iPSC-derived neural precursor cells engineering <i>GBA1</i> recovers acid ß-glucosidase deficiency and diminishes &#945;-synuclein and neuropathology.

Peng, Yanyan; Liou, Benjamin; Lin, Yi; Mayhew, Christopher N; Fleming, Sheila M; Sun, Ying

iPSC-derived neural precursor cells engineering GBA1 recovers acid ß-glucosidase deficiency and diminishes α-synuclein and neuropathology.

Peng, Yanyan; Liou, Benjamin; Lin, Yi; Mayhew, Christopher N; Fleming, Sheila M; Sun, Ying.

Afiliação

Peng Y; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Liou B; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Lin Y; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Mayhew CN; Division of Developmental Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Fleming SM; Center for Stem Cell and Organoid Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Sun Y; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH 45229, USA.

Mol Ther Methods Clin Dev ; 29: 185-201, 2023 Jun 08.

Article em En | MEDLINE | ID: mdl-37063480

Palavras-chave

Parkinson disease; acid ß-glucosidase; alpha-synuclein; cell therapy; neural precursor cell; neurodegeneration; neuronopathic Gaucher disease

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Ano de publicação: 2023 Tipo de documento: Article