LiBIS-seq Cell-free DNA Insertion Site Sequencing

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Solution Overview

Problem

Current methods for analyzing insertion sites in gene therapy are limited, as they primarily rely on invasive tissue biopsies and do not effectively monitor vector-marked cell clones in solid organs, leading to potential late detection of adverse effects such as malignancy.

Innovation Solution

The development of Liquid Biopsy Insertion Site Sequencing (LiBIS-seq) method, which identifies and quantifies viral and vector insertion sites in cell-free DNA from body fluids like blood plasma, allowing non-invasive monitoring of insertion sites in both circulating and solid organ cells.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Measurement precision

If invasive tissue biopsies are used to analyze insertion sites, then direct detection of vector-marked cell clones in solid organs is achieved, but patient safety is compromised and the procedure becomes risky

Engineering Contradiction:
Improvedetection of insertion sitesVSAvoidinvasive biopsy risk
Core Design Contradiction:
Measurement precisionVSObject-affected harmful factors

Solution Approach 1:

The patent uses cell-free DNA from body fluids as a copy of the genomic material to indirect detection of insertion sites. Instead of directly biopsying solid organs, the method sequences insertion sites from circulating cell-free DNA, which contains molecular copies of integrated vector sequences, thereby achieving detection without tissue invasion

Inventive Principle:
Principle #26Copying

Solution Approach 2:

The patent introduces body fluids (blood, plasma, serum) as an intermediary medium to access vector-marked cell clones. Cell-free DNA in these fluids serves as a accessible proxy that reflects the presence of integrated vectors in solid organs without requiring direct tissue sampling

Inventive Principle:
Principle #24Intermediary (Mediator)

2Reliability

If invasive tissue biopsies are performed to monitor solid organ cells, then comprehensive monitoring of vector-marked clones is achieved, but the procedure becomes complex and risky

Engineering Contradiction:
Improvemonitoring of gene therapy safetyVSAvoidbiopsy procedure complexity
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The method creates molecular copies of insertion sites through PCR amplification of cell-free DNA fragments containing vector integration junctions. This copying process enables repeated, non-invasive monitoring of the same insertion sites over time without requiring successive biopsies

Inventive Principle:
Principle #26Copying

Solution Approach 2:

The body's own circulating cell-free DNA serves as the sample source, eliminating the need for external intervention to obtain tissue samples. The method leverages naturally present cell-free DNA in body fluids for self-monitoring of gene therapy outcomes

Inventive Principle:
Principle #25Self-service

3Ease of operation

If cell-free DNA from body fluids is used to identify insertion sites, then non-invasive monitoring is achieved, but detection sensitivity may be reduced

Engineering Contradiction:
Improvenon-invasive samplingVSAvoiddetection sensitivity
Core Design Contradiction:
Ease of operationVSMeasurement precision

Solution Approach 1:

The method performs preliminary enrichment and purification of cell-free DNA from body fluids before sequencing. By concentrating and preparing the DNA sample through specific protocols, the method enhances the quality and quantity of analyzable material to improve detection sensitivity

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent employs PCR amplification to create multiple copies of the target insertion site sequences from limited cell-free DNA input. This partial amplification approach concentrates the signal of interest while filtering out background noise, thereby enhancing detection sensitivity despite the small initial sample amount

Inventive Principle:
Principle #16Partial or excessive action

Data Source

PatentEP3953490B1Method for analysing insertion sites
Publication Date: 2024.03.20 FOND AZIONE TELETHON
  • EP3953490B1 patent drawingFigure 1
  • EP3953490B1 patent drawingFigure 2A~2B
  • EP3953490B1 patent drawingFigure 2C~2D

AI summary

A method for analysing insertion sites of an exogenous nucleotide sequence in a subject's genome, wherein the method comprises: (a) providing a sample from the subject comprising cell-free double-stranded DNA polynucleotides; (b) blunting the ends of the polynucleotides; (c) ligating an oligonucleotide to both ends of the polynucleotides; (d) amplifying polynucleotides comprising an insertion site; and (e) sequencing the product of step (d).