Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
By incorporating exogenous targeting peptides into the capsids of rAAV vectors, the challenge of targeting cardiac and skeletal muscle cells is addressed, resulting in enhanced transduction efficiency and specificity for muscle tissues.
WO2025106661A1 Publication Date: 2025-05-22THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
178 Cites 1 Cited by
Patent Information
- Application Number
- PCT/US2024/055910
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-06-18
- Filing Date
- 2024-11-14
- Publication Date
- 2025-05-22
AI Technical Summary
Technical Problem
Current AAV gene therapy vectors face challenges in effectively targeting and modulating gene expression in specific tissues, such as cardiac and skeletal muscle, limiting their application in treating muscle-related pathologies.
Method used
Development of recombinant adeno-associated virus (rAAV) vectors with modified capsids incorporating exogenous targeting peptides, specifically designed to enhance tropism and specificity for muscle cells, including cardiac and skeletal muscle.
Benefits of technology
The modified rAAV vectors demonstrate improved transduction efficiency and specificity for muscle tissues, potentially leading to more effective gene therapy outcomes for muscle-related disorders.
✦ Generated by Eureka AI based on patent content.
Abstract
Provided herein are compositions including muscle (cardiac and / or skeletal) cell-targeting peptides linked thereto or inserted in a targeting protein of a recombinant vector having at least one exogenous peptide comprising Xn – RGD-n-mer – Xm. Compositions providing such conjugates, targeting peptides, or recombinant vectors having an engineered capsid or envelope protein are provided as are uses thereof.
Need to check novelty before this filing date? Find Prior Art