US 2022/0033826 A1 · ADENO-ASSOCIATED VIRAL VECTORS FOR THE TREATMENT OF BEST DISEASE

Abstract

Aspects of the disclosure relate to methods and compositions useful for treating bestrophinopathies, such as Best Disease.

Dependent claims

A short hairpin RNA (shRNA) comprising:

a) a sense strand comprising the nucleotide sequence CGUCAAAGCUUCACAGUGU (SEQ ID NO:
2) and an antisense strand comprising the nucleotide sequence ACACUGUGAAGCUUUGACG (SEQ ID NO:
3);
and
b) a loop.

2 more elements

Dependencies are inferred from the claim text.

Facts

Application

17/272,203Filed 30 Aug 2019

Priority

31 Aug 2018

Published

3 Feb 2022

Term ends

Not estimatedThe source records don’t support an estimate.

C12N 15/1138
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming…: against…
A61K 48/005
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy:…
C07K 14/705
From animals; from humans: receptors; Cell surface antigens; Cell surface determinants
C12N 7/00
Viruses

Aspects of the disclosure relate to methods and compositions useful for treating bestrophinopathies, such as Best Disease.

Dependent claims

A short hairpin RNA (shRNA) comprising:

a) a sense strand comprising the nucleotide sequence CGUCAAAGCUUCACAGUGU (SEQ ID NO:
2) and an antisense strand comprising the nucleotide sequence ACACUGUGAAGCUUUGACG (SEQ ID NO:
3);
and
b) a loop.

2 more elements

Dependencies are inferred from the claim text.