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Aptamer engineering of lentiviral vectors for cardiac gene therapies

用于心脏基因治疗的慢病毒载体适体工程

基本信息

批准号：
10759105
负责人：
George W Jackson
金额：
$ 28.97万
依托单位：
BASE PAIR BIOTECHNOLOGIES, INC.
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-08-11 至 2024-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10759105
关键词：
Address Adult Affect American Area Base Pairing Binding Biotechnology Cardiac Cardiac Myocytes Cause of Death Cell surface Cells Clinical Cost of Illness DNA Data Set Dependovirus Development Disease Dose Limiting Engineering Gene Expression Gene Transduction Agent Genes Genetic Genetic Diseases Genetic Medicine Genetic Predisposition to Disease Genome Heart Heart Diseases Hepatotoxicity Hypertrophic Cardiomyopathy Hypertrophy In Vitro Individual Lead Legal patent Lentivirus Vector Libraries Life Lilium Methods Names Patients Persons Phase Positioning Attribute Proteins RNA Small Business Innovation Research Grant Societies Specificity Sudden Death Surface Techniques Technology Testing Therapeutic Tissues Toxic effect Tropism Viral Vector aptamer clinical candidate clinical translation commercialization curative treatments delivery vehicle functional restoration gene correction gene delivery system gene therapy heart cell in vivo inherited cardiomyopathy innovation interest loss of function mutation novel overexpression rational design receptor technology platform translational impact vector

项目摘要

PROJECT SUMMARY/ABSTRACT Overview: Hypertrophic cardiomyopathy is the leading cause of sudden death in young individuals, where several loss-of-function mutations in key sarcomeric proteins cause hypertrophy. Gene therapy approaches that drive the overexpression of the correct genes can be used to remodel the heart tissue and restore function. However, no gene therapies have been developed for in vivo targeting of the heart to date. A key bottleneck in the field is the ability to engineer advanced viral vectors with prescribed tropism in the patient’s body. Lentiviral vector (LVV) engineering applies rational design principles to define components of the vector envelope, such as attachment proteins, leading to vectors with programmable tissue specificity and off-target activity.

项目总结/摘要概述：肥厚性心肌病是年轻人猝死的主要原因。个体，其中关键肌节蛋白中的几个功能缺失突变导致肥厚基因治疗方法，驱动正确的过度表达，基因可以用来重塑心脏组织和恢复功能。然而，没有基因迄今为止，已经开发了用于体内靶向心脏的疗法。关键瓶颈在该领域中的一个优势是能够设计出具有规定的嗜性的高级病毒载体，患者的身体。慢病毒载体（LVV）工程应用合理的设计原则，载体包膜的成分，例如附着蛋白，导致载体具有可编程的组织特异性和脱靶活性。