Author

Laetitia van Wittenberghe

Genethon (France)

Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.

Biology Disciplines

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Molecular Biology· 1 Physiology· 1

Signature Assays, Organisms & Methods

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Assay:bulk RNA-seq· 2 Research Focus:differential expression· 2 Research Focus:drug response / drug discovery· 2 Approach:experimental assay· 2 Organism:mouse· 2 Research Focus:cell type annotation· 1 Research Focus:gene regulatory network inference· 1 Approach:statistical model· 1

Frequent Co-Authors

Abbass JaberiD· 1 shared Adeline Miranda· 1 shared Ai Vu HongiD· 1 shared Anaïs BrassieriD· 1 shared Aristide Ferrante· 1 shared Christine Jenny· 1 shared Cláudia PiccoliiD· 1 shared Consiglia PacelliiD· 1 shared

Activity by Year

2025-2025 · peak 2/yr

Co-Author Collaboration Network

10 top collaborators · 21 mutual co-author links

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1 shared paperPhysiology

Abbass JaberORCID

Mutual co-authors in cluster

Author profile →

2 papers

Recent·Most cited

Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer.

Marine Laurent, Rim Harb, Christine Jenny, Julie Oustelandt, Simon Jimenez, Jérémie Cosette, Francesca Landini, Aristide Ferrante, Guillaume Corre, Nemanja Vujić, Cláudia Piccoli, Anaïs Brassier, Laetitia van Wittenberghe, Giuseppe Ronzitti, Dagmar Kratky, , Mario Amendola

The paper develops a liver-targeted gene therapy using recombinant AAV8 to cure Lysosomal acid lipase deficiency by correcting lipid accumulation and normalizing gene expression and mitochondrial function in a mouse model.

Communications Medicine · Apr 11, 2025Peer-reviewedMolecular Biology

Targeting lysosomal damage is a new therapeutic perspective for Duchenne Muscular Dystrophy

Abbass Jaber, Laura Palmieri, Rania Bakour, Elise Lachiver, Ai Vu Hong, Nathalie Bourg, Jérôme Poupiot, Sonia Albini, Daniel Stockholm, Laetitia van Wittenberghe, Adeline Miranda, Nathalie Danièle, Inès Barthélémy, Stéphane Blot, Mai Thao Bui, , David Israeli

This study identifies lysosomal damage as a novel mechanism in Duchenne Muscular Dystrophy and shows that combining trehalose with gene therapy improves outcomes.

bioRxiv · Feb 11, 2025 · PreprintPhysiology