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

Targeted BDNF upregulation via upstream open reading frame disruption To understand the relative contributions of 5ʹ UTR elements to translation output, we performed a comprehensive analysis of upstream open reading fram…

Latest research from my group. We activated BDNF protein expression by disrupting a repressive uORF using a CRISPR base editing strategy: www.sciencedirect.com/science/arti...

#OxfordPaediatrics #IDRMOxford #BDNF #uORF #CRISPR #BaseEditing

AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects - Gene Therapy Gene Therapy - AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects

Latest review article from my group published in Gene Therapy:AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects www.nature.com/articles/s41... #OxfordPaediatrics #IDRMOxford #DMD #Duchenne #Microdystrophin #Genetherapy #AAV