All articles tagged with #gene silencing
Arrowhead Pharmaceuticals' gene-silencing drug candidate ARO-INHBE, in combination with Eli Lilly's Zepbound, showed promising early results in reducing weight and harmful visceral fat in obese patients with diabetes, potentially intensifying competition in weight loss drug development.
Supportive Oligonucleotide Therapy (SOT) is an emerging, experimental gene-silencing treatment showing promise for chronic infections like Lyme disease, but it is not yet FDA-approved and requires further research to confirm safety and efficacy.
The article discusses the development of programmable antisense oligomers (ASOs) delivered via cell-penetrating peptides to study gene function in bacteriophage ΦKZ, revealing essential phage genes and providing a versatile tool for phage research and potential therapeutic applications.
Arrowhead Pharmaceuticals' shares surged after announcing a $11.38 billion licensing deal with Sarepta Therapeutics for gene-silencing products. The agreement includes $825 million upfront, $250 million over five years, $300 million in near-term milestones, and up to $10 billion in future milestones and royalties. This deal strengthens Sarepta's pipeline in neuromuscular and genetic treatments, while extending Arrowhead's cash runway into 2028. Both companies' stocks rose significantly following the announcement.
Researchers at Vilnius University's Life Sciences Center have developed a novel CRISPR system that silences genes without cutting DNA, using an RNA-guided complex to recruit the enzyme DinG for gene suppression. This method, which involves the formation of R-loops, offers a safer alternative for gene modification, potentially advancing genome editing and biotechnology applications.
Biotech companies are exploring RNA interference to develop longer-lasting obesity treatments, potentially shifting from weekly hormone-targeting drugs like Wegovy to less frequent, gene-targeted therapies.
Researchers have developed a hit-and-run epigenome editing strategy for durable and efficient gene silencing in vivo, using ZFP-based engineered transcriptional repressors (ETRs) to target the Pcsk9 gene in mice. The ZFP-based ETRs induced stable and specific epi-silencing of Pcsk9, resulting in reduced circulating levels of PCSK9 and LDL-associated cholesterol. The study also demonstrated the durability of epigenetic silencing even after active cell replication, paving the way for potential clinical applications of the epi-silencing technology.
Two new studies conducted by stem cell scientists at the University of Southern California have shed light on the potential for regenerative hearing treatments that could cure deafness. The research focuses on understanding the process of gene silencing in the inner ear and how it can be reversed to regenerate sensory hearing cells. The studies identify key genes, Sox4 and Sox11, that play a role in the development of sensory hearing cells and suggest that manipulating these genes could promote the recovery of sensory hearing cells in the mature inner ear. The findings offer hope for future treatments to reverse hearing loss.
Researchers from the University of California, Irvine have developed a DNA enzyme that can distinguish between two RNA strands inside a cell and cut the disease-associated strand while leaving the healthy strand intact. The Dz 46 enzyme specifically targets the allele-specific RNA mutation in the KRAS gene, found in 25 percent of all human cancers. This breakthrough "gene silencing" technology could revolutionize the development of DNAzymes for treating cancer, infectious diseases, and neurological disorders, offering patients an innovative, precision medicine treatment.
A new drug called BIIB080 has been found to remove harmful proteins linked to Alzheimer's disease by lowering levels of tau protein with a 'gene silencing' approach. In an early-stage trial, patients with mild Alzheimer's who received the highest dose of the drug had a 50% reduction in levels of tau in their central nervous system after 24 weeks with no serious side-effects. A phase 2 trial of the drug is currently aiming to enroll 700 people, and further studies will be vital to see this treatment approach reach the clinic.