Gene therapy for tuberous sclerosis
US-2024343768-A1 · Oct 17, 2024 · US
US9694048B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9694048-B2 |
| Application number | US-201414773068-A |
| Country | US |
| Kind code | B2 |
| Filing date | Mar 13, 2014 |
| Priority date | Mar 15, 2013 |
| Publication date | Jul 4, 2017 |
| Grant date | Jul 4, 2017 |
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The disclosure relates to the recombinant Gla domain proteins and their use targeting phosphatidylserine (PtdS) moieties on the surface of cells, particularly those expressing elevated levels of PtdS, such as cells undergoing apoptosis.
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The invention claimed is: 1. A method of targeting cell membrane phosphatidylserine comprising: (a) providing an isolated polypeptide of from 4.5 to 30 kD in size comprising a gamma-carboxyglutamic-acid (Gla) domain with 5-15 Gla residues, a protein S EGF domain and lacking a protease or hormone-binding domain; and (b) contacting said peptide with a cell surface in vitro or ex vivo, wherein said polypeptide binds to phosphatidylserine on said cell membrane. 2. The method of claim 1 , wherein said cell membrane is a cardiac cell membrane, a neuronal cell membrane, an endothelial cell membrane, a virus-infected cell membrane, an apoptotic cell membrane, a platelet membrane or a cancer cell membrane. 3. The method of claim 1 , wherein said polypeptide further comprises a detectable label, wherein said detectable label comprises a fluorescent label, a chemiluminescent label, a radio label, an enzyme, a dye, or a ligand. 4. The method of claim 1 , wherein said polypeptide comprises 300 residues or less. 5. The method of claim 1 , wherein said polypeptide comprises from 1-5 disulfide bonds. 6. The method of claim 1 , wherein said polypeptide further comprises an antibody Fc region.
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