Angiopoietin-like protein 3 promotes preservation of stemness during ex vivo expansion of murine hematopoietic stem cells.

Autor: Elnaz Farahbakhshian, Monique M Verstegen, Trudi P Visser, Sima Kheradmandkia, Dirk Geerts, Shazia Arshad, Noveen Riaz, Frank Grosveld, Niek P van Til, Jules P P Meijerink

Publikováno v: PLoS ONE, Vol 9, Iss 8, p e105642 (2014)

Allogeneic hematopoietic stem cell (HSC) transplantations from umbilical cord blood or autologous HSCs for gene therapy purposes are hampered by limited number of stem cells. To test the ability to expand HSCs in vitro prior to transplantation, two g

Externí odkaz: https://doaj.org/article/582a3ab15d1841a4aa688c9dadabc450

CXCR4 expression by mesenchymal stromal cells is lost after use of enzymatic dissociation agents, but preserved by use of non-enzymatic methods

Autor: Gözde Aydin, Fatima Aerts-Kaya, Duygu Uckan-Cetinkaya, Trudi P. Visser, Burcu Pervin

Publikováno v: International Journal of Hematology. 113:5-9

In recent years, multipotent mesenchymal stromal cells (MSCs) have demonstrated tremendous potential for use in regenerative medicine. CXCR4, the receptor for CXCL12, is highly expressed by bone marrow (BM) MSCs and the CXCR4/CXCL12 axis has been sho

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::af9d3be098565296204082c08633d228
https://doi.org/10.1007/s12185-020-03043-0

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

Autor: Marshall W. Huston, Edwin H. Jacobs, Gerard Wagemaker, Onno Roovers, Elza D. van Deel, Helen de Boer, Arnold J.J. Reuser, Dirk J. Duncker, Niek P. van Til, Holger Jahr, Trudi P. Visser, Merel Stok

Publikováno v: Molecular Therapy-Methods & Clinical Development, 17, 1014-1025. Cell Press
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 1014-1025 (2020)
Molecular Therapy. Methods & Clinical Development

Pompe disease is an autosomal recessive lysosomal storage disorder characterized by progressive muscle weakness. The disease is caused by mutations in the acid α-glucosidase (GAA) gene. Despite the currently available enzyme replacement therapy (ERT

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bb5f7dcb16f73f160c5eadec14494bb6
https://pubmed.ncbi.nlm.nih.gov/32462050

Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning

Autor: Axel Schambach, Niek P. van Til, Shazia Arshad, Michael P. Blundell, Ali Nowrouzi, Adrian J. Thrasher, Claudia Cattoglio, Fang Zhang, Trudi P. Visser, Christof von Kalle, Gerard Wagemaker, Marshall W. Huston, Monique M.A. Verstegen, Yuedan Li, Fulvio Mavilio, Martijn H. Brugman, Manfred Schmidt, Christopher Baum

Publikováno v: Molecular Therapy; Vol 19
Molecular Therapy, 19(10), 1867-1877. Cell Press

Clinical trials have demonstrated the potential of ex vivo hematopoietic stem cell gene therapy to treat X-linked severe combined immunodeficiency (SCID-X1) using retroviral vectors, leading to immune system functionality in the majority of treated p

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fa2bb57d5d088b7d21a14eda468a45b0

Human thymus contains multipotent progenitors with T/B lymphoid, myeloid, and erythroid lineage potential

Autor: Martijn H. Brugman, Willem A. Dik, Gerard Wagemaker, Edwin F. E. de Haas, Floor Weerkamp, Miranda R. M. Baert, Trudi P. Visser, Jacques J.M. van Dongen, Christianne J.M. de Groot, Frank J. T. Staal

Publikováno v: Blood, 107(8), 3131-3137. American Society of Hematology

It is a longstanding question which bone marrow–derived cell seeds the thymus and to what level this cell is committed to the T-cell lineage. We sought to elucidate this issue by examining gene expression, lineage potential, and self-renewal capaci

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::619247651f591b30452b8d1c2c2b7f6b
https://doi.org/10.1182/blood-2005-08-3412