0601094 - ÚMG 2025 RIV CH eng J - Journal Article
Augusto Vale, Ana Matilde - Procházka, Jan - Sedláček, Radislav
Towards a Cure for Diamond-Blackfan Anemia: Views on Gene Therapy.
Cells. Roč. 13, č. 11 (2024), č. článku 920. ISSN 2073-4409. E-ISSN 2073-4409
R&D Projects: GA MŠMT LM2023036
EU Projects: European Commission(DK) 101072427 - GetRadi
Grant - others:AV ČR(CZ) StrategieAV21/29
Program: StrategieAV
Institutional support: RVO:68378050
Keywords : bone-marrow failure * lentiviral vectors * mouse model * rps19 * cell * challenges * deficiency * Diamond-Blackfan anemia * ribosomopathy * ribosomal protein genes * rare genetic disorder * hematopoietic stem cell transplantation * gene therapy * lentiviral vector * non-integrating lentiviral vector * CRISPR/Cas9
OECD category: Biochemistry and molecular biology
Impact factor: 5.1, year: 2023 ; AIS: 1.34, rok: 2023
Method of publishing: Open access
Result website:
https://www.mdpi.com/2073-4409/13/11/920DOI: https://doi.org/10.3390/cells13110920
Permanent Link: https://hdl.handle.net/11104/0358307