At the Broad Institute, researchers are revealing deep biological insights about the causes of disease, which are leading to new ways to monitor, diagnose, and treat patients with greater precision. These include new tools that help predict diseases well before symptoms appear, and more effective therapeutics that reduce the chance of relapse or drug resistance. Learn more about #HowWeScience.
Delivering gene-based therapies
Designing new ways to package and deliver genes or gene-editing machinery to specific cells in the body
Gene-based therapies have the potential to treat a range of genetic diseases by introducing working copies of the genes that are causing the disease, by using gene editing to make genetic changes in cells that correct disease-causing mutations or lower risk of disease. To be effective, these therapies must deliver the new genes, or the gene-editing machinery, to enough of the right cells in the body to have a therapeutic effect. But researchers have struggled to design molecular vehicles that can deliver gene-based therapies safely and effectively to the right cells.
Scientists at the Broad Institute are tackling this problem using a variety of delivery strategies. Some are focused on viruses called adeno-associated viruses (AAVs), which have been engineered to act only as a one-time delivery vehicle and not cause disease. Broad researchers are further engineering AAVs to efficiently deliver gene-based therapies to tissues that have been difficult to reach, such as the muscle and brain. Others are developing non-viral systems to deliver enough gene-editing machinery to have a therapeutic effect in cells. Broad researchers are also working on delivery vehicles that are made of proteins produced by the human body or by bacteria.
A few gene-based therapies have been approved by the FDA for some rare genetic diseases. Broad scientists are optimizing existing delivery technologies and developing new ones for the next generation of gene-based therapies.
- Researchers reprogram gene therapy viral vectors to bind specific protein targets
- How one lab uses machine learning to solve a key gene therapy problem
- Bacterial injection system delivers proteins in mice and human cells
- Gene-delivering viruses reach the brain in a step toward gene therapy for neurological diseases
- Engineered particles efficiently deliver gene editing proteins to cells in mice
- A new gene-delivery vehicle could make gene therapy for muscle diseases safer and more effective
- Scientists harness human protein to deliver molecular medicines to cells