In recent years, gene therapies have shown promise as a treatment for a variety of diseases, including those affecting the central nervous system (CNS). Now, researchers at the University of Washington have developed a new delivery system that could improve the efficacy of these therapies and make them more accessible to patients.
The new system is based on a common cold virus that has been modified to only infect cells that lack a specific protein. When this modified virus is injected into the CNS, it specifically targets and infects cells that are missing this protein. These are the cells that are most likely to be diseased or dysfunctional.
Once the virus has infected these cells, it delivers a therapeutic gene that can help to correct the underlying problem. In animal studies, the new delivery system has been shown to be effective in treating a range of CNS diseases, including Huntington’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS).
Importantly, the new system does not seem to cause any significant side effects. This is in contrast to many current treatments for CNS diseases, which can often be quite harsh and cause a range of undesirable side effects.
The new delivery system is still in the early stages of development and will need to undergo further testing before it can be used in humans. However, the results so far are very promising and suggest that this could be a potentially major advance in the treatment of CNS diseases.
The central nervous system (CNS) comprises the brain and the spinal cord. It is responsible for coordinating the body’s responses to internal and external stimuli. The delivery of gene therapies to the CNS is challenging due to the presence of the blood-brain barrier (BBB), which limits the entry of therapeutic genes into the brain.
Now, researchers from the University of Alabama at Birmingham (UAB) have developed a new delivery system that could overcome this obstacle. The system, known as “Adeno-Associated Virus 9 with Fusogenic Peptides” (AAV9-FP), is based on a modified form of the AAV9 virus.
When administered intravenously, AAV9-FP crosses the BBB and targets cells in the CNS. In a mouse model of Huntington’s disease, AAV9-FP delivered a therapeutic gene to the brain and reduced the level of the disease-causing protein.
The AAV9-FP system could potentially be used to deliver a wide range of gene therapies to the CNS, including treatments for neurodegenerative diseases, brain tumors, and stroke. The findings were published in the Journal of Clinical Investigation.