A new study suggests genetic changes allow horses to produce more energy, while minimizing the toll the energy takes on cells ...

A nonsense mutation is a genetic mutation in a DNA sequence that results in a shorter, unfinished protein product. DNA is a chain of many smaller molecules called nucleotides. During protein ...

Many human diseases are caused by point mutations that affect RNA splicing. It has been observed that nonsense mutations that generate premature termination of translation products can drive the use ...

Alltrna’s CEO Michelle Warner said that the approach allows for basket trials that can group patients with different diseases together.

Pluvicto, Novartis’ blockbuster radiopharmaceutical for prostate cancer, has been greenlighted by the FDA for use earlier in ...

A genetic change that boosts a cell's aerobic capacity while also protecting it from excess stress could explain how horses became such powerful athletes, according to a new study in Science.

Inherited retinal degeneration (IRD) encompasses various disorders characterized by progressive loss of retinal photoreceptor ...

More information: Sanghun Kim et al, Comprehensive phenotypic assessment of nonsense mutations in mitochondrial ND5 in mice, Experimental & Molecular Medicine (2024). DOI: 10.1038/s12276-024-01333-9 ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines ...

The patient’s cells must produce the CFTR protein for the existing therapies to work, which leaves patients with nonsense mutations without options. Since its initial discovery, researchers believed ...

PTC Therapeutics (PTCT) announced that the European Commission has adopted the opinion of the Committee for Medicinal Products for Human Use of ...

Alltrna’s approach seeks to change this by focusing on nonsense mutations – premature stop codons – which occur in about 10% of genetic diseases and impact an estimated 30 million people ...