Publications
Below you can find the list of GetRadi publications:
2024
1) Towards a Cure for Diamond–Blackfan Anemia: Views on Gene Therapy.
Matilde Vale, Jan Prochazka, Radislav Sedlacek. Cells, https://doi.org/10.3390/cells13110920
2) Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets.
Maryam Taghdiri, Claudio Mussolino. International Journal of Molecular Sciences, https://doi.org/10.3390/ijms25137333
3) Regulation of Precise DNA Repair by Nuclear Actin Polymerization: A Chance for Improving Gene Therapy?
Xiubin He, Cord Brakebusch. Cells, https://doi.org/10.3390/cells13131093
4) Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction.
Aurora Giommetti, Eleni Papanikolaou. Cells, https://doi.org/10.3390/cells13121039
5) Selection-free precise gene repair using high-capacity adenovector delivery of advanced prime editing systems rescues dystrophin synthesis in DMD muscle cells.
Wang, Qian; Capelletti, Sabrina; Liu, Jin; Janssen, Josephine; Goncalves, Manuel.
Nucleic Acid Research, https://doi.org/10.1093/nar/gkae057
6) On RNA-programmable gene modulation as a versatile set of principles targeting muscular dystrophies.
Sabrina Capelletti, Sofía C. García Soto, Manuel A.F.V. Gonçalves.
Molecular Therapy, https://doi.org/10.1016/j.ymthe.2024.08.016
7) Novel human neurodevelopmental and neurodegenerative disease associated with IRF2BPL gene variants—mechanisms and therapeutic avenues.
Daniel Bauersachs, Louise Bomholtz, Sara del Rey Mateos, Ralf Kühn and Pawel Lisowski.
Frontiers in Neuroscience, https://doi.org/10.3389/fnins.2024.1426177
8) Reporter Mice for Gene Editing: A Key Tool for Advancing Gene Therapy of Rare Diseases.
Siang Li, Cord Brakebusch