Science News– category –
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[Science News] Nature Highlight: Small Molecules Restore Mutant Mitochondrial DNA Polymerase Activity
A new study published in the June 12, 2025 issue of Nature reports that the small molecule PZL-A can directly restore the function of DNA polymerase gamma (POLγ), impaired by POLG mutations causing severe mitochondrial disorders. This wo... -
[Science News] Nuclear Pore Complex Dysfunction Underlies ALS/FTD via C9orf72 Mutations: Nature Summary (With My Thoughts)
A U.S. research team has revealed that repeat expansions in the C9orf72 gene — a major cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) — induce dysfunction of the nuclear pore complex (NPC) in neurons. In C... -
Epigenetic Editing Expands the Reach of Gene Therapy: Drug Discovery News Spotlight
A U.S. research team has demonstrated the therapeutic potential of epigenetic editing — a strategy that regulates gene expression without altering the DNA or RNA sequence itself. Unlike conventional gene editing, this approach employs pr... -
New Gene Therapy Approach for Rare Childhood Blindness: Drug Discovery News Spotlight
A U.S. research team has developed a novel gene therapy for Leber Congenital Amaurosis (LCA), a rare form of childhood blindness. Unlike conventional AAV-based gene augmentation, this approach utilizes RNA editing technology to directly ...
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