Methods of enhancing stem cell differentiation into beta cells
AssigneeVertex Pharmaceuticals Incorporated
InventorsFelicia Pagliuca, George Harb, Mads Gurtler, Austin Thiel, Jihad Yasin, Evrett Thompson
Life of this patent
Estimating the term…
Abstract
Disclosed herein are compositions and methods of enhancing stem cell differentiation into beta cells with use of one or more epigenetic modification compounds. The present disclosure also relates to compositions and methods of sorting and enriching the differentiated beta cells. The present disclosure also relates to compositions and methods of irradiating cell population for reducing proliferation.
Claims60
A method of treating a subject with Type 1 Diabetes, comprising administering to the subject a plurality of autologous or HLA compatible cell clusters comprising cells differentiated from pluripotent stem cells in vitro, wherein the cell clusters comprise NKX6.1-positive and ISL1-positive cells, NKX6.1-negative and ISL1-negative cells, CHGA-positive cells, and C-peptide-positive cells, wherein:
- a) at least 35% of the cells in the cell clusters are NKX6.1-positive and ISL1-a) positive cells;
- b) at most 3.2% of the cells in the cell clusters are NKX6.1-negative and ISL1-negative cells;
- c) at least 60% of the cells in the cell clusters are CHGA-positive cells;
- and
- d) at least 30% of the cells in the cell clusters are C-peptide-positive cells;
- and
- e) the cells were not subjected to a cell sorting process comprising subjecting the cells to flow cytometry based on a fluorescent protein expressed in the cells.
A method of treating a subject with Type 1 Diabetes, comprising:
- administering to the subject a plurality of cell clusters comprising cells differentiated from pluripotent stem cells in vitro, and
- administering to the subject an immune response modulator for reducing rejection of the cell clusters,
- wherein the cell clusters comprise NKX6.1-positive and ISL1-positive cells, NKX6.1-negative and ISL1-negative cells, CHGA-positive cells, and C-peptide-positive cells, wherein:
- a) at least 35% of the cells in the cell clusters are NKX6.1-positive and ISL1-positive cells;
- b) at most 3.2% of the cells in the cell clusters are NKX6.1-negative and ISL1-negative cells;
- c) at least 60% of the cells in the cell clusters are CHGA-positive cells;
- d) at least 30% of the cells in the cell clusters are C-peptide-positive cells;
- and
- e) the cells were not subjected to a cell sorting process comprising subjecting the cells to flow cytometry based on a fluorescent protein expressed in the cells.
Dependencies are inferred from the claim text.