Combined hairpin-antisense compositions and methods for modulating expression
AssigneeBrookhaven Science Associates, LLC
InventorsJohn Shanklin, Tam Nguyen
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Abstract
A nucleotide construct comprising a nucleotide sequence that forms a stem and a loop, wherein the loop comprises a nucleotide sequence that modulates expression of a target, wherein the stem comprises a nucleotide sequence that modulates expression of a target, and wherein the target modulated by the nucleotide sequence in the loop and the target modulated by the nucleotide sequence in the stem may be the same or different. Vectors, methods of regulating target expression, methods of providing a cell, and methods of treating conditions comprising the nucleotide sequence are also disclosed.
Claims33
A nucleotide construct comprising:
- a nucleotide sequence that forms a stem and a loop;
- wherein the loop comprises a first nucleotide sequence that modulates expression of a target;
- wherein the stem comprises a second nucleotide sequence that modulates expression of a target;
- wherein both the first and second nucleotide sequence modulate the expression of a target via binding to a target;
- wherein the first nucleotide sequence and the second nucleotide sequence are not contiguous;
- wherein the first nucleotide sequence is located entirely within the loop;
- and
- wherein the target modulated by the first nucleotide sequence and the target modulated by the second nucleotide sequence may be the same or different.
A nucleotide construct comprising:
- a nucleotide sequence that forms a stem and a loop;
- wherein the loop comprises a first nucleotide sequence that modulates expression of a target;
- wherein the stem comprises a second nucleotide sequence that modulates expression of a target;
- wherein both the first and second nucleotide sequence modulate the expression of a target via binding to a target;
- wherein the target modulated by the first nucleotide sequence and the target modulated by the second nucleotide sequence may be the same or different;
- wherein the first nucleotide sequence is located entirely within the loop;
- and
- wherein the first nucleotide sequence and the second nucleotide sequence each comprise an antisense sequences capable of modulating the expression of a gene selected from the group consisting of FAB1, FAD2, and FAD3.
A nucleotide construct comprising:
- a nucleotide sequence that forms a stem and a loop;
- wherein the loop comprises a first nucleotide sequence of at least ten contiguous bases that are complementary to a target;
- wherein the stem comprises a second nucleotide sequence of at least ten contiguous bases that are complementary to a target;
- wherein both the first and second nucleotide sequence are able to hybridize with a target;
- wherein the first nucleotide sequence and the second nucleotide sequence are not contiguous;
- wherein the first nucleotide sequence is located entirely within the loop;
- and
- wherein the target of the first nucleotide sequence and the target of the second nucleotide sequence may be the same or different.
A nucleotide construct comprising:
- a nucleotide sequence that forms a stem and a loop;
- wherein the loop comprises a first nucleotide sequence that modulates expression of a target;
- wherein the stem comprises a second nucleotide sequence that modulates expression of a target;
- wherein both the first and second nucleotide sequence modulate the expression of a target via binding to a target;
- wherein the first nucleotide sequence and the second nucleotide sequence are not contiguous;
- wherein the first nucleotide sequence is located entirely within the loop;
- wherein the nucleotide construct comprises splice sites configured so as to allow at least a portion of the loop to be spliced away from the stem;
- and
- wherein the target modulated by the first nucleotide sequence and the target modulated by the second nucleotide sequence may be the same or different.
Dependencies are inferred from the claim text.