Fachrepositorium Lebenswissenschaften

8 Treffer

Suche

Publisso-Menue

A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient

2024 . Miesbach, Wolfgang | Mulders, Greta | Breederveld, Daan | Pinachyan, Karen | Le Quellec, Sandra | Pabinger, Ingrid

Towards ethical drug pricing: the European Orphan Genomic Therapies Fund

2024 . Risse, Johanna | Krzemien, Merlin | Schnalke, Jan | Heinemann, Thomas

Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells

2024 . Renner, Alexander | Stahringer, Anika | Ruppel, Katharina Eva | Fricke, Stephan | Koehl, Ulrike | Schmiedel, Dominik

An algorithm for discontinuing mechanical ventilation in boys with x-linked myotubular myopathy after positive response to gene therapy: the ASPIRO experience

2024 . Graham, Robert J. | Amin, Reshma | Demirel, Nadir | Edel, Lisa | Lilien, Charlotte | MacBean, Victoria | Rafferty, Gerrard F. | Sawnani, Hemant | Schön, Carola | Smith, Barbara K. | Syed, Faiza | Sarazen, Micaela | Prasad, Suyash | Rico, Salvador | Perez, Geovanny F.

Targeted cytokine delivery: cell therapy to remodel the pre-metastatic niche

2021 . Morgan, Michael A. | Lange, Lucas | Schambach, Axel

Targeting the undruggable: exploiting neomorphic features of fusion oncoproteins in childhood sarcomas for innovative therapies

2020 . Knott, Maximilian M. L. | Hölting, Tilman L. B. | Ohmura, Shunya | Kirchner, Thomas | Cidre-Aranaz, Florencia | Grünewald, Thomas G. P.

Comprehensive genomic characterization of gene therapy-induced T-cell acute lymphoblastic leukemia

Emerging Targeted Therapeutics for Genetic Subtypes of Parkinsonism

2020 . Schneider, Susanne A. | Hizli, Baccara | Alcalay, Roy N.