Matthew J. A. Wood

This study evaluates cell-specific activity of antisense oligonucleotide conjugates in vivo using a reporter mouse model and finds that palmitate conjugation shows broad extrahepatic activity and preferential skeletal muscle targeting.

This study identifies how the polyglutamine tract in the androgen receptor regulates muscle growth and atrophy by altering transcriptional activity and chromatin binding.

This study shows that exon skipping therapy is more effective when started early in adult mdx mice than in aged mice, leading to better dystrophin restoration and transcriptomic changes.

The paper develops ASO Atlas, a database of RNase H-mediated ASO sequences and their efficacy, and trains OligoAI, a deep learning model to predict ASO efficacy based on sequence and chemical features, reducing screening effort by 5.72-fold.

This study develops a click chemistry-based strategy for siRNA conjugation to enhance extrahepatic RNAi, finding that bis-fatty-acid conjugates improve activity in heart and muscle while showing modality-specific limitations.

This study shows that systemic inflammation significantly prolongs EV plasma half-life and enhances their accumulation in inflamed organs like the liver and spleen.

This study identifies uORF elements regulating BDNF translation and demonstrates that disrupting uORFs can upregulate BDNF expression in cell culture.

This paper develops a method to isolate and characterize SMAPs from NK-92 cells, showing their cytotoxic activity against tumor cells in vitro and in vivo.

The paper identifies ThrEDs, a novel type of extracellular particle that accelerates wound healing and reduces fibrotic responses in patient-derived myofibroblasts.

The paper combines experimental and computational methods to model how uORFs regulate translation, identifying key features and interactions that influence their repressive activity.

This paper introduces a novel DMD mouse model with non-uniform dystrophin expression and shows that partial dystrophin expression protects against muscle pathology.

This study shows MEG3 is upregulated in DM1 and exhibits nuclear retention, with antisense conjugates correcting MEG3 levels in mice.

This study shows TSP-4 is required for TSP-1 association with lytic granules and SMAP-mediated cytotoxicity in CTLs, and its expression is suppressed in CLL cell supernatants.

This paper introduces CRISPR MiRAGE, a miRNA-sensing guide RNA system that enables tissue-specific gene editing by modulating CRISPR activity based on miRNA signatures.

The paper tests uORF-targeting ASOs for RNASEH1 upregulation but finds no reproducible activation, instead observing downregulation in some cases.

This paper develops a PTTG1IP-based platform for efficient extracellular vesicle (EV) cargo delivery, showing improved therapeutic delivery compared to CD63-based scaffolds.

This study characterizes extracellular vesicles from activated CD4 T cells and shows their pro-inflammatory effects on antigen-presenting cells, providing a resource for EV-based therapeutic development.

This study shows that Fn14-depletion improves survival and muscle pathology in SOD1G93A ALS mice, but only when combined with exercise.

This paper develops CRISPR-Lock, a CRISPR/dCas13 RNA blocker that disrupts miRNA-target interactions and rescues DM1 pathology by correcting splicing biomarkers and reducing nuclear foci.

This study uses CRISPR to excise exon 23 in dKO mice, achieving partial dystrophin restoration but with limited efficacy and challenges in DNA repair.

This study shows that PPMO-mediated exon skipping in mdx mice restores uniform dystrophin expression and dose-dependently improves serum microRNA biomarkers and muscle biophysical properties.

This study shows GAPDH facilitates EV biogenesis and enables efficient siRNA delivery to the brain, reducing huntingtin gene expression in a mouse model.

This study demonstrates that systemic delivery of ASO conjugated to a brain-targeting antibody improves SMN2 splicing and survival in SMA mouse models.

The paper shows that most extracellular miRNAs are secreted non-vesicularly, with only <2% associated with EVs, even though EVs can stabilize miRNAs in serum.

This study generates iPSCs from SCA7 patients and shows molecular and electrophysiological abnormalities in derived neurons and photoreceptors.

This study engineers extracellular vesicles with IL6ST decoy receptors to inhibit the IL6 trans-signalling pathway, showing efficacy in vitro and in a Duchenne muscular dystrophy mouse model.

This study identifies novel drug treatments for spinal muscular atrophy by integrating multi-omics data and drug perturbation profiles, showing harmine improves disease phenotypes in models.