Treatment of multiple sclerosis using nt-3 gene therapy
AssigneeResearch Institute At Nationwide Children's Hospital
InventorZarife Sahenk
Life of this patent
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Abstract
The present disclosure relates to methods of treating an autoimmune disease, such as multiple sclerosis, using the rAAV expressing NT-3 gene therapy.
Claims78
A method of treating multiple sclerosis (MS) in a human subject in need thereof comprising the step of administering to the human subject a nucleic acid encoding a NT-3 polypeptide;
- wherein
- a) the nucleic acid comprises a nucleotide sequence that is at least 90% identical to the nucleotide sequence of SEQ ID NO:
- 1;
- b) the nucleic acid comprises the nucleotide sequence of SEQ ID NO:
- 1;
- c) the nucleic acid comprises a nucleotide sequence encoding an amino acid sequence that is at least 90% identical to SEQ ID NO:
- 2;
- or
- d) the nucleic acid comprises a nucleotide sequence encoding the amino acid sequence of SEQ ID NO:
- 2.
A method of treating an autoimmune disease in a human subject in need thereof comprising the step of administering to the human subject a nucleic acid encoding a NT-3 polypeptide;
- wherein
- a) the nucleic acid comprises a nucleotide sequence that is at least 90% identical to the nucleotide sequence of SEQ ID NO:
- 1;
- b) the nucleic acid comprises the nucleotide sequence of SEQ ID NO:
- 1;
- c) the nucleic acid comprises a nucleotide sequence encoding an amino acid sequence that is at least 90% identical to SEQ ID NO:
- 2;
- or
- d) the nucleic acid comprises a nucleotide sequence encoding the amino acid sequence of SEQ ID NO:
- 2.
A viral vector for use in the treatment of multiple sclerosis (MS) in a human subject in need thereof, wherein the composition comprises a nucleic acid encoding a NT-3 polypeptide;
- wherein
- a) the nucleic acid comprises a nucleotide sequence that is at least 90% identical to the nucleotide sequence of SEQ ID NO:
- 1;
- b) the nucleic acid comprises the nucleotide sequence of SEQ ID NO:
- 1;
- c) the nucleic acid comprises a nucleotide sequence encoding an amino acid sequence that is at least 90% identical to SEQ ID NO:
- 2;
- or
- d) the nucleic acid comprises a nucleotide sequence encoding the amino acid sequence of SEQ ID NO:
- 2.
A viral vector for use in treating an autoimmune disease in a human subject in need thereof;
- wherein the composition comprises a nucleic acid encoding a NT-3 polypeptide;
- wherein
- a) the nucleic acid comprises a nucleotide sequence that is at least 90% identical to the nucleotide sequence of SEQ ID NO:
- 1;
- b) the nucleic acid comprises the nucleotide sequence of SEQ ID NO:
- 1;
- c) the nucleic acid comprises a nucleotide sequence encoding an amino acid sequence that is at least 90% identical to SEQ ID NO:
- 2;
- or
- d) the nucleic acid comprises a nucleotide sequence encoding the amino acid sequence of SEQ ID NO:
- 2.
Use of a nucleic acid encoding a NT-3 polypeptide for the preparation of a medicament for treating multiple sclerosis (MS) in a human subject in need thereof, wherein
- a) the nucleic acid comprises a nucleotide sequence that is at least 90% identical to the nucleotide sequence of SEQ ID NO:
- 1;
- b) the nucleic acid comprises the nucleotide sequence of SEQ ID NO:
- 1;
- c) the nucleic acid comprises a nucleotide sequence encoding an amino acid sequence that is at least 90% identical to SEQ ID NO:
- 2;
- or
- d) the nucleic acid comprises a nucleotide sequence encoding the amino acid sequence of SEQ ID NO:
- 2.
Use of a nucleic acid encoding a NT-3 polypeptide for the preparation of a medicament for treating an autoimmune disease in a human subject in need thereof;
- wherein
- a) the nucleic acid comprises a nucleotide sequence that is at least 90% identical to the nucleotide sequence of SEQ ID NO:
- 1;
- b) the nucleic acid comprises the nucleotide sequence of SEQ ID NO:
- 1;
- c) the nucleic acid comprises a nucleotide sequence encoding an amino acid sequence that is at least 90% identical to SEQ ID NO:
- 2;
- or
- d) the nucleic acid comprises a nucleotide sequence encoding the amino acid sequence of SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.