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Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo

2021 . Janosz, Ewa | Hetzel, Miriam | Spielmann, Hanna | Tumpara, Srinu | Rossdam, Charlotte | Schwabbauer, Marc | Kloos, Doreen | von Kaisenberg, Constantin | Schambach, Axel | Buettner, Falk | Janciauskiene, Sabina | Lachmann, Nico | Moritz, Thomas

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.

Analysis of imaging biomarkers and retinal nerve fiber layer thickness in RPGR-associated retinitis pigmentosa

2021 . Birtel, Theresa H. | Birtel, Johannes | Hess, Kristina | Clemens, Amelie C. | Lindner, Moritz | Herrmann, Philipp | Holz, Frank G. | Gliem, Martin

Low efficacy of recombinant SV40 in Ugt1a1-/- mice with severe inherited hyperbilirubinemia

2021 . Shi, Xiaoxia | Bortolussi, Giulia | Bloemendaal, Lysbeth ten | Duijst, Suzanne | Muro, Andrés F. | Bosma, Piter J.

How to package and SEND mRNA: a novel “humanized” vector system based on endogenous retroviruses

2021 . Riecken, Kristoffer | Głów, Dawid | Fehse, Boris

Spotlight on gene therapy in Germany

2021 . Mussolino, Claudio | Harrison, Patrick

Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells

2021 . Mosti, Laura | Langner, Lukas Matthias | Chmielewski, Kay O. | Arbuthnot, Patrick | Alzubi, Jamal | Cathomen, Toni

Implications of hematopoietic stem cells heterogeneity for gene therapies

2021 . Epah, Jeremy | Schäfer, Richard

Development of an AAV9-RNAi-mediated silencing strategy to abrogate TRPM4 expression in the adult heart

2021 . Medert, Rebekka | Jungmann, Andreas | Hildebrand, Staffan | Busch, Martin | Grimm, Dirk | Flockerzi, Veit | Müller, Oliver J. | Most, Patrick | Schumacher, Dagmar | Freichel, Marc

Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery

2020 . De Masi, Claudia | Spitalieri, Paola | Murdocca, Michela | Novelli, Giuseppe | sangiuolo, federica

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