RNA Medicine Group (Oxford)
@ribomedicine.bsky.social
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.
https://www.idrm.ox.ac.uk/research/neurology
https://www.idrm.ox.ac.uk/research/neurology
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
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
www.nature.com
August 15, 2025 at 4:08 PM
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
Article on our recent fundraising activities
www.idrm.ox.ac.uk/news-events/...
www.idrm.ox.ac.uk/news-events/...
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
www.idrm.ox.ac.uk
May 27, 2025 at 2:42 PM
Article on our recent fundraising activities
www.idrm.ox.ac.uk/news-events/...
www.idrm.ox.ac.uk/news-events/...
Some press regarding Orfonyx Bio - a spin-out company that I founded a few years ago #OrfonyxBio #oligonucleotides
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...
www.bioescalator.ox.ac.uk
April 9, 2025 at 2:49 PM
Some press regarding Orfonyx Bio - a spin-out company that I founded a few years ago #OrfonyxBio #oligonucleotides
Recent work from my group concerning efforts to target upstream open reading frames (uORFs) using molecular medicine approaches. (More to come soon!)
Congratulations to the Roberts Group on their recent study, published in Molecular Therapy: Nucleic Acids, challenging the therapeutic potential of a mechanism previously thought to upregulate protein expression in specific genes.
@ribomedicine.bsky.social @oxfordpaediatrics.bsky.social
@ribomedicine.bsky.social @oxfordpaediatrics.bsky.social
A new study challenges a potential gene-activating mechanism using antisense oligonucleotides
A new study from the Roberts Group, Ahlskog et al., published in Molecular Therapy: Nucleic Acids, challenges the therapeutic potential of a mechanism previously thought to upregulate protein
www.idrm.ox.ac.uk
February 14, 2025 at 12:46 PM
Recent work from my group concerning efforts to target upstream open reading frames (uORFs) using molecular medicine approaches. (More to come soon!)
Reposted by RNA Medicine Group (Oxford)
📢 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
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
December 11, 2024 at 10:40 AM
📢 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
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
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...
www.sciencedirect.com/science/arti...
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…
www.sciencedirect.com
November 19, 2024 at 5:36 PM
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...
www.sciencedirect.com/science/arti...
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...
Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications
www.sciencedirect.com
November 19, 2024 at 5:34 PM
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...
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...
Tom Roberts: Functional consequences of EV dose and co-isolates
YouTube video by EVClub: Extracellular Vesicle Club
www.youtube.com
November 19, 2024 at 5:33 PM
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...
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
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...
bit.ly
November 19, 2024 at 5:31 PM
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
Recent work from my group on the role of extracellular vesicles in myogenic differentiation www.cell.com/molecular-th...
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...
www.cell.com
November 19, 2024 at 5:30 PM
Recent work from my group on the role of extracellular vesicles in myogenic differentiation www.cell.com/molecular-th...