Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome-Reference-Cited by-同舟云学术

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

Published:2021-04-22 Issue:8 Volume:6 Page:
ISSN:2379-3708
Container-title:JCI Insight
language:en
Short-container-title:

Author:

Zhao Piming,Tassew Gizachew B.,Lee Joanna Y.,Oskouian Babak,Muñoz Denise P.^ORCID,Hodgin Jeffrey B.^ORCID,Watson Gordon L.,Tang Felicia,Wang Jen-Yeu^ORCID,Luo Jinghui,Yang Yingbao,King Sarah,Krauss Ronald M.,Keller Nancy,Saba Julie D.

Funder

National Institutes of Health

Swim Across America Foundation

Ultragenyx Pharmaceutical

NICHD/NIH

funding sources listed below remain accurate

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Link

https://insight.jci.org/articles/view/145936/files/pdf

Reference66 articles.

1. Sphingosine phosphate lyase insufficiency syndrome (SPLIS): a novel inborn error of sphingolipid metabolism;Choi;Adv Biol Regul,2019

2. A perilous path: the inborn errors of sphingolipid metabolism

3. Mutations in sphingosine-1-phosphate lyase cause nephrosis with ichthyosis and adrenal insufficiency

4. Sphingosine-1-phosphate lyase mutations cause primary adrenal insufficiency and steroid-resistant nephrotic syndrome

5. Deficiency of the sphingosine-1-phosphate lyase SGPL1 is associated with congenital nephrotic syndrome and congenital adrenal calcifications

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