Zobrazeno 1 - 10
of 14
pro vyhledávání: '"Benjamin C Houghton"'
Autor:
Benjamin C. Houghton, Neelam Panchal, Simone A. Haas, Kay O. Chmielewski, Markus Hildenbeutel, Thomas Whittaker, Claudio Mussolino, Toni Cathomen, Adrian J Thrasher, Claire Booth
Publikováno v:
Frontiers in Genome Editing, Vol 4 (2022)
X-linked lymphoproliferative disease is a rare inherited immune disorder, caused by mutations or deletions in the SH2D1A gene that encodes an intracellular adapter protein SAP (Slam-associated protein). SAP is essential for mediating several key immu
Externí odkaz:
https://doaj.org/article/e1802e6cee45487398ad67ff6d224f5a
Autor:
Ying Hong, Marina Casimir, Benjamin C. Houghton, Fang Zhang, Barbara Jensen, Ebun Omoyinmi, Robert Torrance, Charalampia Papadopoulou, Michelle Cummins, Marion Roderick, Adrian J. Thrasher, Paul A. Brogan, Despina Eleftheriou
Publikováno v:
Frontiers in Immunology, Vol 13 (2022)
Deficiency of adenosine deaminase type 2 (DADA2) is an autosomal recessive disease caused by bi-allelic loss-of-function mutations in ADA2. Treatment with anti-TNF is effective for the autoinflammatory and vasculitic components of the disease but doe
Externí odkaz:
https://doaj.org/article/a6c23358d4c647d4a324f1cfe56e4169
Autor:
Benjamin C. Houghton, Claire Booth
Publikováno v:
HemaSphere, Vol 5, Iss 1, p e509 (2021)
Over the past 3 decades, there has been significant progress in refining gene therapy technologies and procedures. Transduction of hematopoietic stem cells ex vivo using lentiviral vectors can now create a highly effective therapeutic product, capabl
Externí odkaz:
https://doaj.org/article/65668c177fe74569980a6e9ce7f36ff8
Autor:
Joseph Topal, Neelam Panchal, Amairelys Barroeta, Anna Roppelt, Annelotte Mudde, H. Bobby Gaspar, Adrian J. Thrasher, Benjamin C. Houghton, Claire Booth
Publikováno v:
Journal of Clinical Immunology. 43:440-451
Background X-linked inhibitor of apoptosis protein (XIAP) deficiency is a severe immunodeficiency with clinical features including hemophagocytic lymphohistiocytosis (HLH) and inflammatory bowel disease (IBD) due to defective NOD2 responses. Manageme
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9ac2153d71f859be873b903841942663
https://doi.org/10.1007/s10875-022-01389-0
https://doi.org/10.1007/s10875-022-01389-0
Autor:
Ying, Hong, Marina, Casimir, Benjamin C, Houghton, Fang, Zhang, Barbara, Jensen, Ebun, Omoyinmi, Robert, Torrance, Charalampia, Papadopoulou, Michelle, Cummins, Marion, Roderick, Adrian J, Thrasher, Paul A, Brogan, Despina, Eleftheriou
Publikováno v:
Frontiers in immunology. 13
Deficiency of adenosine deaminase type 2 (DADA2) is an autosomal recessive disease caused by bi-allelic loss-of-function mutations in
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::f80f16f783cd869f1c8b1c99a12a18a6
https://pubmed.ncbi.nlm.nih.gov/35529868
https://pubmed.ncbi.nlm.nih.gov/35529868
Autor:
Benjamin C. Houghton, Neelam Panchal, Simone A. Haas, Kay O. Chmielewski, Markus Hildenbeutel, Thomas Whittaker, Claudio Mussolino, Toni Cathomen, Adrian J Thrasher, Claire Booth
Publikováno v:
Frontiers in genome editing. 4
X-linked lymphoproliferative disease is a rare inherited immune disorder, caused by mutations or deletions in the SH2D1A gene that encodes an intracellular adapter protein SAP (Slam-associated protein). SAP is essential for mediating several key immu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c46b11746ecdc90f81efcf0cc95302ec
https://pubmed.ncbi.nlm.nih.gov/35677600
https://pubmed.ncbi.nlm.nih.gov/35677600
Autor:
Benjamin C. Houghton, Claire Booth
Publikováno v:
HemaSphere
HemaSphere, Vol 5, Iss 1, p e509 (2021)
HemaSphere, Vol 5, Iss 1, p e509 (2021)
Over the past 3 decades, there has been significant progress in refining gene therapy technologies and procedures. Transduction of hematopoietic stem cells ex vivo using lentiviral vectors can now create a highly effective therapeutic product, capabl
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b8dfceee5088a5a109eab096dbbb6225
https://doi.org/10.1097/hs9.0000000000000509
https://doi.org/10.1097/hs9.0000000000000509
Autor:
Oscar Cordero-Llana, Benjamin C Houghton, Rafael J. Yáñez-Muñoz, James B. Uney, Maeve A. Caldwell, Liang-Fong Wong, Federica Rinaldi, Hannah Taylor
Publikováno v:
Molecular Therapy. 23:244-254
Cerebral Dopamine Neurotrophic Factor (CDNF) and Mesencephalic Astrocyte-derived Neurotrophic factor (MANF) are members of a recently discovered family of neurotrophic factors (NTFs). Here, we used intranigral or intrastriatal lentiviral vector-media
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::72906795fbb719e7d2420d58230e1dac
https://doi.org/10.1038/mt.2014.206
https://doi.org/10.1038/mt.2014.206
Publikováno v:
Current opinion in pharmacology. 24
Lentiviral vectors (LVV) are important tools for the treatment of immune system disorders. Integration of therapeutic genetic material into the haematopoietic stem cell compartment using LVV can mediate long-term correction of haematopoietic lineages
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a36fe56ce7a9cdd943fee7c9ec6eb457
https://pubmed.ncbi.nlm.nih.gov/26363252
https://pubmed.ncbi.nlm.nih.gov/26363252
Publikováno v:
Molecular Therapy. 24:S130-S131
X-linked lymphoproliferative disease is an immunodeficiency arising from mutations in the SH2D1A gene encoding SAP, a key regulator of immune function expressed in T cells, natural killer, and natural killer T cells. Haemopoietic stem-cell transplant
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::c2af8e4f7db9d816c8e39b4e78130ceb
https://doi.org/10.1016/s1525-0016(16)33134-3
https://doi.org/10.1016/s1525-0016(16)33134-3