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Gene Therapy, Very Encouraging

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Old 07-09-2019, 08:58 AM   #1
ashleyk
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Default Gene Therapy, Very Encouraging

This seems like the best news I've seen although it's not clear to me if this would work for advanced PD. If this gene therapy works in monkeys, why not humans? How many years of clinical trials must we wait for now?

Vector-mediated l-3,4-dihydroxyphenylalanine delivery reverses motor impairments in a primate model of Parkinson’s disease


Vector-mediated l-3,4-dihydroxyphenylalanine delivery reverses motor impairments in a primate model of Parkinson’s disease | Brain | Oxford Academic



In summary, the results obtained in this study provide proof-of-principle for AAV-mediated l-DOPA delivery as a therapeutic strategy for Parkinson’s disease. This refined version of classic l-DOPA treatment is attractive in that it is based on a single intervention and targeted to the site in the brain where dopamine is most needed, i.e. the striatum. The constant local supply of l-DOPA obtained with this approach holds promise as a therapeutic intervention that can provide long-lasting clinical improvement and, in the best cases, also avoid the appearance of motor fluctuations and dyskinetic side effects associated with standard oral dopaminergic medication. This gene therapy approach may thus offer the possibility to prolong and sustain the ‘good years’ many patients with Parkinson’s disease experience during the initial stages of l-DOPA therapy.
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Old 07-09-2019, 11:40 PM   #2
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Thanks ashleyk.

One thing which could delay its use in people is the stereotactic surgery which was used to introduce the vector into macaque brains. Getting approval for clinical trials of that in people may not be easy.
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Old 07-16-2019, 10:31 AM   #3
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Default PR001 Gets Fast Track Status for Parkinson Disease With GBA1 Mutation

PR001 Gets Fast Track Status for Parkinson Disease With GBA1 Mutation

PR1 Gets Fast Track Status for Parkinson Disease With GBA1 Mutation - Neurology Advisor


The Food and Drug Administration (FDA) has granted Fast Track designation to PR001 (Prevail Therapeutics) for the treatment of Parkinson disease patients with a GBA1 mutation (PD-GBA).
PR001 is an investigational single-dose gene therapy that utilizes an AAV9 viral vector to deliver the GBA1 gene to a patient’s cells. Mutations in the GBA1 gene can lead to a deficiency of beta-glucocerebrosidase, leading to an accumulation of glycolipids and lysosomal dysfunction in CNS cells. The Company believes that this is what leads to the inflammation and neurodegeneration observed in patients with PD-GBA.
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Old 07-16-2019, 03:34 PM   #4
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Sounds very good. Thanks, AshleyK.
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