Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid Lipid Particle (SNALP)-Formulated siRNAs to the Brain as a Therapeutic Approach: The Sanfilippo Syndrome example

More than two thirds of Lysosomal Storage Diseases (LSDs) present central nervous system (CNS) involvement. Nevertheless, only one of the currently approved therapies has an impact on neuropathology. Therefore, alternative approaches are under development, either addressing the underlying enzymatic...

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Detalhes bibliográficos
Autor principal: Coutinho, Maria Francisca (author)
Outros Autores: Santos, Juliana Inês (author), Mendonça, Liliana Silva (author), Matos, Liliana (author), Prata, Maria João (author), Jurado, Amália Silva (author), Pedroso de Lima, Maria da Conceição (author), Alves, Sandra (author)
Formato: conferenceObject
Idioma:eng
Publicado em: 2021
Assuntos:
Lysosomal Storage Diseases
RNA Therapies
Mucopolysaccharidosis type III
SNALPs
Doenças Genéticas
Texto completo:http://hdl.handle.net/10400.18/7439
País:Portugal
Oai:oai:repositorio.insa.pt:10400.18/7439

Texto Completo

http://hdl.handle.net/10400.18/7439

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