Therapeutics 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... -
CAR T Cell Success in Solid GI Tumors: Drug Discovery News Spotlight
While CAR T cell therapies have shown success primarily in hematologic malignancies, new advances are emerging for gastrointestinal (GI) solid tumors such as colorectal, gastric, and esophageal cancers. U.S. research teams have reported ... -
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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