| Many neurodegenerative diseases share common mechanisms of toxic RNA species accumulation or misfolded protein aggregation which lead to cell toxicity and death. Gene therapy has become an established way of treating genetic diseases and has shown several clinical successes for treatment of eye diseases, blood clotting disorders, neurodegenerative and neuromuscular diseases. Most current gene therapy approaches deliver the missing gene to the target disease organ or cell, leading long-term expression. These include AAV-mediated delivery of siRNAs engineered to bind and degrade toxic disease-associated RNA species that are formed in polyglutamine diseases like HD, ataxias or muscular dystrophies. Furthermore, the vectorization of antibodies that are expressed intracellularly (intrabodies) or extracellularly (secreted) are in development for multiple neurodegenerative diseases like ALS, HD or AD. Gene therapy delivery of such antibodies solves many challenges faced by “classical” antibodies such as long-term expression, crossing of the blood-brain barrier intracellular targeting and wide-spread brain distribution. |  |
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Sprekers: Alies Brandjes & Menno Spits, VectoryTX
Dit webinar vindt plaats op dinsdag 20 april om 14:00 uur
