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

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

Abstract Background Lysosomal acid lipase deficiency (LAL-D) is an autosomal recessive disorder caused by mutations in the LIPA gene, which results in lipid accumulation leading to multi-organ failure. If left untreated, the severe form of LAL-D results in premature death within the first year of li...

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Main Authors: Marine Laurent, Rim Harb, Christine Jenny, Julie Oustelandt, Simon Jimenez, Jeremie Cosette, Francesca Landini, Aristide Ferrante, Guillaume Corre, Nemanja Vujic, Claudia Piccoli, Anais Brassier, Laetitia Van Wittenberghe, Giuseppe Ronzitti, Dagmar Kratky, Consiglia Pacelli, Mario Amendola
Format: Article
Language:English
Published: Nature Portfolio 2025-04-01
Series:Communications Medicine
Online Access:https://doi.org/10.1038/s43856-025-00816-8
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https://doi.org/10.1038/s43856-025-00816-8

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