Ectopic Receptor Expression for Targeted Gene Delivery
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Solution Overview
Problem
Current gene therapy methods are limited in their ability to effectively target and deliver compounds to non-native cells that do not endogenously express certain cell surface receptors, hindering the therapeutic potential for diseases involving these receptors.
Innovation Solution
The use of gene therapy vectors to ectopically express cell surface receptors in non-native cells, combined with conjugate groups that bind to these receptors, such as modified oligonucleotides and ligands like GalNAc, enables targeted delivery of therapeutic compounds to these cells.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional gene therapy methods are used, then delivery to cells expressing endogenous receptors is achieved, but delivery to non-native cells lacking these receptors is ineffective
Solution Approach 1:
The patent changes the biological parameter of receptor expression by using gene therapy vectors to ectopically express cell surface receptors in non-native cells. This transforms cells that originally lacked the receptors into cells that express them, enabling subsequent targeted delivery of conjugated compounds to these previously inaccessible target cells.
Solution Approach 2:
The patent applies preliminary action by first introducing the receptor gene into non-native cells via gene therapy vectors before administering the conjugated compound. This pre-expression of receptors creates the necessary binding sites on the target cells, ensuring that the subsequent compound delivery can effectively reach the intended non-native cells.
2Adaptability or versatility
If gene therapy vectors are used to ectopically express receptors, then targeted delivery to non-native cells is enabled, but the system complexity increases
Solution Approach 1:
The patent employs a universal approach by using conjugated compounds with receptor-specific ligands that can target any cell type expressing the corresponding receptor. This multi-functional system allows the same delivery mechanism to be applied across different disease models and cell types, reducing overall system complexity despite the added gene therapy component.
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 allows for precise reduction of target genes in non-native cells, offering a therapeutic benefit in treating or preventing diseases by enhancing the expression of specific receptors and facilitating the delivery of compounds that can modulate gene expression.
Implementation Method 1
the conjugate group comprises a ligand that binds to the receptor
Data Source
AI summary
Provided herein are methods, compounds, and compositions useful for targeted delivery of compounds to non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions are useful, for example, in gene therapy mediated ectopic expression of cell surface receptors and targeted delivery of compounds, such as conjugated oligonucleotides, to the non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions can be useful, for example, to treat, prevent, delay or ameliorate disease in an individual by targeted reduction of a gene of interest in the non-native cell ectopically expressing cell surface receptors.