Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency

Lysosomal acid lipase deficiency (LAL-D) presents as one of two rare autosomal recessive diseases: Wolman disease (WD), a severe disorder presenting in infancy characterized by absent or very low LAL activity, and cholesteryl ester storage disease (CESD), a less severe, later onset disease form. Rec...

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Detalles Bibliográficos
Autores principales: Lam, Patricia, Ashbrook, Anna, Zygmunt, Deborah A., Yan, Cong, Du, Hong, Martin, Paul T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9403906/
https://www.ncbi.nlm.nih.gov/pubmed/36092360
http://dx.doi.org/10.1016/j.omtm.2022.08.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9403906/
https://www.ncbi.nlm.nih.gov/pubmed/36092360
http://dx.doi.org/10.1016/j.omtm.2022.08.001

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