Zobrazeno 1 - 10
of 26
pro vyhledávání: '"Spencer K. Sullivan"'
Autor:
Prasuna Paluru, Kristin M. Hudock, Xin Cheng, Jason A. Mills, Lei Ying, Aline M. Galvão, Lin Lu, Amita Tiyaboonchai, Xiuli Sim, Spencer K. Sullivan, Deborah L. French, Paul Gadue
Publikováno v:
Stem Cell Research, Vol 12, Iss 2, Pp 441-451 (2014)
The Wnt gene family consists of structurally related genes encoding secreted signaling molecules that have been implicated in many developmental processes, including regulation of cell fate and patterning during embryogenesis. Previously, we found th
Externí odkaz:
https://doaj.org/article/448b895343704a899b23594fb103a169
Autor:
Jason A. Mills, Kristin M. Hudock, Spencer K. Sullivan, Pamela Herrera, Lisa M. Sullivan, Paul Gadue, Deborah L. French
Publikováno v:
Stem Cell Research, Vol 15, Iss 3, Pp 595-597 (2015)
Poikiloderma with neutropenia (PN, Clericuzio-type poikiloderma with neutropenia) is a rare autosomal recessive disorder caused by biallelic mutations in the USB1 gene (Alias C16orf57 and MPN1). To date, there have been only 37 reported cases worldwi
Externí odkaz:
https://doaj.org/article/e9265250f8af4a6fb7492f4185b71f5e
Autor:
Xavier M Anguela, Kristen Jaworski, Stacy E Croteau, John E J Rasko, Tiffany Chang, Federico Mingozzi, Paul E Monahan, Katherine A. High, Kathleen Z Reape, Margaret V Ragni, Lindsey A. George, Amy Macdougall, Spencer K. Sullivan, M Elaine Eyster, Benjamin J. Samelson-Jones, Robert Noble, Michael Recht, Marla Curran, Klaudia Kuranda
Publikováno v:
N Engl J Med
Background The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose. Methods In this phase 1-2 trial, we
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a13db718b1db9c4d1a4f5c07a1a3abc2
https://pubmed.ncbi.nlm.nih.gov/34788507
https://pubmed.ncbi.nlm.nih.gov/34788507
Autor:
Jonathan M. Ducore, John E.J. Rasko, Lynne Smith, Adam Giermasz, Jerome M. Teitel, A Chhabra, Spencer K. Sullivan, Jeremy Rupon, Ben J. Samelson-Jones, Lindsey A. George, Amanda C. O’Brien, Catherine E. McGuinn, Ian Winburn
Publikováno v:
Blood. 138:3975-3975
Fifteen patients with moderately severe to severe hemophilia B (factor IX [FIX] activity ≤2%) were treated with fidanacogene elaparvovec at a dose of 5e11 vg/kg as part of a phase 1/2a study. The study was 52 weeks in duration, after which patients
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::52b8ffb486f165d405e74851019539ff
https://doi.org/10.1182/blood-2021-150541
https://doi.org/10.1182/blood-2021-150541
Autor:
Christopher S. Thom, Vincent Hayes, Danuta Jarocha, Mortimer Poncz, Deborah L. French, Karen K. Vo, Randolph B. Lyde, Spencer K. Sullivan
Publikováno v:
Blood. 129:3486-3494
Friend leukemia virus integration 1 (FLI1), a critical transcription factor (TF) during megakaryocyte differentiation, is among genes hemizygously deleted in Jacobsen syndrome, resulting in a macrothrombocytopenia termed Paris-Trousseau syndrome (PTS
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::52c5eb1b25d3e63c7a9371e1455d78ec
https://doi.org/10.1182/blood-2017-02-770958
https://doi.org/10.1182/blood-2017-02-770958
Autor:
Johannes C.M. Van der Loo, Jerome M. Teitel, Benjamin J. Samelson-Jones, Adam Cuker, Xavier M. Anguela, Marcus E. Carr, Catherine E. McGuinn, Yifeng Chen, Linda B. Couto, Adam Giermasz, Suvankar Majumdar, Katie Wachtel, Jonathan M. Ducore, Stefan Tiefenbacher, Margaret V. Ragni, J. Fraser Wright, Olga Zelenaia, Katherine A. High, Yun Liu, John E.J. Rasko, Daniel Takefman, Lisa M. Sullivan, Angela Winters, Daniel Hui, Alvin Luk, Spencer K. Sullivan, Valder R. Arruda, Lindsey A. George
Publikováno v:
The New England journal of medicine. 377(23)
The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia.We infused a si
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6bbb2657407ff2d3c3546fcc638e6677
https://pubmed.ncbi.nlm.nih.gov/29274719
https://pubmed.ncbi.nlm.nih.gov/29274719
Autor:
Rodney M. Camire, John Tkaczynski, Danuta Jarocha, Denise E. Sabatino, Mortimer Poncz, Randolph B. Lyde, Hyun Sook Ahn, Deborah L. French, Karen K. Vo, Spencer K. Sullivan, Elsa Treffeisen
Publikováno v:
Blood advances. 3(9)
B-domainless factor VIII (FVIII) ectopically expressed in megakaryocytes (MKs) is stored in α granules of platelets (pFVIII) and is capable of restoring hemostasis in FVIIInull mice, even in the presence of circulating inhibitors. However, our prior
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7736679ae7d53b47302efbf237b5467c
https://pubmed.ncbi.nlm.nih.gov/31036722
https://pubmed.ncbi.nlm.nih.gov/31036722
Autor:
Amita Tiyaboonchai, Aline M. Galvão, Xin Cheng, Paul Gadue, Spencer K. Sullivan, Lin Lu, Jason A. Mills, Kristin M. Hudock, Xiuli Sim, Prasuna Paluru, Deborah L. French, Lei Ying
Publikováno v:
Stem Cell Research, Vol 12, Iss 2, Pp 441-451 (2014)
The Wnt gene family consists of structurally related genes encoding secreted signaling molecules that have been implicated in many developmental processes, including regulation of cell fate and patterning during embryogenesis. Previously, we found th
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cb808fc02b50b6ba4d4af8ca8054de45
http://www.sciencedirect.com/science/article/pii/S187350611300175X
http://www.sciencedirect.com/science/article/pii/S187350611300175X
Autor:
Adam C. Gower, Aline M. Galvão, Jason A. Mills, Jyh-Chang Jean, Yu Yao, Lisa M. Sullivan, Spencer K. Sullivan, Paul Gadue, Lin Lu, Gustavo Mostoslavsky, Helen Mac, Mitchell J. Weiss, Prasuna Paluru, Lei Ying, Darrell N. Kotton, Amel Omari, A. Spira, Dongbin Xu, Deborah L. French, Kai Wang, Steven S. Shen
Publikováno v:
Blood. 122:2047-2051
Induced pluripotent stem cells (iPSCs) hold great promise for modeling human hematopoietic diseases. However, intrinsic variability in the capacities of different iPSC lines for hematopoietic development complicates comparative studies and is current
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a84d4b70de53034c0ac440c8573dc400
https://doi.org/10.1182/blood-2013-02-484444
https://doi.org/10.1182/blood-2013-02-484444
Autor:
Deborah L. French, Chiaka Aribeana, Julie Weng, Sarah K. Tasian, Stella T. Chou, Helene Favre, Silvia Bresolin, Mignon L. Loh, Spencer K. Sullivan, Shilpa Gandre-Babbe, Lin Lu, Mitchell J. Weiss, Prasuna Paluru, John K. Choi
Publikováno v:
Blood. 121:4925-4929
Juvenile myelomonocytic leukemia (JMML) is an aggressive myeloproliferative neoplasm of young children initiated by mutations that deregulate cytokine receptor signaling. Studies of JMML are constrained by limited access to patient tissues. We genera
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e35c43a355835be2b6e47f5a4a9a9b80
https://doi.org/10.1182/blood-2013-01-478412
https://doi.org/10.1182/blood-2013-01-478412