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US 2019/0336540 A1Published application
Treatment of krabbe disease with umbilical cord blood transplantion (ucbt) and increased galactocerebrosidase (galc) expression
AssigneeUniversity of Pittsburgh—Of the Commonwealth System of Higher Education
InventorsMaria Luisa Escolar, Paul Szabolcs
Life of this patent
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Abstract
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).
Claims20
A method of treating Krabbe disease in a subject, comprising:
- immunosuppressing the subject;
- administering a therapeutically effective amount of umbilical cord blood to the subject;
- and
- administering a therapeutically effective amount of a nucleic acid molecule encoding galactocerebrosidase (GALC) to the subject.
1 more element
A method of treating a genetic disease in a subject, comprising:
- partially or fully ablating bone marrow in the subject;
- administering a therapeutically effective amount of hematopoietic stem cells (HSCs) to the subject, and
- administering a therapeutically effective amount of a therapeutic nucleic acid molecule to the subject, wherein the nucleic acid molecule corrects the genetic disease.
Dependencies are inferred from the claim text.