miR-143 Regulates Lysosomal Enzyme Transport across the Blood-Brain Barrier and Transforms CNS Treatment for Mucopolysaccharidosis Type I.

Lin, Yi; Wang, Xiaohong; Rose, Kevin P; Dai, Mei; Han, Jingfen; Xin, Mei; Pan, Dao

Lin, Yi; Wang, Xiaohong; Rose, Kevin P; Dai, Mei; Han, Jingfen; Xin, Mei; Pan, Dao.

Afiliação

Lin Y; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45219, USA.
Wang X; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45219, USA.
Rose KP; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45219, USA.
Dai M; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45219, USA.
Han J; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45219, USA.
Xin M; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45219, USA; Department of Pediatrics, University of Cincinnati, Cincinnati, OH 45229, USA.
Pan D; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45219, USA; Department of Pediatrics, University of Cincinnati, Cincinnati, OH 45229, USA. Electronic address: dao.pan@cchmc.org.

Mol Ther ; 28(10): 2161-2176, 2020 10 07.

Article em En | MEDLINE | ID: mdl-32610100

Assuntos

Barreira Hematoencefálica/metabolismo; Sistema Nervoso Central/metabolismo; Lisossomos/metabolismo; MicroRNAs/genética; Mucopolissacaridose I/genética; Mucopolissacaridose I/metabolismo; Animais; Sistema Nervoso Central/patologia; Modelos Animais de Doenças; Células Endoteliais/metabolismo; Regulação da Expressão Gênica; Técnicas de Transferência de Genes; Terapia Genética; Células-Tronco Hematopoéticas/metabolismo; Humanos; Camundongos; Mucopolissacaridose I/terapia; Transporte Proteico; Interferência de RNA; Transdução Genética

Palavras-chave

blood-brain barrier; brain drug delivery; cation-independent mannose 6-phosphate receptor; enzyme replacement therapy; gene therapy; in vivo evaluation; lysosomal storage diseases; microRNA-143; mucopolysaccharidosis type I; neurological diseases

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Barreira Hematoencefálica / Sistema Nervoso Central / Mucopolissacaridose I / MicroRNAs / Lisossomos Limite: Animals / Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google