Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer.
Laurent M, Harb R, Jenny C, Oustelandt J, Jimenez S, Cosette J, Landini F, Ferrante A, Corre G, Vujic N, Piccoli C, Brassier A, Van Wittenberghe L, Ronzitti G, Kratky D, Pacelli C, Amendola M.
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Commun Med (Lond). 2025 Apr 11;5(1):110. doi: 10.1038/s43856-025-00816-8.
Commun Med (Lond). 2025.
PMID: 40216942
Free PMC article.