Methods and compositions for cellular reprogramming
AssigneesYouhealth Biotech, Limited, The Regents of the University of California
InventorsKang ZHANG, Rui HOU, Gen LI
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Abstract
Disclosed herein are methods and pharmaceutical compositions for the treatment of retinitis pigmentosa, macular degeneration and other retinal conditions by interfering with expression of genes, such as those encoding photoreceptor cell-specific nuclear receptor and neural retina-specific leucine zipper protein, in cells of the eye. These methods and compositions employ nucleic acid based therapies.
Claims21
A method of re-programming a cell from a first cell type to a second cell type, comprising contacting the cell with:
- a) a guide RNA that hybridizes to a target site of a gene, wherein the gene encodes a protein that contributes to a cell type specific function of the cell;
- and
- b) a Cas nuclease, or polynucleotide encoding the Cas nuclease, wherein the Cas nuclease cleaves a strand of the gene at the target site,
- wherein cleaving the strand modifies expression of the gene such that the cell can no longer perform the cell type specific function, thereby re-programming the cell to the second cell type.
-7. (canceled)
A method of treating an eye condition comprising administering to a subject in need thereof:
- a) a first guide RNA that hybridizes to a target site of a gene in a first type of cell, wherein the gene encodes a protein that contributes to a first function of the first type of cell;
- and
- b) a Cas nuclease that cleaves a strand of the gene at the target site, wherein cleaving the strand modifies expression of the gene such that the first type of cell is switched from a first type of cell to a second type of cell, wherein a resulting presence or increase in the second type of cell improves the eye condition.
-30. (canceled)
Dependencies are inferred from the claim text.