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Targeting transgenic proteins to alpha granules for platelet-directed gene therapy

2022 . Woods, Vanessa M.A. | Rey, Lisette Johana Latorre | Schenk, Franziska | Rommel, Marcel | Moritz, Thomas | Modlich, Ute

Neugeborenenscreening auf spinale Muskelatrophie

2021 . Müller-Felber, W. | Vill, K. | Schwartz, O. | Blaschek, A. | Nennstiel, U. | Schara, U. | Hoffmann, G. F. | Gläser, D. | Becker, M. | Röschinger, W. | Bernert, G. | Klein, A. | Kölbel, H.

Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

Lentiviral and adeno-associated vectors efficiently transduce mouse T lymphocytes when targeted to murine CD8

2021 . Michels, Alexander | Frank, Annika Melanie | Günther, Dorothee M. | Mataei, Mehryad | Börner, Kathleen | Grimm, Dirk | Hartmann, Jessica | Buchholz, Christian

International Society for Extracellular Vesicles and International Society for Cell and Gene Therapy statement on extracellular vesicles from mesenchymal stromal cells and other cells: considerations for potential therapeutic agents to suppress coronavirus disease-19

Latest Advances for the Sleeping Beauty Transposon System: 23 Years of Insomnia but Prettier than Ever: Refinement and Recent Innovations of the Sleeping Beauty Transposon System Enabling Novel, Nonviral Genetic Engineering Applications

2020 . Amberger, Maximilian | Ivics, Zoltan

RNA-guided retargeting of Sleeping Beauty transposition in human cells

2020 . Kovač, Adrian | Miskey, Csaba | Menzel, Michael | Grueso, Esther | Gogol-Döring, Andreas | Ivics, Zoltan

Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells

2020 . Bittorf, Patrick | Bergmann, Thorsten | Merlin, Simone | olgasi, cristina | Pullig, Oliver | Sanzenbacher, Ralf | Zierau, Martin | Walles, Heike | Follenzi, Antonia | Braspenning, Joris

Genspezifische Therapieansätze bei der Alzheimer-Krankheit und anderen Tauopathien

2020 . Feneberg, E. | Otto, M.

Arzneimittel für neuartige Therapien – Perspektiven, Chancen, Herausforderungen

2020 . Rascher, Wolfgang | Klingebiel, Thomas | Herting, Egbert | Hoffmann, Georg F. | Berner, Reinhard | Krägeloh-Mann, Ingeborg | Gärtner, Jutta | Zepp, Fred | Schara, Ulrike