Genetic Substrate Reduction Therapy for Mucopolysaccharidoses type III: toward a siRNA-containing nanoparticle targeted to brain cells

The classical therapeutic approach for LSDs, enzyme replacement therapy, would hardly rise as a potentially successful tool to reduce the disease burden in MPS III patients, as it is long known to have no impact on neuropathology. A tempting alternative, however, would be to block substrate accumula...

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Bibliographic Details
Main Author: Coutinho, Maria Francisca (author)
Other Authors: Santos, Juliana Inês (author), Gaspar, Paulo (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
MPS III
Substrate Reduction Therapy
Doenças Lisossomais de Sobrecarga
Doenças Genéticas
Online Access:http://hdl.handle.net/10400.18/7440
Country:Portugal
Oai:oai:repositorio.insa.pt:10400.18/7440

Internet

http://hdl.handle.net/10400.18/7440

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