What is it about?
Progranulin (PGRN) plays important role in Gaucher disease. We developed a PGRN derivative can cross the blood brain barrier, penetrate to the brain tissue and rescue the neuropathic Gaucher disease and Parkinson’s disease phenotypes.
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Why is it important?
There is no clinic drugs for treating neuropathic Gaucher disease because they cannot cross the blood brain barrier. The PGRN biologic which can cross blood brain barrier, and protects against neuropathic Gaucher disease and Parkinson’s disease phenotypes.
Perspectives
The PGRN biologic we developed provide a potential treatment for rare lysosomal storage diseases and common neurodegenerative disorders, particularly neuropathic Gaucher disease and Parkinson’s disease.
Dr. Xiangli Zhao
Read the Original
This page is a summary of: PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects,
GBA1
mutation-associated pathologies and diseases, Proceedings of the National Academy of Sciences, December 2022, Proceedings of the National Academy of Sciences,
DOI: 10.1073/pnas.2210442120.
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