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Decreasing ganglioside synthesis delays motor and cognitive symptom onset in Spg11 knockout mice

Biallelic variants in the SPG11 gene account for the most common form of autosomal recessive hereditary spastic paraplegia characterized by motor and cognitive impairment, with currently no therapeutic option. We previously observed in a Spg11 knockout mouse that neurodegeneration is associated with...

Whakaahuatanga katoa

I tiakina i:
Ngā kaituhi matua: Manon Fortier, Margaux Cauhapé, Suzie Buono, Julien Becker, Alexia Menuet, Julien Branchu, Ivana Ricca, Serena Mero, Karim Dorgham, Khalid-Hamid El Hachimi, Kostantin Dobrenis, Benoit Colsch, Dominic Samaroo, Morgan Devaux, Alexandra Durr, Giovanni Stevanin, Filippo M. Santorelli, Sophie Colombo, Belinda Cowling, Frédéric Darios
Hōputu: Artigo
Reo:Inglês
I whakaputaina: Elsevier 2024-09-01
Rangatū:Neurobiology of Disease
Ngā marau:
Hereditary spastic paraplegia
Motor neuron disease
Neurodegeneration
Therapy
Small molecule
Lysosome
Urunga tuihono:http://www.sciencedirect.com/science/article/pii/S0969996124001645
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