Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?
Introduction: Krabbe disease (KD) is an autosomal recessive disorder caused by mutations in the galactocerebrosidase (GALC) gene resulting in neuro-inflammation and defective myelination in the central and peripheral nervous systems. Most infantile patients present with clinical features before six...
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Tabriz University of Medical Sciences
2021-03-01
|
| Series: | BioImpacts |
| Subjects: | |
| Online Access: | https://bi.tbzmed.ac.ir/PDF/bi-11-135.pdf |