Search Results - "Wood, J A"

Advances in oligonucleotide drug delivery by Roberts, Thomas C., Langer, Robert, Wood, Matthew J. A.

“…Oligonucleotides can be used to modulate gene expression via a range of processes including RNAi, target degradation by RNase H-mediated cleavage, splicing…”
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Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes by Wood, Matthew J A, Alvarez-Erviti, Lydia, Seow, Yiqi, Yin, HaiFang, Betts, Corinne, Lakhal, Samira

“…To realize the therapeutic potential of RNA drugs, efficient, tissue-specific and nonimmunogenic delivery technologies must be developed. Here we show that…”
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DNA Cage Delivery to Mammalian Cells by Walsh, Anthony S, Yin, HaiFang, Erben, Christoph M, Wood, Matthew J. A, Turberfield, Andrew J

“…DNA cages are nanometer-scale polyhedral structures formed by self-assembly from synthetic DNA oligonucleotides. Potential applications include in vivo imaging…”
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The role of long non-coding RNAs in neurodevelopment, brain function and neurological disease by Roberts, Thomas C., Morris, Kevin V., Wood, Matthew J. A.

“…Long non-coding RNAs (lncRNAs) are transcripts with low protein-coding potential that represent a large proportion of the transcriptional output of the cell…”
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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study by Cirak, Sebahattin, MD, Arechavala-Gomeza, Virginia, PhD, Guglieri, Michela, MD, Feng, Lucy, PhD, Torelli, Silvia, PhD, Anthony, Karen, PhD, Abbs, Stephen, PhD, Garralda, Maria Elena, Prof, Bourke, John, MD, Wells, Dominic J, Prof, Dickson, George, Prof, Wood, Matthew JA, MD PhD, Wilton, Steve D, Prof, Straub, Volker, Prof, Kole, Ryszard, Prof, Shrewsbury, Stephen B, MD, Sewry, Caroline, Prof, Morgan, Jennifer E, PhD, Bushby, Kate, Prof, Muntoni, Francesco, Prof

“…Summary Background We report clinical safety and biochemical efficacy from a dose-ranging study of intravenously administered AVI-4658 phosphorodiamidate…”
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Alpha-synuclein induces the unfolded protein response in Parkinson's disease SNCA triplication iPSC-derived neurons by Heman-Ackah, Sabrina M, Manzano, Raquel, Hoozemans, Jeroen J M, Scheper, Wiep, Flynn, Rowan, Haerty, Wilfried, Cowley, Sally A, Bassett, Andrew R, Wood, Matthew J A

“…The recent generation of induced pluripotent stem cells (iPSCs) from a patient with Parkinson's disease (PD) resulting from triplication of the α-synuclein…”
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Assessment of RT-qPCR normalization strategies for accurate quantification of extracellular microRNAs in murine serum by Roberts, Thomas C, Coenen-Stass, Anna M L, Wood, Matthew J A

“…Extracellular microRNAs (miRNAs) are under investigation as minimally-invasive biomarkers for a wide range of disease conditions. We have recently shown in a…”
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Targeting RNA to treat neuromuscular disease by Muntoni, Francesco, Wood, Matthew J. A

“…Key Points Neuromuscular disorders comprise a heterogeneous group of clinical conditions that primarily affect one or more components of the neuromuscular unit…”
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A CRISPR-Cas9-based reporter system for single-cell detection of extracellular vesicle-mediated functional transfer of RNA by de Jong, Olivier G., Murphy, Daniel E., Mäger, Imre, Willms, Eduard, Garcia-Guerra, Antonio, Gitz-Francois, Jerney J., Lefferts, Juliet, Gupta, Dhanu, Steenbeek, Sander C., van Rheenen, Jacco, El Andaloussi, Samir, Schiffelers, Raymond M., Wood, Matthew J. A., Vader, Pieter

“…Extracellular vesicles (EVs) form an endogenous transport system for intercellular transfer of biological cargo, including RNA, that plays a pivotal role in…”
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GAPDH controls extracellular vesicle biogenesis and enhances the therapeutic potential of EV mediated siRNA delivery to the brain by Dar, Ghulam Hassan, Mendes, Cláudia C., Kuan, Wei-Li, Speciale, Alfina A., Conceição, Mariana, Görgens, André, Uliyakina, Inna, Lobo, Miguel J., Lim, Wooi F., EL Andaloussi, Samir, Mäger, Imre, Roberts, Thomas C., Barker, Roger A., Goberdhan, Deborah C. I., Wilson, Clive, Wood, Matthew J. A.

“…Extracellular vesicles (EVs) are biological nanoparticles with important roles in intercellular communication, and potential as drug delivery vehicles. Here we…”
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Extracellular Vesicle Heterogeneity: Subpopulations, Isolation Techniques, and Diverse Functions in Cancer Progression by Willms, Eduard, Cabañas, Carlos, Mäger, Imre, Wood, Matthew J A, Vader, Pieter

“…Cells release membrane enclosed nano-sized vesicles termed extracellular vesicles (EVs) that function as mediators of intercellular communication by…”
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Extracellular vesicle in vivo biodistribution is determined by cell source, route of administration and targeting by Wiklander, Oscar P. B., Nordin, Joel Z., O'Loughlin, Aisling, Gustafsson, Ylva, Corso, Giulia, Mäger, Imre, Vader, Pieter, Lee, Yi, Sork, Helena, Seow, Yiqi, Heldring, Nina, Alvarez-Erviti, Lydia, Smith, CI Edvard, Le Blanc, Katarina, Macchiarini, Paolo, Jungebluth, Philipp, Wood, Matthew J. A., Andaloussi, Samir EL

“…Extracellular vesicles (EVs) have emerged as important mediators of intercellular communication in a diverse range of biological processes. For future…”
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An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides by Baker, Ysobel R., Thorpe, Cameron, Chen, Jinfeng, Poller, Laura M., Cox, Lina, Kumar, Pawan, Lim, Wooi F., Lie, Lillian, McClorey, Graham, Epple, Sven, Singleton, Daniel, McDonough, Michael A., Hardwick, Jack S., Christensen, Kirsten E., Wood, Matthew J. A., Hall, James P., El-Sagheer, Afaf H., Brown, Tom

“…Oligonucleotides that target mRNA have great promise as therapeutic agents for life-threatening conditions but suffer from poor bioavailability, hence high…”
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A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice by Yin, HaiFang, Moulton, Hong M., Betts, Corinne, Seow, Yiqi, Boutilier, Jordan, Iverson, Patrick L., Wood, Matthew J.A.

“…Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that abolish the synthesis of dystrophin protein. Antisense oligonucleotides (AOs)…”
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Genetic therapies for RNA mis-splicing diseases by Hammond, Suzan M, Wood, Matthew J.A

“…RNA mis-splicing diseases account for up to 15% of all inherited diseases, ranging from neurological to myogenic and metabolic disorders. With greatly…”
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FKRP-dependent glycosylation of fibronectin regulates muscle pathology in muscular dystrophy by Wood, A. J., Lin, C. H., Li, M., Nishtala, K., Alaei, S., Rossello, F., Sonntag, C., Hersey, L., Miles, L. B., Krisp, C., Dudczig, S., Fulcher, A. J., Gibertini, S., Conroy, P. J., Siegel, A., Mora, M., Jusuf, P., Packer, N. H., Currie, P. D.

“…The muscular dystrophies encompass a broad range of pathologies with varied clinical outcomes. In the case of patients carrying defects in fukutin-related…”
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Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function by Yin, HaiFang, Moulton, Hong M., Seow, Yiqi, Boyd, Corinne, Boutilier, Jordan, Iverson, Patrick, Wood, Matthew J.A.

“…Antisense oligonucleotides (AOs) have the potential to induce functional dystrophin protein expression via exon skipping by restoring in-frame transcripts in…”
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Exosomes and microvesicles: extracellular vesicles for genetic information transfer and gene therapy by LEE, Yi, ANDALOUSSI, Samir El, WOOD, Matthew J. A

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Molecular and electrophysiological features of spinocerebellar ataxia type seven in induced pluripotent stem cells by Burman, Richard J, Watson, Lauren M, Smith, Danielle C, Raimondo, Joseph V, Ballo, Robea, Scholefield, Janine, Cowley, Sally A, Wood, Matthew J A, Kidson, Susan H, Greenberg, Leslie J

“…Spinocerebellar ataxia type 7 (SCA7) is an inherited neurodegenerative disease caused by a polyglutamine repeat expansion in the ATXN7 gene. Patients with this…”
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RNA-targeted splice-correction therapy for neuromuscular disease by Wood, Matthew J. A., Gait, Michael J., Yin, Haifang

“…Splice-modulation therapy, whereby molecular manipulation of premessenger RNA splicing is engineered to yield genetic correction, is a promising novel therapy…”
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