Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

Autor: Hammond, SM, Hazell, G, Shabanpoor, F, Saleh, AF, Bowerman, M, Sleigh, JN, Meijboom, KE, Zhou, H, Muntoni, F, Talbot, K, Gait, MJ, Wood, MJA

Publikováno v: Proceedings of the National Academy of Sciences of the United States of America. 113(39)

The development of antisense oligonucleotide therapy is an important advance in the identification of corrective therapy for neuromuscular diseases, such as spinal muscular atrophy (SMA). Because of difficulties of delivering single-stranded oligonuc

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::32ec371bb2bfa3ca8fd8e03e62503f3f
http://ora.ox.ac.uk/objects/uuid:

Age-specific and compartment-dependent changes in mitochondrial homeostasis and cytoplasmic viscosity in mouse peripheral neurons.

Autor: Sleigh JN; Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, UK.; UK Dementia Research Institute, University College London, London, UK., Mattedi F; Department of Basic and Clinical Neurosciences, Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK., Richter S; Department of Basic and Clinical Neurosciences, Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK., Annuario E; Department of Basic and Clinical Neurosciences, Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK., Ng K; Department of Basic and Clinical Neurosciences, Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK., Steinmark IE; Department of Physics, King's College London, London, UK., Ivanova I; Department of Physics, King's College London, London, UK., Darabán IL; Department of Basic and Clinical Neurosciences, Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK., Joshi PP; Department of Basic and Clinical Neurosciences, Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK., Rhymes ER; Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, UK.; UK Dementia Research Institute, University College London, London, UK., Awale S; Department of Physics, King's College London, London, UK., Yahioglu G; Antikor Biopharma Ltd, Stevenage Bioscience Catalyst, Stevenage, UK., Mitchell JC; Department of Basic and Clinical Neurosciences, Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK., Suhling K; Department of Physics, King's College London, London, UK., Schiavo G; Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, UK.; UK Dementia Research Institute, University College London, London, UK., Vagnoni A; Department of Basic and Clinical Neurosciences, Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK.; MIA-Portugal, Multidisciplinary Institute of Ageing, University of Coimbra, Coimbra, Portugal.

Publikováno v: Aging cell [Aging Cell] 2024 Oct; Vol. 23 (10), pp. e14250. Date of Electronic Publication: 2024 Jun 17.

The tyrosine phosphatases LAR and PTPRδ act as receptors of the nidogen-tetanus toxin complex.

Autor: Surana S; Department of Neuromuscular Diseases, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK. s.surana@ucl.ac.uk.; UCL Queen Square Motor Neuron Disease Centre, University College London, London, WC1N 3BG, UK. s.surana@ucl.ac.uk.; UK Dementia Research Institute, University College London, London, WC1E 6BT, UK. s.surana@ucl.ac.uk., Villarroel-Campos D; Department of Neuromuscular Diseases, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.; UCL Queen Square Motor Neuron Disease Centre, University College London, London, WC1N 3BG, UK.; UK Dementia Research Institute, University College London, London, WC1E 6BT, UK., Rhymes ER; Department of Neuromuscular Diseases, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.; UCL Queen Square Motor Neuron Disease Centre, University College London, London, WC1N 3BG, UK., Kalyukina M; Department of Clinical and Experimental Epilepsy, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK., Panzi C; Department of Neuromuscular Diseases, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.; UCL Queen Square Motor Neuron Disease Centre, University College London, London, WC1N 3BG, UK.; UK Dementia Research Institute, University College London, London, WC1E 6BT, UK., Novoselov SS; Department of Neuromuscular Diseases, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.; UCL Queen Square Motor Neuron Disease Centre, University College London, London, WC1N 3BG, UK., Fabris F; Department of Biomedical Sciences, University of Padova, Padova, 35131, Italy., Richter S; Department of Neuromuscular Diseases, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.; Department of Biomedical Sciences, University of Padova, Padova, 35131, Italy., Pirazzini M; Department of Biomedical Sciences, University of Padova, Padova, 35131, Italy., Zanotti G; Department of Biomedical Sciences, University of Padova, Padova, 35131, Italy., Sleigh JN; Department of Neuromuscular Diseases, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.; UCL Queen Square Motor Neuron Disease Centre, University College London, London, WC1N 3BG, UK.; UK Dementia Research Institute, University College London, London, WC1E 6BT, UK., Schiavo G; Department of Neuromuscular Diseases, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK. giampietro.schiavo@ucl.ac.uk.; UCL Queen Square Motor Neuron Disease Centre, University College London, London, WC1N 3BG, UK. giampietro.schiavo@ucl.ac.uk.; UK Dementia Research Institute, University College London, London, WC1E 6BT, UK. giampietro.schiavo@ucl.ac.uk.

Publikováno v: The EMBO journal [EMBO J] 2024 Aug; Vol. 43 (16), pp. 3358-3387. Date of Electronic Publication: 2024 Jul 08.

Intraperitoneal Injection of Neonatal Mice.

Autor: Pocratsky AM; Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, UK., Sleigh JN; Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, UK.; UK Dementia Research Institute, University College London, London, UK.

Publikováno v: Bio-protocol [Bio Protoc] 2023 Sep 20; Vol. 13 (18), pp. e4826. Date of Electronic Publication: 2023 Sep 20 (Print Publication: 2023).

In vivo imaging of axonal transport in peripheral nerves of rodent forelimbs.

Autor: Lang Q; Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre, Queen Square Institute of Neurology, University College London, London WC1N 3BG, U.K., Schiavo G; Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre, Queen Square Institute of Neurology, University College London, London WC1N 3BG, U.K.; UK Dementia Research Institute, University College London, London WC1E 6BT, U.K., Sleigh JN; Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre, Queen Square Institute of Neurology, University College London, London WC1N 3BG, U.K.; UK Dementia Research Institute, University College London, London WC1E 6BT, U.K.

Publikováno v: Neuronal signaling [Neuronal Signal] 2023 Jan 19; Vol. 7 (1), pp. NS20220098. Date of Electronic Publication: 2023 Jan 19 (Print Publication: 2023).