Human <i>GLB1</i> knockout cerebral organoids: A model system for testing AAV9-mediated <i>GLB1</i> gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis.

Latour, Yvonne L; Yoon, Robin; Thomas, Sarah E; Grant, Christina; Li, Cuiling; Sena-Esteves, Miguel; Allende, Maria L; Proia, Richard L; Tifft, Cynthia J

Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis.

Latour, Yvonne L; Yoon, Robin; Thomas, Sarah E; Grant, Christina; Li, Cuiling; Sena-Esteves, Miguel; Allende, Maria L; Proia, Richard L; Tifft, Cynthia J.

Afiliação

Latour YL; Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Yoon R; Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Thomas SE; Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Grant C; Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Li C; Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Sena-Esteves M; Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Allende ML; Genetics of Development and Disease Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, USA.
Proia RL; Genetics of Development and Disease Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, USA.
Tifft CJ; Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.

Mol Genet Metab Rep ; 21: 100513, 2019 Dec.

Article em En | MEDLINE | ID: mdl-31534909

Palavras-chave

4MU, 4-methylumbelliferyl; AAV, adeno-associated virus; AAV9, AAV serotype 9; BSA, bovine serum albumin; CNS, central nervous system; CPB, citrate phosphate buffer; EB, embryoid body; GFP, green fluorescent protein; HPTLC, high-performance thin-layer chromatography; PBS, phosphate-buffered saline; RT-qPCR, real-time quantitative polymerase chain reaction; SD, standard deviation; X-gal, 5-bromo-4-chloro-3-indolyl-ß-D-galactopyranoside; hiPSC, human induced pluripotent stem cells; iPS, induced pluripotent stem; ß-gal, ß-galactosidase

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Ano de publicação: 2019 Tipo de documento: Article

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Ano de publicação: 2019 Tipo de documento: Article