AAV-SPL 2.0, a Modified Adeno-Associated Virus Gene Therapy Agent for the Treatment of Sphingosine Phosphate Lyase Insufficiency Syndrome

AAV-SPL 2.0, a Modified Adeno-Associated Virus Gene Therapy Agent for the Treatment of Sphingosine Phosphate Lyase Insufficiency Syndrome

Sphingosine-1-phosphate lyase insufficiency syndrome (SPLIS) is an inborn error of metabolism caused by inactivating mutations in <i>SGPL1</i>, the gene encoding sphingosine-1-phosphate lyase (SPL), an essential enzyme needed to degrade sphingolipids. SPLIS features include glomeruloscle...

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Bibliographic Details
Main Authors: Ranjha Khan, Babak Oskouian, Joanna Y. Lee, Jeffrey B. Hodgin, Yingbao Yang, Gizachew Tassew, Julie D. Saba
Format: Article
Language:English
Published: MDPI AG 2023-10-01
Series:International Journal of Molecular Sciences
Subjects:
sphingosine phosphate lyase insufficiency syndrome
<i>Sgpl1</i>
sphingolipid
gene therapy
AAV-SPL 2.0
adeno-associated virus
Online Access:https://www.mdpi.com/1422-0067/24/21/15560

Internet

https://www.mdpi.com/1422-0067/24/21/15560

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