Article

Selection of Adeno-Associated Virus Serotypes For Gene Therapy In Krabbe Patient IPSCs-Derived NSCs

2022-03-07

Abstract excerpt

Krabbe disease is caused by the mutation or deficiency of galactocerebrosidase (GALC) enzyme, which is located in the lysosome and hydrolyzes the galactolipid substrates like psychosine. Psychosine would accumulate abnormally in the myelin forming cells and result in demyelination in the nervous systems with the clinical symptoms of spastic paraparesis and seizures. Adeno-associated virus (AAV) is a well-establish...

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Literature Corpus work: 1ea4304e-e2d6-5e0f-b545-34055754a19b
DOI: 10.21203/rs.3.rs-1416247/v1

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