Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease

Autor: Jane E. Potter, Gemma Petts, Arunabha Ghosh, Fiona J. White, Jane L. Kinsella, Stephen Hughes, Jane Roberts, Adam Hodgkinson, Kathryn Brammeier, Heather Church, Christine Merrigan, Joanne Hughes, Pamela Evans, Helen Campbell, Denise Bonney, William G. Newman, Brian W. Bigger, Alexander Broomfield, Simon A. Jones, Robert F. Wynn

Publikováno v: Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-14 (2021)

Abstract Background Wolman disease is a rare, lysosomal storage disorder in which biallelic variants in the LIPA gene result in reduced or complete lack of lysosomal acid lipase. The accumulation of the substrates; cholesterol esters and triglyceride

Externí odkaz: https://doaj.org/article/d3be9f8f7dcb489b904a47d7c2643970

T-Cell Replete Unrelated Cord Blood Transplant Give Superior Outcomes to Other Donor Sources in High Risk and Relapsed/Refractory Paediatric Myeloid Malignancy

Autor: Claire Horgan, Khushnuma Mullanfiroze, Archana Rauthan, Katharine Patrick, Naeem Akram Butt, Oana Mirci-Danicar, Olya O'Connor, Caroline Furness, Akshay Deshpande, Sarah Lawson, Valerie Broderick, Pamela Evans, Brenda Gibson, Wing Roberts, Sevasti Galani, Amy A. Kirkwood, Beki James, Kanchan Rao, Persis J. Amrolia, Robert F. Wynn

Publikováno v: Blood. 140:4889-4891

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::e9e5f721f4ec08fb30990ff9fd04e644
https://doi.org/10.1182/blood-2022-165327

Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease

Autor: Denise Bonney, Alexander Broomfield, Fiona White, Jane Roberts, Jane Louise Kinsella, Arunabha Ghosh, Robert Wynn, Joanne Hughes, Jane E. Potter, Helen Campbell, Christine Merrigan, Pamela Evans, Adam Hodgkinson, Gemma Petts, Simon Jones, Heather J. Church, Stephen M. Hughes, Brian W. Bigger, William G. Newman, Kathryn Brammeier

Publikováno v: Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-14 (2021)
Potter, J, Newman, W, Jones, S, Wynn, R, Ghosh, A, Broomfield, A & Bigger, B 2021, ' Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease ', Orphanet Journal of Rare Diseases, vol. 16, no. 1, 235 . https://doi.org/10.1186/s13023-021-01849-7
Orphanet Journal of Rare Diseases

BackgroundWolman disease is a rare, lysosomal storage disorder in which biallelic variants in theLIPAgene result in reduced or complete lack of lysosomal acid lipase. The accumulation of the substrates; cholesterol esters and triglycerides, significa

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::646bbf105331fe78fffe045b41d6488f
https://doaj.org/article/d3be9f8f7dcb489b904a47d7c2643970

Graft-versus-host disease reduces lymph node display of tissue-restricted self-antigens and promotes autoimmunity

Autor: Pamela Evans, Simone Dertschnig, Ronjon Chakraverty, Hans J. Stauss, Pedro Santos e Sousa, Teresa Manzo, Clare L. Bennett, Ivana R. Ferrer

Publikováno v: The Journal of Clinical Investigation

Acute graft-versus-host disease (GVHD) is initially triggered by alloreactive T cells, which damage peripheral tissues and lymphoid organs. Subsequent transition to chronic GVHD involves the emergence of autoimmunity, although the underlying mechanis

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a889538a264f4211577a3db1f7ea25c4
http://europepmc.org/articles/PMC7108931

Mother Donors Improve Outcomes after HLA Haploidentical Transplantation: A Study by the Cellular Therapy and Immunobiology Working Party of the European Society for Blood and Marrow Transplantation

Autor: Loredana Ruggeri, Dirk-Jan Eikema, Attilio Bondanza, Maddalena Noviello, Anja van Biezen, Liesbeth C. de Wreede, Lara Crucitti, Luca Vago, Sara Ciardelli, Peter Bader, Yener Koc, Franco Locatelli, Joan H. Veelken, Bernd Gruhn, Pamela Evans, Christian Chabannon, Antoine Toubert, Andrea Velardi

Publikováno v: Transplantation and Cellular Therapy, 28(4). ELSEVIER SCIENCE INC

Transplacental trafficking of maternal and fetal cells during pregnancy establishes long-term reciprocal microchimerism in both mother and child. Consequently, the maternal immune system may become sensitized to paternal histocompatibility antigens.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9e22b53e77b8a40361af0790aa4f606e
https://doi.org/10.1016/j.jtct.2022.01.001