Zobrazeno 1 - 4
of 4
pro vyhledávání: '"Rob Sokolic"'
Severe combined immunodeficiency: improved survival leading to detection of underlying liver disease
Autor:
Anusha Vittal, Nehna Abdul Majeed, Elizabeth Garabedian, Jamie Marko, David E Kleiner, Rob Sokolic, Fabio Candotti, Harry Malech, Theo Heller, Christopher Koh
Publikováno v:
BMC Gastroenterology, Vol 23, Iss 1, Pp 1-8 (2023)
Abstract Background Adenosine deaminase deficiency (ADA) is an autosomal recessive disorder leading to severe combined immunodeficiency (SCID). It is characterized patho-physiologically by intracellular accumulation of toxic products affecting lympho
Externí odkaz:
https://doaj.org/article/256b0a5a135547408f0e2ed934e6b8d6
Severe Combined Immunodeficiency: Improved survival leading to detection of underlying liver disease
Autor:
Anusha Vittal, Nehna Abdul Majeed, Elizabeth Garabedian, Jamie Marko, David E Kleiner, Rob Sokolic, Fabio Candotti, Harry Malech, Theo Heller, Christopher Koh
Background Adenosine deaminase deficiency (ADA) is an autosomal recessive disorder leading to severe combined immunodeficiency (SCID). It is characterized patho-physiologically by intracellular accumulation of toxic products affecting lymphocytes. Ot
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2e36948ae56ed19ce6998ec51532b5e8
https://doi.org/10.21203/rs.3.rs-1915188/v1
https://doi.org/10.21203/rs.3.rs-1915188/v1
Autor:
Sally Shupien, Linda M. Muul, Fabio Candotti, Berkley Brown, Pei Yu Fu, Rob Sokolic, Barbara Nowicki, Otto O. Yang, Aaron R. Cooper, Andrew M. Scharenberg, Christopher Silvin, David W. Gjertson, Alejandra Davila, Kit L. Shaw, Dayna Terrazas, Donald B. Kohn, Alan Ikeda, Beatriz Campo Fernandez, Radha G. Rishi, Neena Kapoor, Xiaoyan Wang, Denise Carbonaro, Suparna Mishra, Arumugam Balamurugan, G. Jayashree Jagadeesh, Satiro N. De Oliveira, Yeong Choi, Kenneth Cornetta, Suzanne Skoda-Smith, Michael S. Hershfield, Roshini S. Abraham, Sabine Geiger, David Buchbinder, Barbara C. Engel, Provaboti Barman, Theodore B. Moore, Kathey Mohan, E. Monika Smogorzewska, Elizabeth Garabedian, Allen Yu, Ken Purdy, John Tse, Greg M. Podsakoff, Stacie M. Anderson
Publikováno v:
Journal of Clinical Investigation. 127:1689-1699
Background Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT that has shown clinical benefit for adenosine deaminase-deficient (ADA-deficient) SC
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c7495591a0b8eb59cc66fe20e2a54576
https://doi.org/10.1172/jci90367
https://doi.org/10.1172/jci90367
Autor:
Kit L. Shaw, Elizabeth Garabedian, Rob Sokolic, Provaboti Barman, Alejandra Davila, Christopher Silvin, Satiro de Oliveira, Ami J. Shah, Dayna Terrazas, Denise Carbonaro, Sabine Geiger, Suparna Mishra, Aaron Cooper, Monika Smogorzewska, Jayashree Jagadeesh, Michael S. Hershfield, Alan Wayne, Gay M. Crooks, Theodore Moore, Fabio Candotti, Donald B. Kohn
Publikováno v:
Molecular Therapy. 23:S13-S14
We report follow-up of subjects treated in a Phase II study of gene therapy for ADA-SCID. Between 2009 and 2012, ten ADA-deficient SCID patients were treated by γ-retroviral-mediated gene transfer (MND-ADA) to their bone marrow CD34+ cells. The subj
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e3063675182e1f6bcda29c8f1571eb54