CORDIS Project
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This project develops advanced lentiviral vectors for targeted gene therapy in motor neurons, aiming to treat conditions like amyotrophic lateral sclerosis. The vectors are engineered for high specificity and efficiency, potentially leading to significant advancements in neuroprotective therapies.
Lentiviral vectors pseudotyped with the rabies-G envelope have been used for the delivery of therapeutic transgenes via the peripheral intamuscular route, increasing lifespan and reducing neuromuscular deficits in mouse models of both amyotrophic lateral sclerosis and spinal muscular atrophy.
Despite these successes clinical translation of gene therapy for ALS with these and other vectors with retrograde transport such as AAV2 has been hampered as a result of limitations experienced with gene t…
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ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France, Paris
Type: Research institute
Activity type: Research Organisations
SME: No
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