Zobrazeno 1 - 10
of 243
pro vyhledávání: '"Dirk Grimm"'
Autor:
Jannik T. Wagner, Sandra M. Müller-Schmucker, Wenjun Wang, Philipp Arnold, Nadja Uhlig, Leila Issmail, Valentina Eberlein, Dominik Damm, Kaveh Roshanbinfar, Armin Ensser, Friederike Oltmanns, Antonia Sophia Peter, Vladimir Temchura, Silke Schrödel, Felix B. Engel, Christian Thirion, Thomas Grunwald, Manfred Wuhrer, Dirk Grimm, Klaus Überla
Publikováno v:
Communications Biology, Vol 7, Iss 1, Pp 1-15 (2024)
Abstract Long-acting passive immunization strategies are needed to protect immunosuppressed vulnerable groups from infectious diseases. To further explore this concept for COVID-19, we constructed Adeno-associated viral (AAV) vectors encoding the hum
Externí odkaz:
https://doaj.org/article/fb0eb88696d04de18d534ac0c445bf7a
Autor:
Felix Bubeck, Dirk Grimm
Publikováno v:
PLoS Biology, Vol 22, Iss 7, p e3002637 (2024)
A new study characterizes and improves a novel small Cas12a variant before adapting it for in vivo genome editing by delivery via adeno-associated virus (AAV) vectors, showcasing the potential of small CRISPR systems and their compatibility with vira
Externí odkaz:
https://doaj.org/article/277b30738c9c4d65b9897d67e829d4d6
Autor:
Sergio Muñoz, Joan Bertolin, Veronica Jimenez, Maria Luisa Jaén, Miquel Garcia, Anna Pujol, Laia Vilà, Victor Sacristan, Elena Barbon, Giuseppe Ronzitti, Jihad El Andari, Warut Tulalamba, Quang Hong Pham, Jesus Ruberte, Thierry VandenDriessche, Marinee K. Chuah, Dirk Grimm, Federico Mingozzi, Fatima Bosch
Publikováno v:
Molecular Metabolism, Vol 81, Iss , Pp 101899- (2024)
Objective: Pompe disease (PD) is caused by deficiency of the lysosomal enzyme acid α-glucosidase (GAA), leading to progressive glycogen accumulation and severe myopathy with progressive muscle weakness. In the Infantile-Onset PD (IOPD), death genera
Externí odkaz:
https://doaj.org/article/51f4889c5f3c445fbc764529166584d8
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-16 (2023)
Abstract Molecular screens comparing different disease states to identify candidate genes rely on the availability of fast, reliable and multiplexable systems to interrogate genes of interest. CRISPR/Cas9-based reverse genetics is a promising method
Externí odkaz:
https://doaj.org/article/7fd74c1a9e8f44f68884782a4b513558
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 191-193 (2023)
Externí odkaz:
https://doaj.org/article/38dd70ad44884f35bf58c86bd5180984
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:
Alice Rovai, BoMee Chung, Qingluan Hu, Sebastian Hook, Qinggong Yuan, Tibor Kempf, Florian Schmidt, Dirk Grimm, Steven R. Talbot, Lars Steinbrück, Jasper Götting, Jens Bohne, Simon A. Krooss, Michael Ott
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-10 (2022)
Hemochromatosis is a metabolic disorder caused by mutations in the HFE gene. Here, the authors show that a single administration of AAV8 vectors expressing an Adenine Base Editor facilitates efficient in vivo gene correction in hepatocytes and leads
Externí odkaz:
https://doaj.org/article/41baa3360d14427b871c13b614264a6d
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 93-95 (2022)
Externí odkaz:
https://doaj.org/article/0046bc0d992b465b96cc77d74dd0d4c1
Autor:
Dirk Grimm, Mathieu Nonnenmacher
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 29, Iss , Pp 364-365 (2023)
Externí odkaz:
https://doaj.org/article/52ef8ed6413947b0a5838448080c3de2
Autor:
Lea Jessica Flitsch, Kathleen Börner, Christian Stüllein, Simon Ziegler, Vera Sonntag-Buck, Ellen Wiedtke, Vesselina Semkova, Si Wah Christina Au Yeung, Julia Schlee, Mohamad Hajo, Mona Mathews, Beatrice Stefanie Ludwig, Susanne Kossatz, Horst Kessler, Dirk Grimm, Oliver Brüstle
Publikováno v:
Scientific Reports, Vol 12, Iss 1, Pp 1-12 (2022)
Abstract Human brain cells generated by in vitro cell programming provide exciting prospects for disease modeling, drug discovery and cell therapy. These applications frequently require efficient and clinically compliant tools for genetic modificatio
Externí odkaz:
https://doaj.org/article/4875817cf7bc4d6497c93a0dff7c11d4