Gene Therapy of Rare Diseases

Project Information

GetRadi

Grant agreement ID: 101072427

DOI

10.3030/101072427

EC signature date 15 July 2022

Start date 1 September 2022

End date 31 August 2026

Funded under

• Marie Skłodowska-Curie Actions (MSCA)

Total cost

No data

EU contribution

€ 2 785 809,60

Coordinated by

KOBENHAVNS UNIVERSITET
Denmark

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Deliverables

Documents, reports (2)

Review article submitted by ESR10UCPH

Review article submitted

Review article submitted by ESR6 (MDC)

Review article submitted

Websites, patent fillings, videos etc. (1)

Project Website

Project website (M2)

Publications

Peer reviewed articles (8)

On RNA-programmable gene modulation as a versatile set of principles targeting muscular dystrophies

Author(s): Sabrina Capelletti, Sofía C. García Soto, Manuel A.F.V. Gonçalves
Published in: Molecular Therapy, 2024, ISSN 1525-0016
Publisher: Elsevier Inc.
DOI: 10.1016/j.ymthe.2024.08.016

Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets

Author(s): Maryam Taghdiri, Claudio Mussolino
Published in: Int. J. Mol. Sci., 2024, ISSN 1422-0067
Publisher: MDPI
DOI: 10.3390/ijms25137333

Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction

Author(s): Aurora Giommetti, Eleni Papanikolaou
Published in: Cells, 2024, ISSN 2073-4409
Publisher: MDPI
DOI: 10.3390/cells13121039

Reporter Mice for Gene Editing: A Key Tool for Advancing Gene Therapy of Rare Diseases

Author(s): Siang Li, Cord Brakebusch
Published in: Cells, 2024, ISSN 2073-4409
Publisher: MDPI
DOI: 10.3390/cells13171508

Selection-free precise gene repair using high-capacity adenovector delivery of advanced prime editing systems rescues dystrophin synthesis in DMD muscle cells

Author(s): Wang, Qian; Capelletti, Sabrina; Liu, Jin`; Janssen, Josephine; Goncalves, Manuel
Published in: Nucleic Acid Research, 2024, ISSN 1362-4962
Publisher: Oxford University Press
DOI: 10.1093/nar/gkae057

Regulation of Precise DNA Repair by Nuclear Actin Polymerization: A Chance for Improving Gene Therapy?

Author(s): Xiubin He, Cord Brakebusch
Published in: Cells, 2024, ISSN 2073-4409
Publisher: MDPI
DOI: 10.3390/cells13131093

Novel human neurodevelopmental and neurodegenerative disease associated with IRF2BPL gene variants—mechanisms and therapeutic avenues

Author(s): Daniel Bauersachs, Louise Bomholtz, Sara del Rey Mateos, Ralf Kühn and Pawel Lisowski
Published in: Frontiers in Neuroscience, 2024, ISSN 1662-4548
Publisher: Frontiers Media SA
DOI: 10.3389/fnins.2024.1426177

Towards a Cure for Diamond–Blackfan Anemia: Views on Gene Therapy

Author(s): Matilde Vale, Jan Prochazka, Radislav Sedlacek
Published in: Cells, 2024, ISSN 2073-4409
Publisher: MDPI
DOI: 10.3390/CELLS13110920