Therapeutic Approaches in Lysosomal Storage Diseases.
Biomolecules
; 11(12)2021 11 26.
Article
em En
| MEDLINE
| ID: mdl-34944420
ABSTRACT
Lysosomal Storage Diseases are multisystemic disorders determined by genetic variants, which affect the proteins involved in lysosomal function and cellular metabolism. Different therapeutic approaches, which are based on the physiologic mechanisms that regulate lysosomal function, have been proposed for these diseases. Currently, enzyme replacement therapy, gene therapy, or small molecules have been approved or are under clinical development to treat lysosomal storage disorders. The present article reviews the main therapeutic strategies that have been proposed so far, highlighting possible limitations and future perspectives.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Biomarcadores
/
Doenças por Armazenamento dos Lisossomos
Limite:
Humans
Idioma:
En
Revista:
Biomolecules
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
Espanha