Gene Therapy for Intervertebral Disc Mineralization

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Solution Overview

Problem

Current methods for spinal fusion, such as surgery, are invasive, costly, and associated with high risks and complications, with a significant failure rate in relieving lower back pain and achieving fusion.

Innovation Solution

Compositions and methods that up-regulate the expression of TNAP and down-regulate ANK and/or ENPP in intervertebral discs to induce mineralization, allowing for spine fusion without major surgical intervention by converting disc tissue into bone.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If traditional surgical spinal fusion is performed, then fusion is achieved, but the procedure is invasive, costly, and associated with high risks and complications

Engineering Contradiction:
Improvefusion success rateVSAvoidsurgical risks and complications
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent replaces the mechanical surgical intervention system with a biochemical system. Instead of using surgical instruments, implants, and mechanical fusion methods, the invention uses gene therapy vectors to deliver therapeutic genes that trigger biological mineralization processes, converting disc tissue into bone through controlled biochemical pathways rather than mechanical force or surgical manipulation

Inventive Principle:
Principle #28Mechanics substitution (Replace mechanical system)

Solution Approach 2:

The patent enables the intervertebral disc to self-transform into bone tissue through endogenous cellular mechanisms. The therapeutic genes activate the disc's own cells to perform mineralization and bone formation, eliminating the need for external surgical intervention, implants, or continuous medical support, thereby reducing complications associated with foreign bodies and surgical procedures

Inventive Principle:
Principle #25Self-service

2Reliability

If traditional surgical spinal fusion is performed, then fusion is achieved, but the procedure is costly and time-consuming

Engineering Contradiction:
Improvefusion achievementVSAvoidsurgery duration and recovery time
Core Design Contradiction:
ReliabilityVSLoss of time

Solution Approach 1:

The patent employs preliminary genetic preparation by delivering therapeutic genes into the disc tissue before fusion is needed. The genes are expressed and accumulate mineralization-promoting proteins in advance, so that when mineralization is triggered, the process occurs rapidly and efficiently without requiring lengthy surgical procedures or extended recovery periods

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent fundamentally changes the parameter of fusion achievement from a mechanically-driven surgical outcome to a biochemically-driven biological process. By altering the biochemical parameters within disc cells through gene therapy, the invention accelerates the fusion timeline and eliminates the need for prolonged surgical intervention and recovery

Inventive Principle:
Principle #35Parameter changes

3Reliability

If traditional surgical spinal fusion is performed, then fusion is achieved, but failure rates remain high in relieving lower back pain

Engineering Contradiction:
Improvefusion outcomeVSAvoidpersistent pain and treatment failure
Core Design Contradiction:
ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The patent replaces the mechanical fusion approach with a biochemical regeneration approach. Instead of forcing vertebrae together through surgical hardware and bone grafts, the invention uses gene therapy to stimulate the disc tissue to naturally transform into bone, creating a more biologically compatible and potentially more effective fusion that addresses the root cause of pain rather than merely stabilizing the spine mechanically

Inventive Principle:
Principle #28Mechanics substitution (Replace mechanical system)

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

This approach enables minimally invasive spine fusion with reduced risk and cost, effectively addressing the limitations of traditional surgical methods by promoting bone formation in intervertebral discs, thereby fusing vertebrae.

Implementation Method 1

TNAP is an enzyme that catalyzes the hydrolysis of inorganic pyrophosphate

Methodology Applied
Scientific EffectHydrolysis: Hydrolysis

Implementation Method 2

increased phosphate concentration ratio, thereby promoting mineralization and/or formation of hydroxyapatite

Methodology Applied
Scientific EffectPrecipitation: Precipitation

Data Source

PatentUS20240252600A1Compositions and methods for promoting the mineralization of biological tissue
Publication Date: 2024.08.01 NEW YORK SOC FOR THE RUPTURED & CRIPPLED MAINTAINING THE HOSPITAL FOR SPECIAL SURGERY
  • US20240252600A1 patent drawing
  • US20240252600A1 patent drawing
  • US20240252600A1 patent drawing

AI summary

This invention relates to compositions and methods for activating and promoting mineralization in tissue that does not normally mineralize, specifically intervertebral discs. The composition comprises agents that increase the expression of the gene that encodes TNAP and/or the activation, amount or activity of INAP protein, and agents that decrease the expression of ANK and/or ENPP and/or the activation, amount or activity of these proteins. The composition can be in the form of a cell or cells. The invention also relates to methods of using the composition.