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Use of AAVrh10-GALC to Treat the Twitcher Mouse Model of Krabbe Disease

✍ Scribed by David Wenger; Mohammad Rafi; Paola Luzi; Han Zhi Rao


Book ID
116989658
Publisher
Elsevier Science
Year
2012
Tongue
English
Weight
53 KB
Volume
105
Category
Article
ISSN
1096-7192

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The inherited deficiency of galactosylceramide pgalactosidase (E.C. 3.2.1.46: galactocerebrosidase) activity results in globoid cell leukodystrophy in humans (Krabbe disease) and in mice (twitcher mutant). To determine whether Krabbe patients' cells complement twitcher cells to produce, in hybrid co