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 SGPL1, the gene encoding sphingosine-1-phosphate lyase (SPL), an essential enzyme needed to degrade sphingolipids. SPLIS features include glomerulosclerosis, adrenal insu...

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Detalles Bibliográficos
Autores principales: Khan, Ranjha, Oskouian, Babak, Lee, Joanna Y., Hodgin, Jeffrey B., Yang, Yingbao, Tassew, Gizachew, Saba, Julie D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10648410/
https://www.ncbi.nlm.nih.gov/pubmed/37958544
http://dx.doi.org/10.3390/ijms242115560

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10648410/
https://www.ncbi.nlm.nih.gov/pubmed/37958544
http://dx.doi.org/10.3390/ijms242115560

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