Sign in

RNA Medicine Group (Oxford)

@ribomedicine.bsky.social
256 followers 197 following 16 posts

Research team of Tom Roberts at the University of Oxford, Department of Paediatrics, and Institute of Developmental and Regenerative Medicine. Our work focuses on RNA-based medicine and neuromuscular disorders. www.idrm.ox.ac.uk/research/neurology

PostsRepliesMedia
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 03/09/2026
New research on the challenges of targeting uORFs for TTN up-regulation: Functional analysis of TTN uORFs reveals context-dependent translational regulation. A great collaboration with Prof Hugh Watkins' team #OxfordPaediatrics #IDRMOxford #TTN #uORF #CRISPR #cardiomyopathy doi.org/10.1016/j.om...
010
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 03/08/2026
New research from my group: treatment timing with peptide-conjugated exon skipping drugs makes a big difference to therapeutic effects in a DMD mouse model. Starting treatment early is more effective. #OxfordPaediatrics #IDRMOxford #DMD #MuscularDystrophy #ExonSkipping #dystrophin
010
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 07/04/2026
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
020
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 06/01/2026
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
sciencedirect.com
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…
094
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 15/08/2025
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
nature.com
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
020
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 27/05/2025
Article on our recent fundraising activities www.idrm.ox.ac.uk/news-events/...
idrm.ox.ac.uk
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
010
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 09/04/2025
Some press regarding Orfonyx Bio - a spin-out company that I founded a few years ago #OrfonyxBio #oligonucleotides
bioescalator.ox.ac.uk
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...
011
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 14/02/2025
Recent work from my group concerning efforts to target upstream open reading frames (uORFs) using molecular medicine approaches. (More to come soon!)
130
Reposted by RNA Medicine Group (Oxford)
Institute of Developmental & Regenerative Medicine @idrm.ox.ac.uk · 11/12/2024
📢 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
0115
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 19/11/2024
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...
sciencedirect.com
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…
021
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 19/11/2024
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...
sciencedirect.com
Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications
100
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 19/11/2024
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...
youtube.com
Tom Roberts: Functional consequences of EV dose and co-isolates
YouTube video by EVClub: Extracellular Vesicle Club
000
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 19/11/2024
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
bit.ly
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...
000
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 19/11/2024
Recent work from my group on the role of extracellular vesicles in myogenic differentiation www.cell.com/molecular-th...
cell.com
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...
010
RNA Medicine Group (Oxford) @ribomedicine.bsky.social · 19/11/2024
Hi BlueSky! I'm going to move some of my old tweets over here #Xodus
020