Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome

Sphingosine-1-phosphate lyase insufficiency syndrome (SPLIS) is a rare metabolic disorder caused by inactivating mutations in sphingosine-1-phosphate lyase 1 (SGPL1), which is required for the final step of sphingolipid metabolism. SPLIS features include steroid-resistant nephrotic syndrome and impa...

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Detalles Bibliográficos
Autores principales: Zhao, Piming, Tassew, Gizachew B., Lee, Joanna Y., Oskouian, Babak, Muñoz, Denise P., Hodgin, Jeffrey B., Watson, Gordon L., Tang, Felicia, Wang, Jen-Yeu, Luo, Jinghui, Yang, Yingbao, King, Sarah, Krauss, Ronald M., Keller, Nancy, Saba, Julie D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8119223/
https://www.ncbi.nlm.nih.gov/pubmed/33755599
http://dx.doi.org/10.1172/jci.insight.145936

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8119223/
https://www.ncbi.nlm.nih.gov/pubmed/33755599
http://dx.doi.org/10.1172/jci.insight.145936

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