Gene therapy for tuberous sclerosis
US-2024343768-A1 · Oct 17, 2024 · US
US11376308B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-11376308-B2 |
| Application number | US-201716343266-A |
| Country | US |
| Kind code | B2 |
| Filing date | Oct 18, 2017 |
| Priority date | Oct 19, 2016 |
| Publication date | Jul 5, 2022 |
| Grant date | Jul 5, 2022 |
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The inventors have determined that increasing the expression level or activity of FKBP-L polypeptide in a subject, which can be provided by expression of nucleic acids encoding FKBP-L or by providing FKBP-L polypeptides to a subject is advantageous for use in the treatment of obesity and obesity-related disorders. In particular increased expression or activity of FKBP-L polypeptide in a subject may be used to treat excessive weight gain (which can be characterised as obesity), glucose intolerance, diabetes and metabolic syndrome, which are closely linked to obesity and insulin resistance. FKBP-L can also be used as a biomarker for obesity and obesity-related disorders.
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The invention claimed is: 1. A method of treating obesity comprising the step of administering to a subject in need thereof, a FK506-binding protein like (FKBP-L) nucleic acid or polypeptide, wherein the FKBP-L nucleic acid or polypeptide is selected from the group consisting of: (a) a FKBP-L polypeptide encoded by the nucleotide sequence of SEQ ID NO: 30; (b) a FKBP-L polypeptide that consists of the amino acid sequence NH2-TIRQQPRDPPTETLELEVSPDPAS-OH (SEQ ID NO: 40); (c) a FKBP-L polypeptide that consists of the amino acid sequence NH2-QIRQQPRDPPTETLELEVSPDPAS-OH (SEQ ID NO: 10); (d) a nucleic acid capable of being expressed to provide a FKBP-L polypeptide that consists of the amino acid sequence NH2-IRQQPRDPPTETLELEVSPDPAS-OH (SEQ ID NO:40); and (e) a nucleic acid capable of being expressed to provide a FKBP-L polypeptide that consists of the amino acid sequence NH2-QIRQQPRDPPTETLELEVSPDPAS-OH (SEQ ID NO: 10). 2. The method of claim 1 , wherein the nucleic acid or polypeptide sequence further comprises a delivery peptide. 3. The method of claim 2 , wherein the delivery peptide is an RALA peptide.
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Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00) · CPC title
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