H19 and IG DMR Knockout in Androgenetic Stem Cells

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Solution Overview

Problem

The low birth rate and rapid decline in viability of semi-cloned mice produced using androgenetic haploid embryonic stem cells (AG-haESCs) due to abnormal imprinting status, particularly the abnormal expression of imprinted genes like H19 and Igf2, hinder efficient genome-wide loss-of-function genetic screening in mammals.

Innovation Solution

Knocking out the differentially methylated regions (DMRs) of the H19-Igf2 and Dlk1-Dio3 imprinted clusters in AG-haESCs, designated as DKO-AG-haESCs, to establish characteristics resembling round spermatids, which when combined with oocytes, result in a higher birth rate and stable production of genetically modified semi-cloned mice, enabling effective gene mutation and screening.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Adaptability or versatility

If AG-haESCs are used for producing semi-cloned mice, then genetic modification capability is improved, but birth rate and viability are severely reduced

Engineering Contradiction:
Improvegenetic modification capabilityVSAvoidbirth rate and viability
Core Design Contradiction:
Adaptability or versatilityVSReliability

Solution Approach 1:

The invention changes the epigenetic parameters of AG-haESCs by knocking out specific DMRs (H19 DMR and IG-DMR) to alter the imprinting status. This parameter change resolves the contradiction by making the cells more suitable for producing viable semi-cloned mice while maintaining their genetic modification capability. The DMR knockouts modify the methylation status and gene expression patterns, thereby improving reliability without sacrificing adaptability.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The invention extracts and removes the problematic differentially methylated regions (DMRs) from the AG-haESCs. By taking out the H19 DMR and IG-DMR elements that cause abnormal imprinting, the invention eliminates the harmful effects on birth rate and viability while preserving the essential genetic modification functions of the cells.

Inventive Principle:
Principle #2Taking out (Extraction)

2Quantity of substance

If long-term culture of AG-haESCs is performed, then cell expansion is achieved, but birth rate of semi-cloned mice declines rapidly

Engineering Contradiction:
Improvecell expansionVSAvoidbirth rate
Core Design Contradiction:
Quantity of substanceVSReliability

Solution Approach 1:

The invention performs preliminary action by knocking out the DMRs in advance during cell culture, before the cells are used for producing semi-cloned mice. This preliminary modification of the epigenetic status ensures that even after long-term culture and expansion, the cells maintain their ability to produce viable offspring, preventing the rapid decline in birth rate that normally occurs with prolonged culture.

Inventive Principle:
Principle #10Preliminary action

3Stability of the object's composition

If imprinted genes are expressed abnormally, then development of uniparental embryos is hindered, but this is the natural state of AG-haESCs

Engineering Contradiction:
Improveimprinting statusVSAvoidembryo development
Core Design Contradiction:
Stability of the object's compositionVSEase of operation

Solution Approach 1:

The invention changes the imprinting parameter status by selectively knocking out specific DMRs (H19 DMR and IG-DMR) rather than attempting to restore all imprinted genes. This selective parameter change resolves the contradiction by creating a modified imprinting status that is stable in the cells but permissive for embryo development, thereby improving ease of operation without completely altering the uniparental nature of AG-haESCs.

Inventive Principle:
Principle #35Parameter changes

Data Source

PatentEP3318639B1Androgenetic haploid embryonic stem cell, and preparation method and use thereof
Publication Date: 2024.12.11 CENT FOR EXCELLENCE IN MOLECULAR CELL SCI CHINESE ACAD OF SCI
  • EP3318639B1 patent drawingFigure 1A~1F
  • EP3318639B1 patent drawingFigure 1G~S1C
  • EP3318639B1 patent drawingFigure S1D~S1F

AI summary

The present invention relates to an AG-haESCs in which H19 DMR and IG-DMR are knocked out, a method for preparing the AG-haESCs, and use of the AG-haESCs in constructing a genetically modified semi-cloned animal and a library of a genetically modified semi-cloned animal. The AG-haESCs is capable of obtaining characteristics resembling a round spermatid, and upon injection into an oocyte, a viable SC mouse is stably obtained. The present invention is capable of being effectively used in multi-gene genetic manipulation, advancing the acquisition of animals with multiple genetic modifications.