LGR5-Positive Supporting Cells Survive Ototoxic Trauma in the Adult Mouse Cochlea

Autor: Natalia Smith-Cortinez, Rana Yadak, Ferry G. J. Hendriksen, Eefje Sanders, Dyan Ramekers, Robert J. Stokroos, Huib Versnel, Louise V. Straatman

Publikováno v: Frontiers in Molecular Neuroscience, Vol 14 (2021)

Sensorineural hearing loss is mainly caused by irreversible damage to sensory hair cells (HCs). A subgroup of supporting cells (SCs) in the cochlea express leucine-rich repeat-containing G-protein coupled receptor 5 (LGR5), a marker for tissue-reside

Externí odkaz: https://doaj.org/article/1bafb91347d444e98b0f0bcc45d69bfd

Transplantation, gene therapy and intestinal pathology in MNGIE patients and mice

Autor: Rana Yadak, Max V. Boot, Niek P. van Til, Dominique Cazals-Hatem, Armin Finkenstedt, Elly Bogaerts, Irenaeus F. de Coo, Marianna Bugiani

Publikováno v: BMC Gastroenterology, Vol 18, Iss 1, Pp 1-6 (2018)

Abstract Background Gastrointestinal complications are the main cause of death in patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). Available treatments often restore biochemical homeostasis, but fail to cure gastrointestin

Externí odkaz: https://doaj.org/article/d086ac5e51a34af49fc629842d942533

Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

Autor: Ramon Martí, Niek P. van Til, Joost C. Haeck, Lorena Leonardelli, Luca Biasco, Elly Bogaerts, Irenaeus F.M. de Coo, Merel Stok, Raquel Cabrera-Pérez, Gerard Wagemaker, Steffi Goffart, Marshall W. Huston, Marianna Bugiani, George J G Ruijter, Edwin H. Jacobs, Javier Torres-Torronteras, Monique R. Bernsen, Rana Yadak, Robert M. Verdijk

Publikováno v: Dipòsit Digital de Documents de la UAB
Universitat Autònoma de Barcelona
BASE-Bielefeld Academic Search Engine
Molecular Therapy. Methods & Clinical Development
Molecular Therapy-Methods and Clinical Development, 8, 152-165. Cell Press
Yadak, R, Cabrera-Pérez, R, Torres-Torronteras, J, Bugiani, M, Haeck, J C, Huston, M W, Bogaerts, E, Goffart, S, Jacobs, E H, Stok, M, Leonardelli, L, Biasco, L, Verdijk, R M, Bernsen, M R, Ruijter, G, Martí, R, Wagemaker, G, van Til, N P & de Coo, I F M 2018, ' Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE ', Molecular Therapy-Methods and Clinical Development, vol. 8, pp. 152-165 . https://doi.org/10.1016/j.omtm.2018.01.001
Molecular Therapy-Methods & Clinical Development, 8, 152-165. Cell Press
Molecular Therapy: Methods & Clinical Development, Vol 8, Iss C, Pp 152-165 (2018)

Altres ajuts: The authors acknowledge the financial support for this study by Join4energy, Ride4Kids, the Sophia Foundation (SSW0645), Stichting NeMo, in the context of funding provided by the European Commission's 5th, 6th, and 7th Framework Program

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::465edf7ab8e734f6e13ca5ace6e62bb9
https://doi.org/10.1016/j.omtm.2018.01.001

Pretransplant Mobilization with Granulocyte Colony-Stimulating Factor Improves B-Cell Reconstitution by Lentiviral Vector Gene Therapy in SCID-X1 Mice

Autor: Adriaan R.A. Riegman, Rana Yadak, Marshall W. Huston, Helen de Boer, Niek P. van Til, Yvette van Helsdingen, Gerard Wagemaker

Publikováno v: Human Gene Therapy, 25(10), 905-914. Mary Ann Liebert Inc.

Hematopoietic stem cell (HSC) gene therapy is a demonstrated effective treatment for X-linked severe combined immunodeficiency (SCID-X1), but B-cell reconstitution and function has been deficient in many of the gene therapy treated patients. Cytoredu

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c362f1494ecbca2f0b1dd3f2d68685dc
https://doi.org/10.1089/hum.2014.101

279. Efficient and Safe Lentiviral Vector-Mediated Hematopoietic Stem Cell Gene Therapy in MNGIE Mice

Autor: Rana Yadak, Elly Bogaerts, Jordi Barquinero, George de Ruijter, René F.M. de Coo, Ramon Martí, Javier Torres Torronteras, Cabrera Pérez Raquel, Gerard Wagemaker, Niek P. van Til, Bert Smeets, Marshall W. Huston

Publikováno v: Molecular Therapy. 23:S111-S112

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) patients are deficient in thymidine phosphorylase(TP) resulting in systemic thymidine(Thd) and deoxyuridine(dUrd) accumulation affecting mtDNA replication and causing mitochondrial dysfunc

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d4ca41fd7efcf79c32ec08e09d5ccec5