Single drug provides first evidence of 'nearly universal' pharmacological chaperone for rare disease

Published Date: 24 Sep 2025

A study published in Nature Structural & Molecular Biology is the first time researchers have shown evidence that a single drug, already licensed for medical use, can stabilize nearly all mutated versions of a human protein, ...

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In a surprising discovery, a "sticky molecule" that occurs naturally in our blood vessels could be both a culprit behind blood clots and organ failure during COVID and long COVID and the key to new treatments to counter COVID-related ...

A new discovery from the lab of YouYang Zhao, Ph.D., from Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago, opens promising new directions for treatment of life-threatening ...

Genetic susceptibility to neurodevelopmental conditions such as schizophrenia, autism, and ADHD can be linked, in part, to distinct epigenetic signatures in newborn cord blood. An analysis of nearly 6,000 newborns published ...

In a major step forward for cancer care, researchers at ChristianaCare's Gene Editing Institute have shown that disabling the NRF2 gene with CRISPR technology can reverse chemotherapy resistance in lung cancer. The approach ...

Mice pups conceived with in vitro fertilization (IVF) in the lab have slightly increased rates of DNA errors, or mutations, compared to pups conceived naturally, a new study on artificial reproductive technologies suggests.

Researchers have identified that the precisely timed transcription of two genes named grim and reaper is responsible for the targeted death of neurons within the developing nervous system of female flies. This group of neurons ...

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