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

IDRM Researchers Raise Over £1,300 for Muscular Dystrophy UK at Oxford Town & Gown 10k Race On Sunday 11 May, researchers from the MDUK Oxford Neuromuscular Centre, based at the Institute of Developmental & Regenerative Medicine (IDRM), joined more than 6,000 participants in the historic

Article on our recent fundraising activities
www.idrm.ox.ac.uk/news-events/...

Orfonyx Bio – big ambitions in protein upregulation Orfonyx Bio is looking to make its mark as a leader in protein upregulation for clinical benefit – and has made technical progress towards this goal over the past two years. In that time as a BioEscal...

Some press regarding Orfonyx Bio - a spin-out company that I founded a few years ago #OrfonyxBio #oligonucleotides

uORF-targeting steric block antisense oligonucleotides do not reproducibly increase RNASEH1 expression Roberts and colleagues report a reproduction study using steric block oligonucleotides targeting an upstream open reading frame (uORF) in the RNASEH1 gene. uORFs are typically negative regulators of t...

www.cell.com/molecular-th...

Recent work from my group concerning efforts to target upstream open reading frames (uORFs) using molecular medicine approaches. (More to come soon!)

📢 Huge News! 📢

The MRC has launched two centres of research excellence to transform gene therapies: IDRM’s Paul Riley will co-direct the MRC/BHF CoRE in Advanced Cardiac Therapies & Stephan Sanders will direct the MRC CoRE in Therapeutic Genomics.
Read more: lnkd.in/g-3CmAsP
#GeneTherapies

An extracellular vesicle delivery platform based on the PTTG1IP protein Extracellular vesicles (EVs) are promising therapeutic delivery vehicles, although their potential is limited by a lack of efficient engineering strat…

Latest research from my group. An N-glycosylated protein, PTTG1IP, can be utilised to load extracellular vesicles with therapeutic cargo molecules.
www.sciencedirect.com/science/arti...

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by mutations in the DMD gene that prevent the expression of a functional dy…

Here is original the paper from Pro Kevin Flanigan's lab: www.sciencedirect.com/science/arti...

Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications

A brief commentary that I wrote for Methods & Clinical Development on exon skipping therapy for DMD patients with exon 2 duplications (from the Flanigan lab) www.sciencedirect.com/science/arti...

Tom Roberts: Functional consequences of EV dose and co-isolates YouTube video by EVClub: Extracellular Vesicle Club

A talk that I gave for the EVClub on recent work from my group on the subject of extracellular vesicles and their role in the regulation of myogenic differentiation. www.youtube.com/watch?v=CMxr...

Therapeutic approaches for Duchenne muscular dystrophy - Nature Reviews Drug Discovery Duchenne muscular dystrophy is an inherited muscle-wasting disease caused by mutations that disrupt production of dystrophin protein. This Review discusses the plethora of therapeutic approaches being...

A recent review that I wrote for readers interested in the pipeline of therapeutics for Duchenne muscular dystrophy, here's a comprehensive review that has just been published bit.ly/3r48QU2

EV-mediated promotion of myogenic differentiation is dependent on dose, collection medium, and isolation method Extracellular vesicles (EVs) have been reported to promote myogenic differentiation. Here, we show confirm this finding in mouse myoblasts with a number of technical caveats. Opposing phenotypic outco...

Recent work from my group on the role of extracellular vesicles in myogenic differentiation www.cell.com/molecular-th...

Hi BlueSky! I'm going to move some of my old tweets over here #Xodus