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Akademický článek

Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease

Autor: Yewande Pearse, Don Clarke, Shih-hsin Kan, Steven Q. Le, Valentina Sanghez, Anna Luzzi, Ivy Pham, Lina R. Nih, Jonathan D. Cooper, Patricia I. Dickson, Michelina Iacovino

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 452-463 (2022)

Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB) is a recessive genetic disorder that severely affects the brain due to a deficiency in the enzyme α-N-acetylglucosaminidase (NAGLU), leading to intra-lysosomal accumulation of partially de

Externí odkaz: https://doaj.org/article/776a0875754d4cc8976309f509235d55

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Akademický článek

Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice

Autor: Don Clarke, Yewande Pearse, Shih-hsin Kan, Steven Q. Le, Valentina Sanghez, Jonathan D. Cooper, Patricia I. Dickson, Michelina Iacovino

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 113-127 (2018)

Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB [MPS IIIB]) is a lysosomal storage disorder primarily affecting the brain that is caused by a deficiency in the enzyme α-N-acetylglucosaminidase (NAGLU), leading to intralysosomal accumulat

Externí odkaz: https://doaj.org/article/a51965c026fa4337a9814a580dc89e80

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Akademický článek

A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I

Autor: Steven Q. Le, Shih-hsin Kan, Don Clarke, Valentina Sanghez, Martin Egeland, Kristen N. Vondrak, Terence M. Doherty, Moin U. Vera, Michelina Iacovino, Jonathan D. Cooper, Mark S. Sands, Patricia I. Dickson

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 8, Iss C, Pp 42-51 (2018)

Antibodies against recombinant proteins can significantly reduce their effectiveness in unanticipated ways. We evaluated the humoral response of mice with the lysosomal storage disease mucopolysaccharidosis type I treated with weekly intravenous reco

Externí odkaz: https://doaj.org/article/458c35de4ec1464eb7de993788d68d45

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Akademický článek

Notch activation is required for downregulation of HoxA3-dependent endothelial cell phenotype during blood formation.

Autor: Valentina Sanghez, Anna Luzzi, Don Clarke, Dustin Kee, Steven Beuder, Danielle Rux, Mitsujiro Osawa, Joaquín Madrenas, Tsui-Fen Chou, Michael Kyba, Michelina Iacovino

Publikováno v: PLoS ONE, Vol 12, Iss 10, p e0186818 (2017)

Hemogenic endothelium (HE) undergoes endothelial-to-hematopoietic transition (EHT) to generate blood, a process that requires progressive down-regulation of endothelial genes and induction of hematopoietic ones. Previously, we have shown that the tra

Externí odkaz: https://doaj.org/article/cbbdf91f80c6484ea0168b76418e3943

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Brain transplantation of genetically corrected Sanfilippo B Neural Stem Cells induces partial cross-correction of the disease

Autor: Yewande Pearse, Don Clarke, Shih-hsin Kan, Steven Q. Le, Valentina Sanghez, Anna Luzzi, Ivy Pham, Lina R. Nih, Jonathan D. Cooper, Patricia I. Dickson, Michelina Iacovino

Sanfilippo syndrome type B (Mucopolysaccharidosis type IIIB or MPS IIIB) is a recessive genetic disorder that severely affects the brain due to a deficiency in the enzyme α-N-acetylglucosaminidase (NAGLU), leading to intralysosomal accumulation of p

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::0291867037a3af9b333959e73610963a
https://doi.org/10.1101/2022.06.30.498131

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Quantifying Forearm Soft Tissue Motion from Massless Skin Markers following Forward Fall Hand Impacts

Autor: Don Clarke, Paula M. van Wyk, William Altenhof, Danielle L. Gyemi, David M. Andrews

Publikováno v: INTERNATIONAL JOURNAL OF KINESIOLOGY AND SPORTS SCIENCE, Vol 6, Iss 3, Pp 1-11 (2018)

Background: Investigating soft tissue motion related to impact events is important for understanding how the body mitigates potentially injurious forces through shock attenuation. Objectives: The aims of this study were to: 1) quantify displacement a

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::120b3f91d472972ac9785097fb7bda57
https://journals.aiac.org.au/index.php/IJKSS/article/view/4577

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