Compositions and methods for inducing and regulating bone formation
OwnerPeter Lonai, Rozmari Stirbu-Lonai +1inventors
InventorsPeter Lonai, Rozmari Stirbu-Lonai, Veraragavan Eswarakumar
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Abstract
The present invention relates to the regulation of bone formation, particularly to the regulation of bone formation by Fibroblast Growth Factor Receptor (FGFR) subtypes, and more particularly the FGFR2-IIIc subtype, to compositions containing active variants of FGFR2-IIIc, and to methods of using such compositions to treat bone and cartilage defects. Also, a non-human transgenic animal having an altered gene that includes a DNA encoding a gain of function mutation in FGFR2c, wherein the gain of function mutation results in the expression of an active variant of FGFR2c in the transgenic animal.
Claims51
A polynucleotide construct comprising a polynucleotide sequence encoding an active variant of FGFR2c and a polynucleotide sequence encoding at least one FGFR2c specific ligand.
The polynucleotide construct of claim 3, wherein the promoter is tissue specific.
The polynucleotide construct of claim 4, wherein the tissue specific promoter is a bone specific promoter.
A vector comprising a construct comprising a polynucleotide sequence encoding an active variant of FGFR2c and a polynucleotide sequence encoding at least one FGFR2c specific ligand.
A host cell comprising a vector comprising a polynucleotide sequence encoding an active variant of FGFR2c and a polynucleotide sequence encoding at least one FGFR2c specific ligand.
A pharmaceutical composition for regulating bone or cartilage growth or organization comprising as an active ingredient a vector comprising the polynucleotide sequence encoding an active variant of FGFR2c.
A method for treating a subject suffering from bone and cartilage defects, disorders or diseases, comprising the step of treating said subject with a therapeutically effective amount of a vector comprising a polynucleotide sequence encoding an active FGFR2c.
A method for treating a subject suffering from bone or cartilage defects, disorders or diseases, comprising the step of treating said subject with a therapeutically effective amount of recombinant cells expressing at least one of the following receptors:
- an active variant of FGFR2c, a constitutively active ligand-independent FGFR2c, a constitutively active ligand-independent FGFR2c stable dimer.
A non-human transgenic animal having an altered gene, the altered gene comprises a DNA encoding a gain of function mutation in FGFR2c, wherein the gain of function mutation results in the expression of an active variant of FGFR2c in the transgenic animal.
Dependencies are inferred from the claim text.