TREATMENT OF KRABBE DISEASE WITH UMBILICAL CORD BLOOD TRANSPLANTION (UCBT) AND INCREASED GALACTOCEREBROSIDASE (GALC) EXPRESSION
This application provides methods of treating Krabbe disease, for example in an infant. Such methods can include immunosuppressing the patient, for example by administration of a myeloablative regimen, administering an umbilical cord blood transplant (UCBT) (such as an allogenic UCBT), and increasin...
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Main Authors | , |
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Format | Patent |
Language | English French German |
Published |
27.11.2019
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Subjects | |
Online Access | Get full text |
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Summary: | This application provides methods of treating Krabbe disease, for example in an infant. Such methods can include immunosuppressing the patient, for example by administration of a myeloablative regimen, administering an umbilical cord blood transplant (UCBT) (such as an allogenic UCBT), and increasing expression of galactocerebrosidase (GALC) in the patient (e.g., by using gene editing). |
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Bibliography: | Application Number: EP20180741529 |