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.
Upper and lower motor neuron degenerations are somatotopically related and temporally ordered in the SOD1 mouse model of amyotrophic lateral sclerosis
Marques C. et al, (2021), Brain Sciences, 11
Evidence that corticofugal propagation of ALS pathology is not mediated by prion-like mechanism.
Scekic-Zahirovic J. et al, (2020), Prog Neurobiol
Absence of subcerebral projection neurons is beneficial in a mouse model of ALS.
Burg T. et al, (2020), Ann Neurol