Antisense Oligonucleotides Targeting Transposable Element RNA
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Solution Overview
Problem
Current methods are inadequate for effectively detecting and treating the deleterious effects of transposable element insertions, such as those caused by LINE-1, Alu, SVA, and ERV, which contribute to somatic alterations and cancer in mammals.
Innovation Solution
The use of antisense oligonucleotides (AONs) that are complementary to transposable elements, administered to target and inhibit their activity, thereby correcting genetic defects and splicing errors associated with these elements.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Measurement precision
If current detection methods are used for transposable element insertions, then the detection process is simple, but the detection accuracy and effectiveness are insufficient
Solution Approach 1:
The patent uses antisense oligonucleotides as intermediary molecules that bind to transposable element RNA transcripts. These AONs serve as detectable proxies that reveal the presence of transposable elements through hybridization, enabling accurate detection without directly sequencing the complex genomic regions.
Solution Approach 2:
The patent replaces complex mechanical sequencing and genotyping methods with a molecular biology-based detection system using nucleic acid hybridization. This substitution allows for more accurate detection of transposable element insertions through RNA binding assays rather than direct genomic sequencing.
2Reliability
If current treatment methods are used for transposable element-related diseases, then the treatment approach is simple, but the therapeutic effectiveness is inadequate
Solution Approach 1:
The patent extracts and targets the RNA transcripts of specific transposable elements using sequence-specific antisense oligonucleotides. By removing or inhibiting the transcription of these elements, the therapy eliminates the harmful effects of transposable element insertion without affecting the entire genome.
Solution Approach 2:
The patent applies local quality by designing antisense oligonucleotides with specific sequences complementary to particular transposable element families (e.g., LINE-1, Alu, SVA). This allows targeted therapy against specific harmful elements while preserving the function of other genomic regions.
3Reliability
If antisense oligonucleotides are used to target transposable elements, then the therapeutic effectiveness improves, but the complexity of the treatment protocol increases
Solution Approach 1:
The patent designs antisense oligonucleotides that can be administered via multiple routes (inhalation, intravenous, intramuscular) and target multiple transposable element families with a single therapeutic platform. This multi-functionality simplifies the overall treatment approach despite the molecular complexity of the AONs themselves.
Applied Scientific Principles
This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.
Function Achieved in This Case
The antisense oligonucleotides effectively ameliorate the effects of transposable element insertions by reducing their expression and correcting genetic mutations, providing a therapeutic approach to treat disorders linked to these elements.
Implementation Method 1
antisense oligonucleotides (AONs) that are complementary to transposable elements
Data Source
AI summary
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.