Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations. (Q39851951)

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9 February 2020

title

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations (English)

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9 February 2020

0 references

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Infantile neuronal ceroid lipofuscinosis

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Jansky–Bielschowsky disease

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2

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9 February 2020

Stephen M. Kaminsky

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9 February 2020

author name string

Mark M Souweidane

1

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9 February 2020

Lisa M Arkin

3

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9 February 2020

Dolan Sondhi

4

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9 February 2020

Neil R Hackett

5

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9 February 2020

Linda Heier

7

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9 February 2020

Barry E Kosofsky

8

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Stefan Worgall

9

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Ronald G Crystal

10

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9 February 2020

Michael G Kaplitt

11

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publication date

1 August 2010

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Journal of neurosurgery. Pediatrics

9 February 2020

published in

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volume

6

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issue

2

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page(s)

115-122

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Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

9 February 2020

cites work

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Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

22 September 2017

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22 September 2017

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22 September 2017

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22 September 2017

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Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

22 September 2017

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Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease

22 September 2017

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Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

22 September 2017

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Monitoring response to convection-enhanced taxol delivery in brain tumor patients using diffusion-weighted magnetic resonance imaging

22 September 2017

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Prospects for gene therapy in pediatric neurosurgery

22 September 2017

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Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis

25 September 2017

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Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

17 June 2018

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Real-time image-guided direct convective perfusion of intrinsic brainstem lesions. Technical note

20 August 2018

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Image-guided convection-enhanced delivery of gemcitabine to the brainstem

20 August 2018

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Induction of hyperintense signal on T2-weighted MR images correlates with infusion distribution from intracerebral convection-enhanced delivery of a tumor-targeted cytotoxin

20 August 2018

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Intracerebral infusate distribution by convection-enhanced delivery in humans with malignant gliomas: descriptive effects of target anatomy and catheter positioning

20 August 2018

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Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

20 August 2018

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Immune responses to AAV in a phase I study for Canavan disease

20 August 2018

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Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.

20 August 2018

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Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates

20 August 2018

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AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.

20 August 2018

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A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease

20 August 2018

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Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis

20 August 2018

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20 August 2018

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20 August 2018

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20 August 2018

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Assessing Disease Severity in Late Infantile Neuronal Ceroid Lipofuscinosis Using Quantitative MR Diffusion-Weighted Imaging

20 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/20672930

inferred from PubMed ID database lookup

12 December 2020

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Identifiers

DOI

10.3171/2010.4.PEDS09507

1 reference

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release_bdm36bso3fbjbd6z3vrtw5aib4

9 February 2020

Fatcat ID

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Fatcat

https://api.fatcat.wiki/v0/release/bdm36bso3fbjbd6z3vrtw5aib4

mapped directly with Wikidata item

24 November 2022

based on heuristic

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9 February 2020

PubMed publication ID

20672930

1 reference