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Therapeutic reduction of ataxin 2 extends lifespan and rescues pathological features of ALS in TDP-43 transgenic mice| old_uid | 12003 |
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| title | Therapeutic reduction of ataxin 2 extends lifespan and rescues pathological features of ALS in TDP-43 transgenic mice |
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| start_date | 2016/09/05 |
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| schedule | 11h-12h |
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| online | no |
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| details | Conférence de l'ICM. Invité par Edor Kabashi et Séverine Boillée |
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| summary | Amyotrophic lateral sclerosis (ALS) is a rapidly progressing neurodegenerative disease
characterized by motor neuron loss, leading to paralysis and death 2-5 years following
disease onset. Nearly all ALS patients contain aggregates of the RNA-binding protein
TDP-43 in the brain and spinal cord and rare mutations in the gene encoding TDP-43
can cause ALS. There are no effective TDP-43-directed therapies for ALS or related TDP-
43 proteinopathies, such as frontotemporal dementia (FTD). Antisense oligonucleotides
(ASOs) and RNA interference approaches are emerging as attractive therapeutic
strategies in neurological diseases. Indeed, treating a rodent model of inherited ALS
(caused by a mutation in SOD1) with ASOs to SOD1 significantly slowed disease
progression. But since SOD1 mutations account for only ~2-5% of ALS cases, additional
therapeutic strategies are needed. Silencing TDP-43 itself is probably not warranted
given its critical normal cellular functions. Here we present an unexpectedly powerful
alternative therapeutic strategy for ALS, by targeting ataxin 2. |
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| responsibles | Oliviero |
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