A new gene editing tool that helps cellular machinery skip parts of genes responsible for diseases has been applied to reduce the formation of amyloid-beta plaque precursors in a mouse model of ...

This study provides important insights into the regulation of a retained intron in the mRNA coding for OGT, a process known to be regulated by the O-GlcNAc cycling system, and highlights the ...

Cell and gene therapies are revolutionizing medicine. Looking to 2025, advancements in allogeneic therapies, streamlined ...

The full spectrum of gene expression regulation associated with many neurodegenerative diseases, including Alzheimer’s, is ...

Del-brax (AOC 1020) for FSHD showed a 50% reduction in DUX4 gene expression ... marking a promising advancement for exon 44 skipping therapies. I reiterate my “Strong Buy” due to its pipeline ...

For example, an sQTL of the TCHP gene has been identified ... the high average sequencing depth and the "exon painting" effect (incomplete reverse transcription along with stochastic mRNA cleavage ...

2024) Pluripotency is thought to have emerged with the appearance of multicellular animals some 700 million years ago, so it stands to reason that transcription factors associated with stem cell ...

Dec. 4, 2024 — Researchers have uncovered a serious side effect of using the CRISPR-Cas gene scissors. A molecule designed to make the process more efficient destroys parts of the ...

Combined, our findings provide significant insight into the mechanisms underlying RBP-mediated gene expression regulation in response to growth ... AS events across all four treatment comparisons by ...