Fraldi and colleagues demonstrated that multiple amyloid proteins progressively aggregate in neurons of a severe lysosomal storage disease, impairing autophagy degradation and triggering neurodegeneration. They also showed that inhibiting amyloid deposition protects against neurodegeneration, thus providing evidence that amyloid aggregation is a new attractive target for the treatment of LSDs.

The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease / Monaco, A.; Maffia, V.; Sorrentino, N. C.; Sambri, I.; Ezhova, Y.; Giuliano, T.; Cacace, V.; Nusco, E.; De Risi, M.; De Leonibus, E.; Schrader, T.; Klarner, F. -G.; Bitan, G.; Fraldi, A.. - In: MOLECULAR THERAPY. - ISSN 1525-0016. - (2020). [10.1016/j.ymthe.2020.02.005]

The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease

Sorrentino N. C.;Fraldi A.
2020

Abstract

Fraldi and colleagues demonstrated that multiple amyloid proteins progressively aggregate in neurons of a severe lysosomal storage disease, impairing autophagy degradation and triggering neurodegeneration. They also showed that inhibiting amyloid deposition protects against neurodegeneration, thus providing evidence that amyloid aggregation is a new attractive target for the treatment of LSDs.
2020
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease / Monaco, A.; Maffia, V.; Sorrentino, N. C.; Sambri, I.; Ezhova, Y.; Giuliano, T.; Cacace, V.; Nusco, E.; De Risi, M.; De Leonibus, E.; Schrader, T.; Klarner, F. -G.; Bitan, G.; Fraldi, A.. - In: MOLECULAR THERAPY. - ISSN 1525-0016. - (2020). [10.1016/j.ymthe.2020.02.005]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/795949
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