Zobrazeno 1 - 9
of 9
pro vyhledávání: '"Franziska Horling"'
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:
Friedrich Scheiflinger, Qizhao Wang, Sean Roberts, Biao Dong, Adrian R. Moore, Birgit M. Reipert, Weidong Xiao, Johannes Lengler, Maurus de la Rosa, Carmen J. Booth, Wenjing Cao, Wenman Wu, Dong Li, Franziska Horling, Carol H. Miao, Hanspeter Rottensteiner, Werner Hoellriegl, Hongying Wei, Matthias Klugmann, Jenni Firrman, Barbara Konkle
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss, Pp 486-495 (2020)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
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://explore.openaire.eu/search/publication?articleId=doi_dedup___::a67fa182441ec5d13d18eb3571877b28
http://www.sciencedirect.com/science/article/pii/S2329050120302175
http://www.sciencedirect.com/science/article/pii/S2329050120302175
Autor:
Birgit M. Reipert, Franziska Horling, Christian Lubich, Seok-Joo Kim, Agostina Carestia, Craig N. Jenne, Brian A. Crowe, Hanspeter Rottensteiner
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 95-108 (2021)
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://explore.openaire.eu/search/publication?articleId=doi_dedup___::b55368657361708e566edfdde0c9a5c2
http://europepmc.org/articles/PMC7750493
http://europepmc.org/articles/PMC7750493
Autor:
Biao Dong, Hanspeter Rottensteiner, Bagirath Gangadharan, Franziska Horling, Friedrich Scheiflinger, Wenjing Cao, Maurus de la Rosa, Werner Hoellriegl, Johannes Lengler, Birgit M. Reipert, Weidong Xiao
Publikováno v:
Blood. 134:3356-3356
Introduction. Adeno-associated virus (AAV)-based factor VIII (FVIII) gene therapy holds great promise to provide clinical benefit in patients with hemophilia A. However, very high doses are currently needed to achieve therapeutic factor levels and th
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::aa5f8bbf7805c1e83eae8bf72e540c21
https://doi.org/10.1182/blood-2019-126489
https://doi.org/10.1182/blood-2019-126489
Autor:
Rainer Schreiber, Andrea Milenkovic, Franziska Horling, Potchanart Wanitchakool, Caroline Brandl, Vladimir M. Milenkovic, Thomas Jendryke, Bernhard H. F. Weber, Stephanie Zimmermann, Karl Kunzelmann, Lalida Sirianant, Heinrich Schrewe, Charlotte Reiff, Christian H. Wetzel
Publikováno v:
Proceedings of the National Academy of Sciences of the United States of America
In response to cell swelling, volume-regulated anion channels (VRACs) participate in a process known as regulatory volume decrease (RVD). Only recently, first insight into the molecular identity of mammalian VRACs was obtained by the discovery of the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d3598880ccc0b6e0d73dd3e70dd26199
https://hdl.handle.net/11858/00-001M-0000-0028-92FB-011858/00-001M-0000-0028-92F9-3
https://hdl.handle.net/11858/00-001M-0000-0028-92FB-011858/00-001M-0000-0028-92F9-3
Publikováno v:
Cytogenetic and Genome Research. 115:35-44
Early schisis cavities in the retinal bipolar cell layer accompanied by progressive loss of cone and rod photoreceptor cells are the hallmark of the retinoschisin-deficient (Rs1h–/Y) murine retina. With this study we aimed at elucidating the molecu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5b2025a1f9c29ef3cc2d86d89760cd01
https://doi.org/10.1159/000094799
https://doi.org/10.1159/000094799
Autor:
Michael Bonin, Bernhard H. F. Weber, Andreas Janssen, Sven Poths, Michael Walter, Franziska Horling, Andrea Gehrig, Thomas Langmann
purpose. The Rs1h knockout mouse displays retinal features typical for X-linked juvenile retinoschisis (RS). Consequently, this mouse line represents an excellent model to study early molecular events in RS. methods. Whole genome expression profiling
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ee0afbdada71578d99c83d56b4099f94
Publikováno v:
The Journal of biological chemistry. 282(2)
The vitelliform macular dystrophy type 2 (VMD2) gene mutated in Best macular dystrophy encodes a 585-amino acid putative transmembrane protein termed bestrophin-1. The vast majority of known disease-associated alterations are of the missense type, wh
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7cdba9878cdfe19cbd93d358487f452e
https://pubmed.ncbi.nlm.nih.gov/17110374
https://pubmed.ncbi.nlm.nih.gov/17110374