Methods for treating inherited eye defects
AssigneeThe Regents of the University of California
InventorStephanie Cherqui
Life of this patent
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Abstract
Provided herein are methods for treating an inherited eye disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject's eyes in need of treatment. Also provided are vectors containing the nucleic acid molecule.
Claims30
A method of treating an inherited eye disease or disorder in a subject comprising:
- introducing a corresponding functional human protein associated with the inherited eye disease or disorder into hematopoietic stem and progenitor cells (HSPCs) of the subject;
- and
- transplanting the HSPCs into an eye of the subject, thereby treating the inherited eye disease or disorder.
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A method of treating or ameliorating an inherited eye disease or disorder in a subject comprising:
- isolating hematopoietic stem and progenitor cells (HSPCs) from blood or bone marrow of the subject;
- introducing a functional human gene into the HSPCs, wherein the gene encodes a protein corresponding to the inherited eye disease or disorder;
- and
- transplanting the HSPCs back into an eye of the subject, thereby treating or ameliorating the lysosomal protein disease or disorder.
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A method of treating or ameliorating an inherited eye disease or disorder in a subject comprising:
- producing a functional human gene associated with the inherited eye disease or disorder in the subject using a gene editing system.
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A method of treating or ameliorating an inherited eye disease or disorder in a subject comprising contacting cells expressing a defective protein associated with the inherited eye disease or disorder from the subject with a vector encoding a gene editing system that, when transfected into the cells, corrects a mutation of an endogenous gene encoding the defective protein, thereby treating the inherited eye disease or disorder.
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Dependencies are inferred from the claim text.