Parkinson's disease gene therapy: success by design meets failure by efficacy (Q26851325)

Crossref

Marc S Weinberg

series ordinal

2

1 reference

Crossref

R. Jude Samulski

series ordinal

3

1 reference

Crossref

language of work or name

1 reference

March 2014

1 reference

1 March 2014

6 references

25 April 2019

25 June 2019

12 September 2019

19 September 2019

29 October 2020

8 October 2022

7 references

25 April 2019

25 June 2019

12 September 2019

19 September 2019

29 October 2020

8 October 2022

22

1 reference

issue

3

1 reference

page(s)

487-97

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Creative Commons Attribution-NonCommercial-ShareAlike 3.0 Unported

copyright license

6 references

25 April 2019

25 June 2019

12 September 2019

19 September 2019

29 October 2020

on focus list of Wikimedia project

8 October 2022

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copyrighted

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ScienceSource

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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Expression, bioactivity, and safety 1 year after adeno-associated viral vector type 2-mediated delivery of neurturin to the monkey nigrostriatal system support cere-120 for Parkinson's disease

28 September 2017

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Dynamic changes in presynaptic and axonal transport proteins combined with striatal neuroinflammation precede dopaminergic neuronal loss in a rat model of AAV alpha-synucleinopathy.

28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

28 September 2017

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Enhanced gene transfer efficiency in the murine striatum and an orthotopic glioblastoma tumor model, using AAV-7- and AAV-8-pseudotyped vectors

28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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Protection and regeneration of nigral dopaminergic neurons by neurturin or GDNF in a partial lesion model of Parkinson's disease after administration into the striatum or the lateral ventricle

28 September 2017

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Gene transfer provides a practical means for safe, long-term, targeted delivery of biologically active neurotrophic factor proteins for neurodegenerative diseases

28 September 2017

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Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC.

28 September 2017

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Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

31 May 2018

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Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120).

31 May 2018

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Gene therapy for Parkinson's disease

28 November 2018

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Gene therapy for aromatic L-amino acid decarboxylase deficiency

28 November 2018

1 reference

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28 November 2018

1 reference

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Transgene expression, bioactivity, and safety of CERE-120 (AAV2-neurturin) following delivery to the monkey striatum

28 November 2018

1 reference

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28 November 2018

1 reference

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28 November 2018

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Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys

28 November 2018

1 reference

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Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys.

28 November 2018

1 reference

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Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolism

28 November 2018

1 reference

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Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

28 November 2018

1 reference

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Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

28 November 2018

1 reference

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A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease

28 November 2018

1 reference

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Minocycline inhibits cytochrome c release and delays progression of amyotrophic lateral sclerosis in mice

28 November 2018

1 reference

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Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

28 November 2018

1 reference

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Parkinson-like neurodegeneration induced by targeted overexpression of alpha-synuclein in the nigrostriatal system.

28 November 2018

1 reference

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Intraventricular infusion of nerve growth factor as the cause of sympathetic fiber sprouting in sensory ganglia

28 November 2018

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Intracerebroventricular infusion of nerve growth factor in three patients with Alzheimer's disease

28 November 2018

1 reference

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Treatment of septic shock with human monoclonal antibody HA-1A. A randomized, double-blind, placebo-controlled trial. CHESS Trial Study Group.

28 November 2018

1 reference

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Gene therapy for Parkinson's disease

28 November 2018

1 reference

https://pubmed.ncbi.nlm.nih.gov/24356252

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

The long march of antisense

1 reference

https://pubmed.ncbi.nlm.nih.gov/24356252

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2013.281

1 reference

1 reference

1 reference

PubMed publication ID

24356252

1 reference