Intrathecal HGF AAV Vector Composition for Higher Expression Yield

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Solution Overview

Problem

Conventional adeno-associated virus (AAV) vectors exhibit low productivity and protein expression levels when administered intrathecally, necessitating the development of optimized vectors for improved intrathecal administration.

Innovation Solution

The development of an AAV vector incorporating specific promoters, such as CMV or Elongation Factor 1-alpha, and polyadenylation sequences, along with AAV serotypes, enhances productivity and protein expression levels when administered intrathecally.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Productivity

If conventional AAV vectors are used for intrathecal administration, then the vector can be delivered to target cells, but the productivity and protein expression levels are significantly low

Engineering Contradiction:

ImproveAAV particle productivityVSAvoidprotein expression level

Core Design Contradiction:

ProductivityVSReliability

Solution Approach 1:

The patent applies parameter changes by modifying the AAV vector composition parameters - specifically changing the promoter type (CMV or EF-1α), polyadenylation sequence (hGH or bGH), and AAV serotype (AAV1 or AAV9) to optimize both productivity and protein expression levels for intrathecal administration

Inventive Principle:

Principle #35Parameter changes

Solution Approach 2:

The patent creates a composite AAV vector system by combining multiple elements - specific promoters (CMV/EF-1α), specific polyadenylation sequences (hGH/bGH), and specific AAV serotypes (AAV1/AAV9) with the HGF gene to achieve enhanced productivity and expression levels that none of the individual components could achieve alone

Inventive Principle:

Principle #40Composite materials

2Reliability

If conventional AAV vectors are administered intrathecally, then the vector can reach the target area, but the HGF protein expression efficiency is significantly low

Engineering Contradiction:

ImproveHGF protein expression levelVSAvoidAAV particle production efficiency

Core Design Contradiction:

ReliabilityVSProductivity

Solution Approach 1:

The patent optimizes expression reliability by changing the promoter parameter to CMV or EF-1α and the polyadenylation sequence parameter to hGH or bGH, which specifically enhance HGF protein expression efficiency when administered intrathecally

Inventive Principle:

Principle #35Parameter changes

Solution Approach 2:

The patent uses specific promoters (CMV or EF-1α) as intermediaries to mediate between the AAV vector delivery system and the HGF gene, enabling efficient transcription and high-level protein expression in the intrathecal environment

Inventive Principle:

Principle #24Intermediary (Mediator)

Data Source

PatentUS20260015628A1AAV vector optimized for intrathecal administration into which hepatocyte growth factor gene is introduced

Publication Date: 2026.01.15 HELIXMITH CO LTD

AI summary

The present invention relates to an adeno-associated virus (AAV) vector optimized for intrathecal administration into which a hepatocyte growth factor gene is introduced. The AAV vector of the present invention and AAV particles produced through the vector have excellent productivity of AAV particles comprising a hepatocyte growth factor gene, and when administered intrathecally to a mammal, the expression efficiency of hepatocyte growth factor is also very high. Therefore, the AAV vector of the present invention and the AAV particles induce the expression of hepatocyte growth factor protein when administered intrathecally, and thus can be effectively used in the prevention or treatment of related diseases.