Treatments that target a fragment of the mutant protein that causes Huntington's disease might be more effective than treatments—now in clinical trials—that target the whole protein but leave this ...

Peptide conjugation techniques are advancing CNS delivery of oligonucleotides, addressing blood-brain barrier challenges and ...

Acurastem received a grant to develop ALS therapies targeting SYF2, a regulator of TDP-43, in collaboration with academic research teams.

Researchers found that targeting a protein fragment, huntingtin 1a, restored baseline gene activity and reduced aggregate ...

Luxna Biotech & Inabata ink license agreement for manufacture and sale of modified nucleic acid GuNA amidite: Osaka, Japan Monday, March 16, 2026, 09:00 Hrs [IST] Luxna Biotech Co ...

The commentary titled 'Establishing a Commercial Solution for Extremely Rare Genetic Diseases' was published in Nature Biotechnology Commentary coincides with draft guidance from the FDA outlining a ...

Living with a rare disease often means years of uncertainty, misdiagnosis, and limited treatment options. But advances in ...

With AI, it's now possible for researchers to predict the three-dimensional structures of proteins directly from their ...

Cells in our bodies produce RNA based on genetic information stored in DNA, and RNA serves as a blueprint for making proteins ...

On Monday, a paper announcing that all four DNA bases had been found on an asteroid sparked a lot of headlines. But many of ...