Zobrazeno 1 - 10
of 108
pro vyhledávání: '"Brian C. Beard"'
Autor:
Susana Navarro, Oscar Quintana-Bustamante, Rebeca Sanchez-Dominguez, Sergio Lopez-Manzaneda, Isabel Ojeda-Perez, Aida Garcia-Torralba, Omaira Alberquilla, Kenneth Law, Brian C. Beard, Antonella Bastone, Michael Rothe, Mariela Villanueva, Juan C. Ramirez, Sara Fañanas-Baquero, Virginia Nieto-Romero, Andrea Molinos-Vicente, Sonia Gutierrez, Eileen Nicoletti, María García-Bravo, Juan A. Bueren, Jonathan D. Schwartz, Jose-Carlos Segovia
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss , Pp 350-359 (2021)
Pyruvate kinase deficiency (PKD) is a rare autosomal recessive disorder caused by mutations in the PKLR gene. PKD is characterized by non-spherocytic hemolytic anemia of variable severity and may be fatal in some cases during early childhood. Althoug
Externí odkaz:
https://doaj.org/article/520b1bb80d5c442ebaab57f472bd9735
Publikováno v:
Viruses, Vol 4, Iss 12, Pp 3572-3588 (2012)
Foamy virus (FV) vectors have shown great promise for hematopoietic stem cell (HSC) gene therapy. Their ability to efficiently deliver transgenes to multi-lineage long-term repopulating cells in large animal models suggests they will be effective for
Externí odkaz:
https://doaj.org/article/be0a21789d0748dba2a495fad10b711f
Autor:
Hans-Peter Kiem, Paritha I Arumugam, Christopher R Burtner, Catherine F Fox, Brian C Beard, Phillip Dexheimer, Jennifer E Adair, Punam Malik
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Safely achieving long-term engraftment of genetically modified hematopoietic stem cells (HSCs) that maintain therapeutic transgene expression is the benchmark for successful application of gene therapy for hemoglobinopathies. We used the pigtailed ma
Externí odkaz:
https://doaj.org/article/b46866ce35c945febe5e04935167b2b3
Correction: Stem Cell Selection In Vivo Using Foamy Vectors Cures Canine Pyruvate Kinase Deficiency.
Autor:
Grant D. Trobridge, Brian C. Beard, Robert A. Wu, Christina Ironside, Punam Malik, Hans-Peter Kiem
Publikováno v:
PLoS ONE, Vol 8, Iss 10 (2013)
Externí odkaz:
https://doaj.org/article/e32a0f5314b740b29e9c2aa08a6b83d4
Autor:
Carolina Berger, Michael Berger, Brian C Beard, Hans-Peter Kiem, Theodore A Gooley, Stanley R Riddell
Publikováno v:
PLoS ONE, Vol 8, Iss 2, p e56268 (2013)
The adoptive transfer of antigen-specific effector T cells is being used to treat human infections and malignancy. T cell persistence is a prerequisite for therapeutic efficacy, but reliably establishing a high-level and durable T cell response by tr
Externí odkaz:
https://doaj.org/article/53f43b84593f4f80abd90c37a3119236
Autor:
Grant D Trobridge, Brian C Beard, Robert A Wu, Christina Ironside, Punam Malik, Hans-Peter Kiem
Publikováno v:
PLoS ONE, Vol 7, Iss 9, p e45173 (2012)
Hematopoietic stem cell (HSC) gene therapy has cured immunodeficiencies including X-linked severe combined immunodeficiency (SCID-X1) and adenine deaminase deficiency (ADA). For these immunodeficiencies corrected cells have a selective advantage in v
Externí odkaz:
https://doaj.org/article/cffd402cd33649b3b1ccd91604dae5fa
Autor:
Grant D Trobridge, Robert A Wu, Brian C Beard, Sum Ying Chiu, Nina M Muñoz, Dorothee von Laer, John J Rossi, Hans-Peter Kiem
Publikováno v:
PLoS ONE, Vol 4, Iss 11, p e7693 (2009)
There is currently no effective AIDS vaccine, emphasizing the importance of developing alternative therapies. Recently, a patient was successfully transplanted with allogeneic, naturally resistant CCR5-negative (CCR5Delta32) cells, setting the stage
Externí odkaz:
https://doaj.org/article/7c4ffe9431fe482d980c8837ef4f4757
Autor:
Susana Navarro, Gayatri R Rao, Camilla Duran-Persson, Maria Grazia Roncarolo, Josune Zubicaray, Philip Ancliff, John E. Wagner, Julián Sevilla, Brian C. Beard, Jonathan D. Schwartz, Rajni Agarwal, Claire Booth, Miriam Zeini, Elena Sebastián, Grace Choi, Kenneth Law, Juan A. Bueren, Paula Río, Adrian J. Thrasher, Agnieszka Czechowicz, Eileen Nicoletti
Publikováno v:
Blood. 138:3968-3968
Background: Fanconi anemia (FA) is a disorder of defective deoxyribonucleic acid (DNA) repair, progressive bone marrow failure (BMF), and a predisposition to hematologic malignancies and solid tumors. Approximately 60 to 70% of all cases result from
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ae173e5f4a6caa5034b030200c2d18dc
https://doi.org/10.1182/blood-2021-147071
https://doi.org/10.1182/blood-2021-147071
Autor:
Eileen Nicoletti, Bert Glader, Grace Choi, Maria Grazia Roncarolo, Sol Sanchez, Juan A. Bueren, Begoña Pérez de Camino Gaisse, Brian C. Beard, José C. Segovia, Ami J. Shah, Susana Navarro, Oscar Quintana Bustamante, Lucía Llanos, Julián Sevilla, Kenneth Law, Gayatri R Rao, Miriam Zeini, Jose Luis Lopez Lorenzo, Jonathan D. Schwartz, May Chien
Publikováno v:
Blood. 138:563-563
Background: Pyruvate kinase deficiency (PKD) is a rare inherited hemolytic anemia caused by mutations in the PKLR gene resulting in decreased red cell pyruvate kinase activity and impaired erythrocyte metabolism. Manifestations include anemia, reticu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::5d6df8e462ce0cbab4f915dd187f835d
https://doi.org/10.1182/blood-2021-148161
https://doi.org/10.1182/blood-2021-148161
Autor:
Elena Almarza, Brian C. Beard, Theodore B. Moore, Gayatri R Rao, Claire Booth, Kenneth Law, Donald B. Kohn, Satiro N. De Oliveira, Jonathan D. Schwartz, Juan A. Bueren, Caroline Y. Kuo, Eileen Nicoletti, Adrian J. Thrasher, Miriam Zeini, Augustine Fernandes, Grace Choi, Cristina Mesa-Núñez, Dayna Terrazas, Julián Sevilla
Publikováno v:
Blood. 138:2932-2932
Background: Leukocyte Adhesion Deficiency-I (LAD-I) is a rare disorder of neutrophil adhesion resulting from ITGB2 mutations encoding for the β2-integrin component, CD18. Severe LAD-I (CD18 Methods: Pediatric patients ≥ 3 months old with severe LA
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::db6471738fe605e2ed22270803c3622c
https://doi.org/10.1182/blood-2021-151898
https://doi.org/10.1182/blood-2021-151898