CRISPR Gene Regulation for Degenerative Disc Pain Relief

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Solution Overview

Problem

Current treatments for degenerative disc disease-induced low back pain fail to prevent the progression of disc degeneration and often lead to symptom recurrence, while stem cell therapies struggle to maintain an exemplary phenotype in challenging disease environments.

Innovation Solution

Utilizing CRISPR-Cas systems with guide RNAs and RNA-directed nucleases to modulate gene expression in cells, specifically targeting inflammatory cytokine receptors and regulatory elements to inhibit catabolic signaling and promote therapeutic effects.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If stem cell therapies are used to treat degenerative disc disease, then pain relief and tissue regeneration are achieved, but the stem cells struggle to maintain their phenotype in the challenging disease environment

Engineering Contradiction:

Improvestem cell phenotype maintenanceVSAvoidchallenging disease environment

Core Design Contradiction:

ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies preliminary action by pre-modifying stem cells ex vivo using CRISPR-Cas9 technology to introduce protective genetic modifications before implantation. The stem cells are engineered to express anti-inflammatory cytokines and matrix proteins that enable them to survive and maintain their phenotype in the harsh inflammatory environment of degenerative disc disease, thereby resolving the contradiction between stem cell phenotype maintenance and the challenging disease environment.

Inventive Principle:

Principle #10Preliminary action

2Ease of operation

If conventional treatments (surgical or non-surgical) are used for DDD-induced LBP, then symptoms are alleviated, but the progression of disc degeneration is not prevented and symptoms return

Engineering Contradiction:

Improvesymptom alleviationVSAvoidlong-term pain relief

Core Design Contradiction:

Ease of operationVSDuration of action of stationary object

Solution Approach 1:

The patent applies continuity of useful action by using CRISPR-Cas9 mediated gene editing to create permanent genetic modifications in stem cells that continuously produce anti-inflammatory and matrix-protective factors. This continuous cellular action prevents the recurrence of symptoms and halts disc degeneration progression, transforming temporary symptom relief into long-term therapeutic effect.

Inventive Principle:

Principle #20Continuity of useful action

Solution Approach 2:

The engineered stem cells perform self-service by autonomously secreting protective factors and maintaining their phenotype without requiring external growth factors or continuous intervention. The CRISPR-modified stem cells self-regulate their gene expression to maintain their therapeutic phenotype in the disease environment, enabling long-term pain relief without symptom recurrence.

Inventive Principle:

Principle #25Self-service

3Adaptability or versatility

If growth factor treatment is used to control stem cell phenotype, then cell differentiation is achieved, but the complexity and cost of the treatment regimen increases

Engineering Contradiction:

Improvecell phenotype controlVSAvoidgrowth factor treatment regimen

Core Design Contradiction:

Adaptability or versatilityVSDevice complexity

Solution Approach 1:

The patent applies the extraction principle by removing the need for external growth factor treatments and extracting phenotype control capability directly into the stem cells themselves through CRISPR-Cas9 genetic modification. The stem cells are engineered to autonomously produce the necessary proteins and cytokines for maintaining their phenotype, eliminating the complexity of external growth factor regimens while preserving adaptability.

Inventive Principle:

Principle #2Taking out (Extraction)

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 CRISPR-Cas system effectively modulates gene expression to slow down degenerative disc disease progression, reducing inflammation and enhancing the therapeutic efficacy of stem cell treatments.

Implementation Method 1

the gRNA hybridizes with a target sequence of a DNA molecule in a cell

Methodology Applied

Scientific EffectHybridization:

Data Source

PatentUS20250354139A1RNA-guided transcriptional regulation and methods of using the same for the treatment of back pain

Publication Date: 2025.11.20 UNIV OF UTAH RES FOUND

AI summary

Described herein are compositions and methods for treatment and prevention of low back pain. The compositions include vectors comprising nucleotide sequences encoding one or more CRISPR-Cas system guide RNAs and a RNA-directed nuclease. The methods include modulating expression of one or more genes in a cell using said compositions, introducing a CRISPR-Cas system into a cell comprising one or more vectors comprising said compositions, inducing site-specific DNA cleavage in a cell, and treating a subject having lower back pain, and lower back pain caused by degenerative disc disease using the compositions disclosed herein.