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Silence the gene, save the cell: RNA interference as promising therapy for ALS

a gene replacement technology that allows the knock-down of all mutant SOD1 forms while permitting the expression of a normal type SOD1 protein that is resistant to RNA interference-based silencing. Both these effects are expressed long-term via delivery by a single lentiviral vector.

Aebischer is optimistic about the future of gene silencing as a potential therapy, particularly in incurable progressive neurological diseases such as ALS. "I would not be surprised to see, in the next ten years, this technology used for treating diseases of the nervous system, particularly diseases that involve toxic gain-of-function, such as inherited forms of Parkinson's disease or Huntington's disease," notes Aebischer. "But it's important to note that the safety of delivering lentiviral vectors to the nervous system will have to be carefully examined prior to treating patients."


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Source:Ecole Polytechnique F茅d茅rale de Lausanne


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