US EditionScientists Use Gene Editing to Correct Harmful Mitochondrial Mutations in Human Cells
- Researchers in the Netherlands successfully edited harmful mitochondrial DNA mutations using a base editor, marking progress toward treating mitochondrial diseases.
- This development builds on challenges since CRISPR could not cross the mitochondrial membrane, and new base editors like DdCBE enable targeted single-letter DNA changes without cutting.
- The team corrected mutations in disease-linked human cells, including liver cells engineered with energy-impairing mutations and patient-derived skin cells with Gitelman-like syndrome.
- The authors highlighted that mitochondrial base editing holds significant promise for advancing disease modeling and developing new therapeutic strategies.
- These advances offer new hope for gene therapies addressing rare mitochondrial disorders affecting 1 in 5,000 people and could enable clinical treatments using mRNA and lipid nanoparticle delivery.
14 Articles
14 Articles
Scientists Used Gene Editing to Make Baby Mice From Two Males
Since the dawn of humanity, biology has followed a strict and elegant rule: you need chromosomes from mom and some from dad if you want to make a kid. But now, researchers are using genetic editing techniques to make babies from two male mice. According to a study published in PNAS and reported by Ars Technica, researchers custom-engineered methylation, using CRISPR-style tech, and surgically modified the imprinting sites on a sperm genome, esse…
Developing mitochondrial base editors with diverse context compatibility and high fidelity via saturated spacer library
DddA-derived cytosine base editors (DdCBEs) greatly facilitated the basic and therapeutic research of mitochondrial DNA mutation diseases. Here we devise a saturated spacer library and successfully identify seven DddA homologs by performing high-throughput sequencing based screen. DddAs of Streptomyces sp. BK438 and Lachnospiraceae bacterium sunii NSJ-8 display high deaminase activity with a strong GC context preference, and DddA of Ruminococcus…
Eldiario.es
elDiarioARResearchers have used a genetic tool known as the base editor, which allows them to get to where CRISPR does not arrive and opens new hopes for the treatment of rare genetic diseases inherited via maternal Hemeroteca - “Why do I have to be like this?” The documentary that puts before the mirror the inequality in the heel test A team of researchers from the Netherlands has corrected for the first time, by genetic editing, some mutations in mitoch…
Scientists use gene editing to correct harmful mitochondrial mutations in human cells
In a step toward treating mitochondrial diseases, researchers in the Netherlands have successfully edited harmful mutations in mitochondrial DNA using a genetic tool known as a base editor. The results, published in the open-access journal PLOS Biology, offer new hope for people with rare genetic conditions.
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