Engineered Mammalian Cells With Gene Editing for Transplant Survival

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Solution Overview

Problem

Transplanted cells and tissues face a stressful and hostile environment in host subjects, necessitating the need for cells with improved survivability and reduced immunogenicity.

Innovation Solution

Genetically engineered mammalian cells with adjusted gene expression profiles, including decreased expression of renalase, ABO, CXCL10, B2M, and F3 genes, and increased or modified expression of CD47, to enhance survivability and reduce immunogenicity.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If cells are transplanted into host subjects, then treatment for diseases such as diabetes can be achieved, but the cells encounter a stressful and hostile environment leading to poor survivability and increased immunogenicity

Engineering Contradiction:

Improvecell survivabilityVSAvoidhost environment stress and hostility

Core Design Contradiction:

ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies preliminary action by pre-engineering cells with specific gene modifications (decreasing expression of renalase, ABO, CXCL10, B2M, and F3 genes) before transplantation. This preparatory genetic engineering equips the cells with enhanced survivability and reduced immunogenicity traits in advance, allowing them to withstand the hostile host environment more effectively after transplantation.

Inventive Principle:

Principle #10Preliminary action

2Reliability

If cells are transplanted into host subjects, then treatment for diseases such as diabetes can be achieved, but the cells encounter increased immunogenicity leading to rejection

Engineering Contradiction:

Improvecell survivabilityVSAvoidcell immunogenicity

Core Design Contradiction:

ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The patent applies the extraction principle by removing or decreasing the expression of specific genes (renalase, ABO, CXCL10, B2M, F3) that contribute to immunogenicity. This genetic extraction eliminates the harmful factors that trigger immune rejection, allowing transplanted cells to survive longer in the host subject.

Inventive Principle:

Principle #2Taking out (Extraction)

Solution Approach 2:

The patent applies parameter changes by modifying the expression levels of specific genes through genetic engineering. By changing the expression parameters of renalase, ABO, CXCL10, B2M, and F3 genes, the cell's immunogenicity profile is altered, reducing its visibility to the host immune system and improving transplant outcomes.

Inventive Principle:

Principle #35Parameter changes

Data Source

PatentUS20260053859A1Genetically engineered cells

Publication Date: 2026.02.26 VERTEX PHARMACEUTICALS INC

AI summary

Disclosed herein are compositions and methods related to genetically engineered mammalian cells comprising adjusted expression of select genes. The genetically engineered mammalian cells described herein advantageously possess improved survivability.