Scientists receive $14.7 million to improve vector design for gene therapies targeting alpha-1 antitrypsin deficiency

In 'Spider-Man: Brand New Day,' Peter Parker devises a superpower inhibitor based on a real therapy using RNA.

Erik J. Sontheimer, PhD, the Pillar Chair in Biomedical Research, professor and vice chair of RNA therapeutics, has been appointed chair of the RNA Therapeutics Institute, effective in January of 2027...

Li Li, PhD, discusses how the FDA's approval of the first mRNA flu vaccine could accelerate the development of future mRNA-based therapies while highlighting the scientific challenges that remain.

Viral vectors for gene and cell therapy are typically produced by transfecting plasmids into mammalian cells. Because plasmid DNA is not replicated in these cells, large quantities are required to express...

Biomolecular condensates, such as germ granules, organize RNAi pathways critical for fertility and genome regulation. However, the protein composition and functional contributions of these condensates...

The dysferlinopathies are a spectrum of autosomal recessive muscle diseases caused by mutations in the dysferlin gene (DYSF) gene. Clinical manifestations vary from asymptomatic hyperCKemia to severe...

Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder caused by a dominant mutation in the <i>ACVR1</i> gene (R206H, 97% of cases), leading to debilitating heterotopic ossification (HO)...

Homologous recombination (HR) requires efficient homology search and strand invasion, yet how homologous templates are identified within nuclear space remains unclear. Here, we identify G-quadruplex (G4)...

Precision engineering of CRISPR/Cas9 components has advanced genome editing toward therapeutic applications. Completely chemically stabilized guide RNAs (gRNAs) have the potential to improve <i>in vivo</i>...

These findings establish Ninj1 as a critical mediator of chronic lung inflammation and a translationally relevant therapeutic target, highlighting Ninj1-directed ASO therapy as a promising strategy for...