Projects funded by the NCN
Miniature CRISPR/Cas nucleases for upregulation of utrophin expression: a novel approach for mutation-independent therapy of Duchenne muscular dystrophy
2024/52/L/NZ3/00142
Keywords:
Duchenne muscular dystrophy CRISPR/Cas dystrophin utrophin induced pluripotent stem cells iPSC cardiomyocytes 3D organoids gene therapy cardiomyopathy
Descriptors:
- NZ3_08:
- NZ1_05:
- NZ1_01:
Panel:
NZ3 - Cellular and developmental biology: cell biology, developmental biology, ageing biology, neurobiology
Host institution :
Uniwersytet Jagielloński
woj. małopolskie
Principal investigator (from the host institution):
Number of co-investigators in the project: 6
Call: DAINA 3 - announced on 2023-10-17
Amount awarded: 1 000 000 PLN
Project start date (Y-m-d): 2025-01-02
Project end date (Y-m-d): 2028-01-01
Project duration:: 36 months (the same as in the proposal)
Project status: Pending project
Project description
Download the project description in a pdf file
Note - project descriptions were prepared by the authors of the applications themselves and placed in the system in an unchanged form.
Information in the final report
- Publication in academic press/journals (2)
- Genetic strategies for therapy of Duchenne muscular dystrophy.Authors:Łoboda A, Chamberlain JS, Dulak JAcademic press:Molecular Therapy Nucleic Acidss (rok: 2025, tom: 36, strony: 2025 Oct 31;36(4):102759), Wydawca: Cell PressStatus:PublishedDOI:10.1016/j.omtn.2025.102759. - link to the publication
- Cell therapy for Duchenne muscular dystrophy: promises, challenges, and controversies.Authors:Łoboda A, Dulak J.Academic press:Cellular and Molecular Life Sciences (rok: 2025, tom: 82, strony: 356), Wydawca: SpringerStatus:PublishedDOI:10.1007/s00018-025-05904-5 - link to the publication