Exon-skipping offers a highly targeted and transformative approach to treating Duchenne muscular dystrophy (#DMD). These therapies work by restoring the reading frame of the DMD gene, enabling #Dystrophin synthesis.
Read here: https://bit.ly/4vjwaJK
#RareDisease #MedSky
New research from my group using a novel mouse model to better understand spatial dystrophin localisation in dystrophic muscle.
onlinelibrary.wiley.com/doi/10.1002/...
#OxfordPaediatrics #IDRMOxford #DMD #MuscularDystrophy #MyonuclearDomains #dystrophin
Tevard Biosciences to Present Data Demonstrating tRNA-based Therapy Rescued Full-Length Dystrophin and Motor Function in Duchenne Muscular Dystrophy Model Tevard Biosciences' tRNA-based therapy promises full-length dystrophin for Duchenne Muscular Dystrophy patients. Highlights: 📌Preclinical results from studies using the D2-mdx mouse model, which contains a nonsense mutation in the DMD gene and recapitulates key aspects of DMD pathology in humans 📌Muscles of treated animals expressed full-length dystrophin protein in a dose-dependent manner at 6 weeks post-dosing 📌Rescued protein is organized in a fashion similar to wild-type dystrophin protein at 6 weeks 📌At 12 weeks post-dosing, there was a significant restoration of motor function as demonstrated by an increase in latency time in the rotarod performance test and significantly increased forelimb and hindlimb grip strength 📌There was no evidence of adverse treatment effects as measured by behavioral or histologic changes in major organs or in blood chemistry 👉 DMDWarrioR.com
Tevard Biosciences to Present Data Demonstrating tRNA-based Therapy Rescued Full-Length Dystrophin and Motor Function in Duchenne Muscular Dystrophy Model
👉 DMDWarrioR.com
#dmd #duchenne #tRNA #Dystrophin
Structural details of the closed conformation adopted by the dystrophin ABD1 monomer in solution are provided, confirming that the domain-wapped closed state seen for dystrophin ABD1 dimers also occurs in the monomer #ActinBinding #Dystrophin doi.org/10.1107/S205...
New Research on Duchenne Muscular Dystrophy Protein Interactions Lead to More Targeted Therapies Study results explain how dystrophin works molecularly, revealing how muscular dystrophy medicines can be developed. A pioneering discovery has shown the complicated relationships between dystrophin, a muscle stability protein, and its companion protein, dystrobrevin, opening new avenues for studying and treating Duchenne Muscular Dystrophy. Researchers describe dystrophin cryptic C-terminal (CT) region and its role in maintaining cellular membranes across tissues in the December Journal of Biological Chemistry. “This research highlights the intricate dynamics of dystrophin and dystrobrevin interactions, providing critical insights that could inform future treatment development,” said study lead author Krishna Mallela, University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences professor. Our understanding of how these proteins work in diverse tissues is one step closer to finding DMD treatments that target the root causes.
New Research on Duchenne Muscular Dystrophy Protein Interactions Lead to More Targeted Therapies
Read More: dmdwarrior.com/new-research...
#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchenne #musculardystrophy #gene #dna #dystrophin #dystrobrevin
