CORDIS Project
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This project focuses on improving gene therapy for motor neuron diseases like amyotrophic lateral sclerosis and spinal muscular atrophy using targeted lentiviral vectors. By investigating retrograde transport mechanisms and developing new vectors, the aim is to enhance gene delivery to motor neurons and test neuroprote…
Lentiviral vectors can be targeted to specific cell types by varying the envelope proteins, a process called pseudotyping.
The rabies-G pseudotyped lentivectors are useful for distal targeting of neurons because they are retrogradely transported to the nucleus where they integrate and express the transgene, as first demonstrated by us.
Motor neuron (MN) diseases are incurable neurodegenerative diseases causing progressive paralysis and premature death.
Most amyotrophic lateral sclerosis (ALS) ca…
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