PCD Research reposted this
Chair and Founder PCDResearch.org, Rare disease advocate, Drug Development Clinician, former paediatric oncology doctor. PhD - functional dependencies of osteosarcoma.
Ending the end of PCD awareness month in the same style we kicked off with our #GettingCiliaMoving Industry Accelerator Day, I’m excited to announce that PCD Research and NATA - Nucleic Acid Therapy Accelerator have agreed to jointly award Prof Stephen Hart at UCL a £250k two-year research grant to develop mRNA therapy for PCD. This grant targets the third most common disease causing gene CCDC39, known for causing severe clinical outcomes. The project aims to restore the movement of cilia (tiny hair-like structures in the lungs) by delivering mRNA to replace the faulty CCDC39 protein. If successful, this method could be a significant advance in creating more effective, targeted treatments for PCD across many disease causing genes. This is a first not just for PCD Research but also the first research towards developing a treatment to restore ciliary function that has been funded by a charity. #raredisease #rarediseases #fundingcollaboration #medicalresearch #mrna #mrnatreatments #GettingCiliaMoving #primaryciliarydyskinesia #pcdawareness #geneticTherapies #geneticdisease #ciliopathies #raredisease #rarediseases #pcdawarenessmonth #pcdwarriors #pcdwarrior #kartageners #kartagenersSyndrome Medical Research Council