Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.
Mol Ther
; 31(3): 657-675, 2023 03 01.
Article
em En
| MEDLINE
| ID: mdl-36457248
ABSTRACT
Lysosomal storage diseases (LSDs) are multisystem inherited metabolic disorders caused by dysfunctional lysosomal activity, resulting in the accumulation of undegraded macromolecules in a variety of organs/tissues, including the central nervous system (CNS). Treatments include enzyme replacement therapy, stem/progenitor cell transplantation, and in vivo gene therapy. However, these treatments are not fully effective in treating the CNS as neither enzymes, stem cells, nor viral vectors efficiently cross the blood-brain barrier. Here, we review the latest advancements in improving delivery of different therapeutic agents to the CNS and comment upon outstanding questions in the field of neurological LSDs.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Barreira Hematoencefálica
/
Doenças por Armazenamento dos Lisossomos
Limite:
Humans
Idioma:
En
Ano de publicação:
2023
Tipo de documento:
Article