👏 Wonderful to see our research featured on the cover of @natureportfolio.nature.com journal Bone Research (@brjournal.bsky.social)! Congratulations to my co-authors from the @royalvetcollege.bsky.social and UCB .
#sclerosteosis #raredisease #drugdiscovery
📢 New Publication: I am delighted to share our most recent paper, published in @natureportfolio.nature.com Journal Bone Research "Porcupine inhibition is a promising pharmacological treatment for severe sclerosteosis pathologies".
www.nature.com/articles/s41...
Excited to share the latest chapter of our work on fat-joint interorgan crosstalk in knee OA, featuring the role of complement factor D secreted by fat in knee pain. Link here: bit.ly/42Og5hJ
Thanks to @farsh-g.bsky.social for all of your support - @thehopefulscie1.bsky.social and all coauthors!
Annemarie’s latest paper is out today!
CYR61 delivery promotes angiogenesis during bone fracture repair.
www.nature.com/articles/s41...
@amilangscience.bsky.social @madhura0327.bsky.social @cpane94.bsky.social @eseidlscience.bsky.social @ricgottardi.bsky.social et al.
Enzyme Porcupine inhibition of Wnt signaling-a promising treatment forsclerosteosis - rare Dz caused by SOST mutations & loss of functional sclerostin. Results in high bone mass & skeletal overgrowth (Sxs of facial paralysis, hearing loss, incr intracranial pressure www.nature.com/articles/s41...
This study was published in Bone Research, the team investigates the impact of LGK974, a Porcupine inhibitor, on bone mass regulation in a sclerosteosis mouse model.
#Bone #Sclerosteosis
Details: doi.org/10.1038/s414...
Porcupine inhibition, targeting the Wnt signaling pathway, shows promise as a treatment for sclerosteosis, potentially reducing the need for high-risk surgeries in this rare genetic disorder. doi.org/g9fm67
💡A huge shout out to all of the authors of this study Tim Dreyer, Jacob Keen, Leah Wells, Mark Hopkinson, Isabel Orriss, Gill Holdsworth and Andy Pitsillides - you are all great! Also many thanks to our funders UCB Pharma for your continued support.
📋 Importance: LGK974 therapy would likely be an option for treating disease early in children. This would potentially avoid risky surgical treatments such as skull decompression surgery.
💊 What We Found: A drug called LGK974 reduced skull and ear bone mass of young mice with sclerosteosis disease, indicating that it effectively limits skeletal overgrowth at sites with severe/fatal pathology.
🔬 What We Did: We screened distinct drugs in cells, and a mouse model of sclerosteosis. This is a disease characterised by skeletal overgrowth, potentially fatal elevated intracranial pressure, and deafness. Sclerosteosis has no pharmaceutical therapy, and is managed only through high-risk surgery.