Therapeutics for the treatment of neurodegenerative disorders
AssigneesThe United States of America, as represented by the Secretary, Department of Health and Human Servic, UCL Business PLC
InventorsPietro Fratta, Anna Leigh Brown, Oscar Wilkins, Matthew Keuss, Michael Ward, Sarah Hill
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Abstract
Antisense oligonucleotides (ASOs) are provided which are capable of modulating splicing by preventing inclusion of an UNC13A cryptic exon into an UNC13A mature mRNA. Such ASOs may be used as a medicament, for example, to treat neurodegenerative disorders, particularly those associated with TDP-43 pathology.
Claims29
1-23. (canceled)
A method of reducing expression of a UNC13A cryptic exon splice variant of UNC13A mature mRNA in a cell comprising contacting the cell with an antisense oligonucleotide thereby reducing expression of the UNC13A cryptic exon splice variant of UNC13A mature mRNA in the cell, wherein the oligonucleotide is complementary to at least 8 contiguous nucleotides of SEQ ID NO:
- 1.
A method of modulating splicing of UNC13A in a cell comprising contacting the cell with an antisense oligonucleotide thereby modulating splicing of UNC13A in the cell, wherein the antisense oligonucleotide is complementary to at least 8 contiguous nucleotides of SEQ ID NO:1.
A method of reducing expression of a UNC13A cryptic exon splice variant of UNC13A mature mRNA in a cell comprising contacting the cell with an antisense oligonucleotide thereby reducing expression of the UNC13A cryptic exon splice variant of UNC13A mature mRNA in the cell, wherein the antisense oligonucleotide is complementary to at least 8 contiguous nucleotides of any one of SEQ ID NO:
- 547-554.
Dependencies are inferred from the claim text.