Zobrazeno 1 - 7
of 7
pro vyhledávání: '"Chiara Krämer"'
Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy
Autor:
Markus Grosch, Laura Schraft, Adrian Chan, Leonie Küchenhoff, Kleopatra Rapti, Anne-Maud Ferreira, Julia Kornienko, Shengdi Li, Michael H. Radke, Chiara Krämer, Sandra Clauder-Münster, Emerald Perlas, Johannes Backs, Michael Gotthardt, Christoph Dieterich, Maarten M. G. van den Hoogenhof, Dirk Grimm, Lars M. Steinmetz
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-15 (2023)
Abstract Dilated cardiomyopathy is the second most common cause for heart failure with no cure except a high-risk heart transplantation. Approximately 30% of patients harbor heritable mutations which are amenable to CRISPR-based gene therapy. However
Externí odkaz:
https://doaj.org/article/287d1894c5f548b689b58cdf403063c5
Autor:
Dirk Grimm, Ellen Wiedtke, Chiara Krämer, Emma Gerstmann, Jixin Liu, Felix Bubeck, Margarita Zayas, Joanna Szumska, Jonas Becker, Olena Maiakovska, Kleopatra Rapti
Publikováno v:
Journal of Visualized Experiments.
Gene delivery vectors derived from Adeno-associated virus (AAV) are one of the most promising tools for the treatment of genetic diseases, evidenced by encouraging clinical data and the approval of several AAV gene therapies. Two major reasons for th
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7d49b499bcfc6eae98bab46f952222b7
https://doi.org/10.3791/64389
https://doi.org/10.3791/64389
Publikováno v:
Methods in molecular biology (Clifton, N.J.). 2173
Optogenetics, that is, the use of photoswitchable/-activatable moieties to precisely control or monitor the activity of cells and genes at unprecedented spatiotemporal resolution, holds tremendous promise for a wide array of applications in fundament
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::901e0dec22c9025efafb47ec568b143e
https://pubmed.ncbi.nlm.nih.gov/32651911
https://pubmed.ncbi.nlm.nih.gov/32651911
Publikováno v:
Methods in Molecular Biology ISBN: 9781071607541
Optogenetics, that is, the use of photoswitchable/-activatable moieties to precisely control or monitor the activity of cells and genes at unprecedented spatiotemporal resolution, holds tremendous promise for a wide array of applications in fundament
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::9e0d30d50d84d6095d2c3a5c92b08489
https://doi.org/10.1007/978-1-0716-0755-8_5
https://doi.org/10.1007/978-1-0716-0755-8_5
Autor:
Kathleen Börner, Manuel Gunkel, Chiara Krämer, Anne-Kathrin Herrmann, Dirk Grimm, Stefanie Große, Ellen Wiedtke
Publikováno v:
Human gene therapy. 30(1)
Over the last decade, the role of the assembly-activating protein (AAP) has begun to be dissected for the formation of adeno-associated virus (AAV) capsids based on different viral serotypes. Recently, the authors' group has specifically studied AAP'
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::65613d778469caa452c4192f801d6261
https://pubmed.ncbi.nlm.nih.gov/29978729
https://pubmed.ncbi.nlm.nih.gov/29978729
Autor:
Manuel Gunkel, Kathleen Börner, Stefanie Grosse, Lucie Ménard, Ellen Wiedtke, Eduard Ayuso, Chiara Krämer, Julia Fakhiri, Anne-Kathrin Herrmann, Magalie Penaud-Budloo, Dirk Grimm, Vibor Laketa
Publikováno v:
Journal of Virology
Journal of Virology, American Society for Microbiology, 2017, 91 (20), pp.275-281. ⟨10.1128/JVI.01198-17⟩
Journal of Virology, American Society for Microbiology, 2017, 91 (20), pp.275-281. ⟨10.1128/JVI.01198-17⟩
The discovery that adeno-associated virus 2 (AAV2) encodes an eighth protein, called assembly-activating protein (AAP), transformed our understanding of wild-type AAV biology. Concurrently, it raised questions about the role of AAP during production
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5d39f5029758db2cc96d7ee7a2d0698d
https://www.hal.inserm.fr/inserm-01785930/document
https://www.hal.inserm.fr/inserm-01785930/document
Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy
Autor:
Markus Grosch, Laura Schraft, Adrian Chan, Leonie Küchenhoff, Kleopatra Rapti, Anne-Maud Ferreira, Julia Kornienko, Shengdi Li, Michael H Radke, Chiara Krämer, Sandra Clauder-Münster, Emerald Perlas, Michael Gotthardt, Christoph Dieterich, Maarten MG van den Hoogenhof, Dirk Grimm, Lars M Steinmetz
Publikováno v:
bioRxiv
Dilated cardiomyopathy (DCM) is the second most common cause for heart failure with no cure except a high-risk heart transplantation. Approximately 30% of DCM patients harbor heritable mutations which are amenable to CRISPR-based gene therapy1. Howev
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::29e407e94e11bc1fe6a614b3a1046d8e