Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?

Horgan, Claire; Jones, Simon A; Bigger, Brian W; Wynn, Robert

Horgan, Claire; Jones, Simon A; Bigger, Brian W; Wynn, Robert.

Afiliação

Horgan C; Blood and Marrow Transplant Unit, Department of Paediatric Haematology, Royal Manchester Children's Hospital, Manchester University NHS Foundation Trust, Manchester M13 9WL, UK.
Jones SA; Willink Unit, Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Manchester M13 9WL, UK.
Bigger BW; Stem Cell and Neuropathies, Division of Cell Matrix Biology & Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK.
Wynn R; Blood and Marrow Transplant Unit, Department of Paediatric Haematology, Royal Manchester Children's Hospital, Manchester University NHS Foundation Trust, Manchester M13 9WL, UK.

Int J Mol Sci ; 23(9)2022 Apr 27.

Article em En | MEDLINE | ID: mdl-35563245

Assuntos

Transplante de Células-Tronco Hematopoéticas; Iduronato Sulfatase; Mucopolissacaridose II; Encéfalo/metabolismo; Encéfalo/patologia; Terapia Genética/métodos; Células-Tronco Hematopoéticas/metabolismo; Humanos; Iduronato Sulfatase/genética; Iduronato Sulfatase/metabolismo; Iduronato Sulfatase/uso terapêutico; Mucopolissacaridose II/genética; Mucopolissacaridose II/patologia; Mucopolissacaridose II/terapia

Palavras-chave

bone marrow transplant; mucopolysaccharidosis type II; stem cell gene therapy

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridose II / Transplante de Células-Tronco Hematopoéticas / Iduronato Sulfatase Limite: Humans Idioma: En Revista: Int J Mol Sci Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Reino Unido

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