Zobrazeno 1 - 10
of 121
pro vyhledávání: '"Hanspeter Rottensteiner"'
Autor:
Paolo Rossato, Enrica Federti, Alessandro Matte, Helmut Glantschnig, Fabio Canneva, Maria Schuster, Sogue Coulibaly, Gerald Schrenk, Dirk Voelkel, Michael Dockal, Barbara Plaimauer, Immacolata Andolfo, Achille Iolascon, Hanspeter Rottensteiner, Herbert Gritsch, Friedrich Scheiflinger, Werner Hoellriegl, Lucia De Franceschi
Publikováno v:
Haematologica, Vol 107, Iss 11 (2022)
Sickle cell disease (SCD) is an inherited red blood cell disorder that occurs worldwide. Acute vaso-occlusive crisis is the main cause of hospitalization in patients with SCD. There is growing evidence that inflammatory vasculopathy plays a key role
Externí odkaz:
https://doaj.org/article/8c70e0b3cd1f416b96b51f9736f32503
Autor:
Agostina Carestia, Seok-Joo Kim, Franziska Horling, Hanspeter Rottensteiner, Christian Lubich, Birgit M. Reipert, Brian A. Crowe, Craig N. Jenne
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 95-108 (2021)
Adeno-associated viruses (AAVs) are emerging as one of the vehicles of choice for gene therapy. However, the potential immunogenicity of these vectors is a major limitation of their use, leading to the necessity of a better understanding of how viral
Externí odkaz:
https://doaj.org/article/f9b06ca4620a45d3afde7ccb0fe02637
Autor:
Wenjing Cao, Biao Dong, Franziska Horling, Jenni A. Firrman, Johannes Lengler, Matthias Klugmann, Maurus de la Rosa, Wenman Wu, Qizhao Wang, Hongying Wei, Andrea R. Moore, Sean A. Roberts, Carmen J. Booth, Werner Hoellriegl, Dong Li, Barbara Konkle, Carol Miao, Birgit M. Reipert, Friedrich Scheiflinger, Hanspeter Rottensteiner, Weidong Xiao
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss , Pp 486-495 (2020)
One important limitation for achieving therapeutic expression of human factor VIII (FVIII) in hemophilia A gene therapy is inefficient secretion of the FVIII protein. Substitution of five amino acids in the A1 domain of human FVIII with the correspon
Externí odkaz:
https://doaj.org/article/db9d4486e2234e9ab30e776cd2f5ab10
Autor:
Johannes Lengler, Sogue Coulibaly, Bernadette Gruber, Reinhard Ilk, Josef Mayrhofer, Friedrich Scheiflinger, Werner Hoellriegl, Falko G. Falkner, Hanspeter Rottensteiner
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 581-588 (2020)
Gene therapy product release requires reliable and consistent demonstration of biopotency. In hemophilia B vectors, this is usually determined in vivo by measuring the plasma levels of the expressed human factor IX (FIX) transgene product in FIX knoc
Externí odkaz:
https://doaj.org/article/385fb00f577f4457ac3b71706cbf6f70
Autor:
Alfred Weber, Andrea Engelmaier, Dirk Voelkel, Robert Pachlinger, Friedrich Scheiflinger, Paul E. Monahan, Hanspeter Rottensteiner
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 29-37 (2018)
The description of hyper-functional factor IX (FIX) Padua triggered the development of BAX 335, an AAV8-based hemophilia B gene therapy vector designed to compensate for low FIX protein expression levels by expressing the FIX Padua variant, thereby r
Externí odkaz:
https://doaj.org/article/fa9ae81b998a4cfb8406bb20995b7a98
Autor:
Louis Deforche, Claudia Tersteeg, Elien Roose, Aline Vandenbulcke, Nele Vandeputte, Inge Pareyn, Elien De Cock, Hanspeter Rottensteiner, Hans Deckmyn, Simon F De Meyer, Karen Vanhoorelbeke
Publikováno v:
PLoS ONE, Vol 11, Iss 8, p e0160388 (2016)
Thrombotic thrombocytopenic purpura (TTP) is a life-threatening thrombotic microangiopathy linked to a deficiency in the metalloprotease ADAMTS13. In the current study, a novel mouse model for acquired TTP was generated to facilitate development and
Externí odkaz:
https://doaj.org/article/f1966b3c66014898b88b297e085fb9e8
Autor:
Rana Grillberger, Veronica C. Casina, Peter L. Turecek, X. Long Zheng, Hanspeter Rottensteiner, Friedrich Scheiflinger
Publikováno v:
Haematologica, Vol 99, Iss 4 (2014)
Externí odkaz:
https://doaj.org/article/a046d4a26d2c4da9a23b86a3c3bf1126
Autor:
Silvia Ferrari, Kristina Palavra, Bernadette Gruber, Johanna A. Kremer Hovinga, Paul Knöbl, Claudine Caron, Caroline Cromwell, Louis Aledort, Barbara Plaimauer, Peter L. Turecek, Hanspeter Rottensteiner, Friedrich Scheiflinger
Publikováno v:
Haematologica, Vol 99, Iss 4 (2014)
Anti-ADAMTS13 autoantibodies are the main cause of acquired thrombotic thrombocytopenic purpura. Binding of these antibodies to ADAMTS13 eventually results in the formation of antigen-antibody immune complexes. Circulating ADAMTS13-specific immune co
Externí odkaz:
https://doaj.org/article/6088089c03124975a2c1d1ed3b9c8981
Autor:
Ygal Benhamou, Cyrielle Assié, Pierre-Yves Boelle, Marc Buffet, Rana Grillberger, Sandrine Malot, Alain Wynckel, Claire Presne, Gabriel Choukroun, Pascale Poullin, François Provôt, Didier Gruson, Mohamed Hamidou, Dominique Bordessoule, Jacques Pourrat, Jean-Paul Mira, Véronique Le Guern, Claire Pouteil-Noble, Cédric Daubin, Philippe Vanhille, Eric Rondeau, Jean-Bernard Palcoux, Christiane Mousson, Cécile Vigneau, Guy Bonmarchand, Bertrand Guidet, Lionel Galicier, Elie Azoulay, Hanspeter Rottensteiner, Agnès Veyradier, Paul Coppo
Publikováno v:
Haematologica, Vol 97, Iss 8 (2012)
Background Acquired thrombotic thrombocytopenic purpura is still associated with a 10–20% death rate. It has still not been possible to clearly identify early prognostic factors of death. This study involved thrombotic thrombocytopenic purpura pati
Externí odkaz:
https://doaj.org/article/98254f5b30564a27b002a550e1650bc6
Autor:
Bruce Ewenstein, Maurus de la Rosa, Barbara A. Konkle, Ivan Bilic, Birgit M. Reipert, Paul E. Monahan, Scott W.J. McPhee, R. Jude Samulski, Hanspeter Rottensteiner, Miguel A. Escobar, Neil C. Josephson, John C. Chapin, Friedrich Scheiflinger, Annette von Drygalski, Guy Young, Christopher E. Walsh
Publikováno v:
Blood
Gene therapy has the potential to maintain therapeutic blood clotting factor IX (FIX) levels in patients with hemophilia B by delivering a functional human F9 gene into liver cells. This phase 1/2, open-label dose-escalation study investigated BAX 33