Fabry disease gene therapy
AssigneeUCL Business PLC
InventorsAmit Nathwani, Deepak Raj
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Abstract
There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional α-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.
Claims21
1-17. (canceled)
A method of administering a nucleic acid molecule to a subject, comprising administering to the subject the nucleic acid molecule comprising a nucleotide sequence encoding a polypeptide having α-galactosidase A activity, wherein the nucleotide sequence has at least 91% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the subject suffers from Fabry disease.
The method of claim 1, wherein the nucleotide sequence has at least 92% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the nucleotide sequence has at least 93% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the nucleotide sequence has at least 94% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the nucleotide sequence has at least 95% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the nucleotide sequence has at least 96% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the nucleotide sequence has at least 97% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the nucleotide sequence has at least 98% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the nucleotide sequence has at least 99% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 1, wherein the nucleotide sequence has the nucleotide sequence of SEQ ID NO:
- 1.
A method of administering a vector to a subject, comprising administering to the subject the vector comprising a nucleic acid molecule, wherein the nucleic acid molecule comprises a nucleotide sequence encoding a polypeptide having α-galactosidase A activity, and wherein the nucleotide sequence has at least 91% identity to the nucleotide sequence of SEQ ID NO:
- 1.
The method of claim 12, wherein the subject suffers from Fabry disease.
The method of claim 12, wherein the vector is an AAV vector.
The method of claim 12, wherein the vector is a single stranded vector.
The method of claim 12, wherein the vector comprises a nucleotide sequence having at least 90% identity to the nucleotide sequence of SEQ ID NO:
- 3.
The method of claim 12, wherein the vector comprises a nucleotide sequence having at least 95% identity to the nucleotide sequence of SEQ ID NO:
- 3.
The method of claim 12, wherein the vector comprises a nucleotide sequence having at least 98% identity to the nucleotide sequence of SEQ ID NO:
- 3.
The method of claim 12, wherein the vector comprises a nucleotide sequence having the nucleotide sequence of SEQ ID NO:
- 3.
The method of claim 12, wherein the nucleotide sequence is operably linked to a liver specific promoter.
Dependencies are inferred from the claim text.