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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Bibliographic Details
Main Author: Coutinho, Maria Francisca (author)
Other Authors: 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)
Format: conferenceObject
Language:eng
Published: 2021
Subjects:
Lysosomal Storage Diseases
RNA Therapies
Mucopolysaccharidosis type III
SNALPs
Doenças Genéticas
Online Access:http://hdl.handle.net/10400.18/7439
Country:Portugal
Oai:oai:repositorio.insa.pt:10400.18/7439

Internet

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

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