Invention Grant
- Patent Title: Compositions and methods for treating transposon associated diseases
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Application No.: US16645083Application Date: 2018-09-12
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Publication No.: US11999953B2Publication Date: 2024-06-04
- Inventor: Timothy Yu , Eunjung Alice Lee
- Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION
- Applicant Address: US MA Boston
- Assignee: The Children's Medical Center Corporation
- Current Assignee: The Children's Medical Center Corporation
- Current Assignee Address: US MA Boston
- Agency: Greenberg Traurig, LLP
- Agent Melissa Hunter-Ensor; Nathan Hsu
- International Application: PCT/US2018/050576 2018.09.12
- International Announcement: WO2019/055460A 2019.03.21
- Date entered country: 2020-03-06
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C12Q1/68

Abstract:
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.
Public/Granted literature
- US20200263173A1 COMPOSITIONS AND METHODS FOR TREATING TRANSPOSON ASSOCIATED DISEASES Public/Granted day:2020-08-20
Information query
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