Loading patent…
AssigneeChildren's Medical Center Corporation
InventorsTIMOTHY YU, EUNJUNG ALICE LEE
Estimating the term…
The present invention features antisense oligonucleotides (AONs) for the treatment of diseases and disorders associated with the deleterious effects of transposable element insertion (e.g., long interspersed nuclear element-i (LINE-1), Arthrobacter luteus element (Alu), short interspersed nuclear element variable number tandem repeat Arthrobacter luteus element (SINE-VNTR-Alu) or (SVA), or endogenous retrovirus (ERV). In one aspect, the invention provides one or more antisense oligonucleotides complementary to a transposable element present in an intronic sequence within a gene. In another aspect, the invention provides a method for treating a subject having a genetic disorder associated with the insertion of a transposable element, the method involving administering to the subject one or more antisense oligonucleotides of any aspect delineated herein.
One or more antisense oligonucleotides complementary to a transposable element present in an intronic sequence within a genome.
(canceled)
(canceled)
(canceled)
(canceled)
21-22. (canceled)
An isolated cell that is heterozygous for a mutation associated with a genetic defect, comprising a genetic mutation and a second allele comprising a retrotransposon insertion.
(canceled)
A method of identifying an agent that inhibits a splicing event caused by a retrotransposon insertion, the method comprising contacting the cell, and detecting a splice product that does not comprise a retransposon sequence or fragment thereof.
Dependencies are inferred from the claim text.