Treatment with a cell-penetrating peptide that prevented nuclear export of unprocessed C9ORF72 RNA and its subsequent translation into neurotoxic dipeptide repeat proteins reduced motor neuron damage and death both in fruit fly models of amyotrophic lateral sclerosis (ALS), and in patient-derived induced neuronal precursor cells (iNPCs). The work suggests that targeting nuclear export could be a therapeutic option in ALS, and possibly also frontotemporal dementia (FTD), where C9ORF72 mutations also play a role.
Genascence Corp. has been awarded US$11.6 million over 4 years from the California Institute for Regenerative Medicine (CIRM) to help advance the company's GNSC-001 gene therapy for knee osteoarthritis (OA). The funding will support a phase Ib trial and manufacturing activities.
Using single-cell RNA sequencing of deer antler at different stages of their annual cycle of regeneration, Chinese researchers have identified a progenitor cell population that drove antler regeneration. The authors of an accompanying editorial wrote the findings, which were published in the Feb. 24, 2023, issue of Science, “add to the emerging idea that blastema progenitor cells are a common stem cell type in mammalian appendage regeneration.”
Antibodies against tumor necrosis factor (TNF) and IL-6 receptor are the most effective available therapies against rheumatoid arthritis (RA). However, no cure for RA exists. In work published in Science Translational Medicine, scientists from Sanofi R&D (Sanofi SA) investigated whether dual inhibition of TNF and IL-6 could improve the therapeutic efficacy against RA.
Hebei Feinisi Biotechnology Co. Ltd. has synthesized shared epitope-containing HLA DR molecule (SE-DR) affinity peptides reported to be useful for the treatment of rheumatic diseases.
Globus Medical Inc. entered into a definitive agreement to buy Nuvasive Inc. in an all-stock transaction that will combine two leaders in the musculoskeletal industry into one of the largest companies in the spinal surgery market. While there are many companies in the spine market, the acquisition may still set off an alarm among regulators concerned about consolidation in the field. Both boards unanimously approved the deal.
Bone-resorbing osteoclasts are key players in bone remodeling and their dysfunction has been implicated in several bone disorders such as fibrous dysplasia. Osteoclasts derive from monocytes stimulated by several cytokines, such as M-CSF and RANKL.
The lack of dystrophin causes Duchenne muscular dystrophy (DMD), a muscle-wasting disease that is often accompanied by heart failure due to cardiomyocyte death and fibrosis that can lead to death of the patient. It has been proven that telomere shortening is a hallmark of DMD cardiomyocytes. Researchers from the Stanford School of Medicine have recently investigated whether preventing telomere shortening and attrition may be a therapeutic approach in DMD.
Osteoarthritis (OA) is the most common joint disease and a leading cause of disability worldwide. Its treatment is still elusive due to difficulties with early diagnoses and patient risk identification. This leads to a need for reliable biomarkers for OA prognosis or to identify patients at risk of progression.
Dice Alpha Inc. has synthesized phenyl acetamide compounds acting as interleukin-17A (IL-17A) production inhibitors reported to be useful for the treatment of psoriasis, radiographic axial spondyloarthritis (ankylosing spondylitis), hidradenitis suppurativa, spondyloarthritis, psoriatic and rheumatoid arthritis.
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