Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
Patent Information
- Authority / Receiving Office
- DE · DE
- Patent Type
- Patents
- Current Assignee / Owner
- CENT NAT DE LA RECH SCI (C N R S)
- Filing Date
- 2008-10-03
- Publication Date
- 2026-06-10
AI Technical Summary
Current methods for delivering therapeutic genes to motor neurons are hindered by the blood-brain barrier and invasive surgical procedures, leading to inefficient and risky gene transfer strategies for treating motor neuron diseases like spinal muscular atrophy and amyotrophic lateral sclerosis.
The use of recombinant AAV vectors, particularly self-complementary AAV9 vectors, administered via intravenous injection, bypasses the blood-brain barrier and achieves widespread gene delivery to spinal cord motor neurons and other CNS cells, including glial cells, through peripheral administration.
This method enables efficient and non-invasive gene transfer to motor neurons and glial cells, providing a new avenue for treating motor neuron diseases with a single intravenous injection, achieving broad gene delivery from the cervical to lumbar segments of the spinal cord.