Treatment of Parkinson's Disease and Related Disorders
AssigneeCedars-Sinai Medical Center
InventorsPedro Lowenstein, Maria Castro
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Abstract
Described herein are compositions and methods for the treatment of Parkinson's disease (PD) and/or to protect dopaminergic nigrostriatal neuronal cell bodies from 6-OHDA-induced neurotoxicity in a mammal. In various embodiments of the invention, the dopaminergic neuron differentiation factor sonic hedgehog (Shh) and/or its downstream transcription factor target Gli-1 are used in connection with gene therapeutic techniques or direct peptide injection for the aforementioned indications. Kits useful in practicing the inventive method are also disclosed, as are animal models useful for studying various neurodegenerative conditions.
Claims21
A method for treating Parkinson's disease in a mammal, comprising:
- providing a quantity of a viral vector expressing an N-terminal of sonic hedgehog (“ShhN”);
- and
- administering a therapeutically effective amount of the quantity of viral vector to the mammal.
A method for treating Parkinson's disease in a mammal, comprising:
- providing a quantity of a viral vector expressing Gli-1;
- and
- administering a therapeutically effective amount of the quantity of viral vector to the mammal.
A method for protecting dopaminergic nigrostriatal neuronal cell bodies from 6-OHDA-induced neurotoxicity in a mammal, comprising:
- providing a quantity of a viral vector expressing an N-terminal of sonic hedgehog (“ShhN”);
- and
- administering a therapeutically effective amount of the quantity of viral vector to the mammal.
A method for protecting dopaminergic nigrostriatal neuronal cell bodies from 6-OHDA-induced neurotoxicity in a mammal, comprising:
- providing a quantity of a viral vector expressing Gli-1;
- and
- administering a therapeutically effective amount of the quantity of viral vector to the mammal.
A method for treating Parkinson's disease in a mammal, comprising:
- providing a composition comprising an N-terminal of sonic hedgehog (“ShhN”) protein, a Gli-1 protein, or both;
- and
- administering a therapeutically effective amount of the composition to the mammal.
A method for protecting dopaminergic nigrostriatal neuronal cell bodies from 6-OHDA-induced neurotoxicity in a mammal, comprising:
- providing a composition comprising an N-terminal of sonic hedgehog (“ShhN”) protein, a Gli-1 protein, or both;
- and
- administering a therapeutically effective amount of the composition to the mammal.
A kit, comprising:
- a composition comprising a viral vector expressing an N-terminal of sonic hedgehog (“ShhN”);
- and
- instructions for the use of said composition for treating Parkinson's disease in a mammal.
A kit, comprising:
- a composition comprising a viral vector expressing Gli-1;
- and
- instructions for the use of said composition for treating Parkinson's disease in a mammal.
A kit, comprising:
- a composition comprising an N-terminal of sonic hedgehog (“ShhN”) protein, a Gli-1 protein, or both;
- and
- instructions for the use of said composition for treating Parkinson's disease in a mammal.
An in vivo model of nigrostriatal neurodegeneration, comprising a non-human mammal, wherein the non-human mammal carries in at least a portion of the cells of its brain at least one exogenous N-terminal of sonic hedgehog (“ShhN”) DNA encoding a ShhN peptide.
An in vivo model of nigrostriatal neurodegeneration, comprising a non-human mammal, wherein the non-human mammal carries in at least a portion of the cells of its brain at least one exogenous Gli-1 DNA encoding a Gli-1 peptide.
Dependencies are inferred from the claim text.