Genetics in Medicine Open (Jan 2024)
P135: X-linked Alport syndrome: From transcriptomic diagnosis to preclinical assessment of splice-switching oligonucleotide therapy using patient-derived cells and kidney organoids*
- Hassan Saei,
- Marie Boisson,
- Christelle Arrondel,
- Bruno Estebe,
- Nicolas Cagnard,
- Marc Bras,
- Vincent Morinière,
- Zaïna Aït Arkoub,
- Laurence Heidet,
- Olivier Gribouval,
- Patrick Nitschké,
- Corinne Antignac,
- Géraldine Mollet,
- Guillaume Dorval
Affiliations
- Hassan Saei
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Marie Boisson
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Christelle Arrondel
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Bruno Estebe
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Nicolas Cagnard
- Bioinformatics Platform, Imagine Institute, Paris, France
- Marc Bras
- Bioinformatics Platform, Imagine Institute, Paris, France
- Vincent Morinière
- Medical Genetics Services for Rare Diseases, Necker Children Hospital, Paris, France
- Zaïna Aït Arkoub
- Medical Genetics Services for Rare Diseases, Necker Children Hospital, Paris, France
- Laurence Heidet
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Olivier Gribouval
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Patrick Nitschké
- Bioinformatics Platform, Imagine Institute, Paris, France
- Corinne Antignac
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Géraldine Mollet
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Guillaume Dorval
- Laboratory of Hereditary Kidney Diseases, Necker Branch, INSERM UMR1163, Imagine Institute, Paris, France
- Journal volume & issue
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Vol. 2
p. 101032