CFTR gene regulator
AssigneeIsis Innovation Limited
InventorAnn Harris
Life of this patent
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Abstract
The invention relates to a novel CFTR gene regulatory element capable of increasing the activity of the CFTR gene promoter, and to nucleic acid constructs comprising the element together with the CFTR gene coding region. The element and constructs containing it are useful in gene therapy for treating cystic fibrosis.
Claims15
The oligonucleotide GTCACTTAT and complementary oligonucleotides.
The oligonucleotide ACAATGTGATCTT and complementary oligonucleotides.
An oligonucleotide which is one of the following sequences, or a fragment of one of the following sequences including one or both of the underlined sequences;
- and oligonucleotides complementary thereto:
- 3 [SEQ ID NO:1] AATCCTAACTCTGTCACTTAT [SEQ ID NO:2] TAACAATGTGATCTTAGGCAATTTACTT [SEQ ID NO:3] AATCCTAACTCTGTCACTTATTAACAATGTGATCTTAGGCAATTTACTT
A nucleic acid element that is capable of controlling expression of the CFTR gene, which nucleic acid element has at least 70% homology with the sequence shown in FIG. 1C or a fragment thereof.
A nucleic acid element that is a functional CFTR gene regulatory sequence, which nucleic acid element is derived from intron 1 of the CFTR gene, and which is not the sequence TGTCACTTATCTTGC.
Dependencies are inferred from the claim text.