2 documents found, page 1 of 1
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clear...
Lopes, Miguel M.; Paysan, Jacques; Rino, José; Lopes, Sara M.; Pereira de Almeida, Luís; Cortes, Luísa; Nobre, Rui JorgeRecombinant adeno-associated virus (rAAV) has become one of the most promising gene delivery systems for both in vitro and in vivo applications. However, a key challenge is the lack of suitable imaging technologies to evaluate delivery, biodistribution and tropism of rAAVs and efficiently monitor disease amelioration promoted by AAV-based therapies at a whole-organ level with single-cell resolution. Therefore, ...
ULK overexpression mitigates motor deficits and neuropathology in mouse models ...
Vasconcelos-Ferreira, Ana; Martins, Inês Morgado; Lobo, Diana; Pereira, Dina; Lopes, Miguel M.; Faro, Rosário; Lopes, Sara M.; Verbeek, DinekeMachado-Joseph disease (MJD) is a fatal neurodegenerative disorder clinically characterized by prominent ataxia. It is caused by an expansion of a CAG trinucleotide in ATXN3, translating into an expanded polyglutamine (polyQ) tract in the ATXN3 protein, that becomes prone to misfolding and aggregation. The pathogenesis of the disease has been associated with the dysfunction of several cellular mechanisms, inclu...