Gene therapy for tuberous sclerosis
US-2024343768-A1 · Oct 17, 2024 · US
US10792330B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10792330-B2 |
| Application number | US-201715805534-A |
| Country | US |
| Kind code | B2 |
| Filing date | Nov 7, 2017 |
| Priority date | Jul 11, 2012 |
| Publication date | Oct 6, 2020 |
| Grant date | Oct 6, 2020 |
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The invention relates, in part, to methods and compositions that are useful to modulate metabolic function of cells in vivo or in vitro. In some aspects the invention includes methods and/or compositions that increase metabolism in cells, tissues, organs, and/or subjects. In certain aspects the invention includes methods and/or compositions useful to decrease metabolism in cells, tissues, organs, and/or in subjects.
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What is claimed is: 1. A method of increasing mitochondrial metabolism in a CD 8+ T cell, the method comprising contacting a CD 8+ T cell in vitro with an exogenous MCJ-modulating compound that decreases an MCJ activity in an amount effective to decrease MCJ activity and increase mitochondrial metabolism in the CD 8+ T cell, wherein the MCJ-modulating compound comprises an MCJ siRNA molecule. 2. The method of claim 1 , wherein decreasing the MCJ activity comprises decreasing a level or function of MCJ polypeptide in the CD 8+ T cell. 3. The method of claim 1 , wherein mitochondrial metabolism is mitochondrial respiration. 4. The method of claim 1 , wherein the MCJ-modulating compound genetically targets MCJ expression and reduces the amount of MCJ polypeptide expressed in the CD 8+ T cell. 5. The method of claim 1 , wherein the MCJ-modulating compound further comprises a mitochondrial targeting agent. 6. The method of claim 1 , wherein the CD 8+ T cell is an ex vivo CD 8+ T cell.
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