio long read: Faulty mitochondria cause deadly diseases — fixing them is about to get a lot easier
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Nov 28, 2025 Discover how researchers are tackling the challenge of editing mitochondrial DNA to combat deadly diseases. Learn why traditional CRISPR techniques fall short and the innovative methods being developed, like DddA toxin-based editors. The potential of base editing and the creation of animal models are explored, showcasing early successes in reversing disease phenotypes. Challenges in safe delivery and genetic accuracy remain, but the landscape of mitochondrial therapies is rapidly evolving with promising biotech efforts on the horizon.
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Mitochondria Missed The CRISPR Revolution
- Mitochondrial DNA (mtDNA) is inaccessible to standard CRISPR tools and requires different approaches.
- Faulty mtDNA causes many incurable diseases, so accessing it is crucial for understanding and treating metabolic disorders.
Why Mitochondrial DNA Is Hard To Edit
- mtDNA differs from nuclear DNA: maternal inheritance, many copies per cell, higher mutation rate, and limited repair.
- These differences constrain editing strategies because mitochondria lack many nuclear DNA repair pathways.
Cutting Out Mutant mtDNA Can Shift The Balance
- Mitochondria often discard broken DNA rather than repair it, so cutting tools can selectively remove mutant genomes.
- Reducing mutant mtDNA lets healthy copies replicate and can lower disease-causing heteroplasmy levels.