Back to results
US 12,599,636 B2Granted
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
Estimating the term…
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).
Claims39
A method of treating Krabbe disease in a human subject, comprising:
- immunosuppressing the human subject;
- administering a therapeutically effective amount of human umbilical cord blood to the human subject;
- and
- administering a therapeutically effective amount of a nucleic acid molecule encoding galactocerebrosidase (GALC) to the human subject,
- wherein the nucleic acid molecule encodes a GALC protein comprising at least 95% sequence identity to SEQ ID NO:
- 2 or 6, and
- wherein the umbilical cord blood is administered at least 3 weeks prior to the nucleic acid molecule encoding GALC.
4 more elements
Dependencies are inferred from the claim text.