Mathieu Fischer
Research Assistant
Now I take part in a preclinical project utilizing peptide delivered oligonucleotides for the treatment of Myotonic Dystrophy type 1 (DM1) under the supervision of Dr Miguel Varela-Muino. I am responsible for assisting in the day-to-day execution of the project and take care of the animal experiments.
The research project will evaluate antisense oligonucleotides for the therapy of DM1, the most common form of muscular dystrophy in adults and a genetic neuromuscular disease characterized by progressive muscle degeneration. There is currently no disease-modifying therapy for DM1. Our therapeutic solution is an advanced peptide-conjugated antisense oligonucleotide that permits highly effective systemic delivery to all affected tissues. Our project aims to identify and optimize a lead compound suitable for clinical development in the future.
Recent publications
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Upper and lower motor neuron degenerations are somatotopically related and temporally ordered in the SOD1 mouse model of amyotrophic lateral sclerosis
Journal article
Marques C. et al, (2021), Brain Sciences, 11
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Evidence that corticofugal propagation of ALS pathology is not mediated by prion-like mechanism.
Journal article
Scekic-Zahirovic J. et al, (2020), Prog Neurobiol
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Absence of subcerebral projection neurons is beneficial in a mouse model of ALS.
Journal article
Burg T. et al, (2020), Ann Neurol
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Genetic ablation of SOD1G37R selectively from corticofugal projection neurons protects corticospinal neurons from degeneration without affecting ALS onset and progression
Journal article
Scekic-Zahirovic J. et al, (2020)
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Absence of subcerebral projection neurons delays disease onset and extends survival in a mouse model of ALS
Journal article
Burg T. et al, (2019)