Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders (Q34205620)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21811247%20AND%20SRC:MED&resulttype=core&format=json

9 February 2020

title

Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders (English)

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9 February 2020

10

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Arthur Burghes

14

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9 February 2020

author name string

Adam K Bevan

1

1 reference

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9 February 2020

Sandra Duque

2

1 reference

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9 February 2020

Kevin D Foust

3

1 reference

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9 February 2020

Pablo R Morales

4

1 reference

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9 February 2020

Lyndsey Braun

5

1 reference

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9 February 2020

Leah Schmelzer

6

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9 February 2020

Curtis M Chan

7

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9 February 2020

Mary McCrate

8

1 reference

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9 February 2020

Louis G Chicoine

9

1 reference

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9 February 2020

Paul N Porensky

11

1 reference

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9 February 2020

Stephen J Kolb

12

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9 February 2020

Jerry R Mendell

13

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9 February 2020

Brian K Kaspar

15

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9 February 2020

publication date

2 August 2011

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9 February 2020

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21811247%20AND%20SRC:MED&resulttype=core&format=json

9 February 2020

volume

19

1 reference

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9 February 2020

page(s)

1971-1980

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9 February 2020

issue

11

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Rapid and efficient generation of functional motor neurons from human pluripotent stem cells using gene delivered transcription factor codes.

9 February 2020

cites work

1 reference

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AAV9: over the fence and into the woods . . .

5 July 2018

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Engineering biomaterial systems to enhance viral vector gene delivery

5 July 2018

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Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system

5 July 2018

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Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

5 July 2018

1 reference

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Spinal muscular atrophy: a timely review

5 July 2018

1 reference

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Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery

5 July 2018

1 reference

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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

5 July 2018

1 reference

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Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer

5 July 2018

1 reference

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Survival of motor neuron protein over-expression prevents calpain-mediated cleavage and activation of procaspase-3 in differentiated human SH-SY5Y cells

5 July 2018

1 reference

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Standardizing the use of whole slide images in digital pathology.

5 July 2018

1 reference

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MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Expansive gene transfer in the rat CNS rapidly produces amyotrophic lateral sclerosis relevant sequelae when TDP-43 is overexpressed

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Cardiac defects contribute to the pathology of spinal muscular atrophy models

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Arrhythmia and cardiac defects are a feature of spinal muscular atrophy model mice

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Inflammation in neurodegenerative diseases

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Spinal muscular atrophy: new and emerging insights from model mice

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Follistatin gene delivery enhances muscle growth and strength in nonhuman primates

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Elevated expression of MeCP2 in cardiac and skeletal tissues is detrimental for normal development

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB).

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Non-cell autonomous toxicity in neurodegenerative disorders: ALS and beyond

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

5 July 2018

1 reference

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Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

5 July 2018

1 reference

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Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

5 July 2018

1 reference

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Non-cell autonomous influence of MeCP2-deficient glia on neuronal dendritic morphology

5 July 2018

1 reference

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A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

5 July 2018

1 reference

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Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice.

5 July 2018

1 reference

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Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat.

5 July 2018

1 reference

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SMN deficiency causes tissue-specific perturbations in the repertoire of snRNAs and widespread defects in splicing

5 July 2018

1 reference

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Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

5 July 2018

1 reference

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Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

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

5 July 2018

1 reference

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Molecular characterization of adeno-associated viruses infecting children

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Deletion of murine Smn exon 7 directed to liver leads to severe defect of liver development associated with iron overload

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Clades of Adeno-associated viruses are widely disseminated in human tissues.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Expression and subcellular localization of two isoforms of the survival motor neuron protein in different cell types

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Development of the blood vessels and extracellular spaces during postnatal maturation of rat cerebral cortex

5 July 2018

1 reference

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Neutralizing antibodies against adeno-associated viruses in inflammatory bowel disease patients: implications for gene therapy

5 July 2018

1 reference

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Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV).

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222525

A collaborative study on the natural history of childhood and juvenile onset proximal spinal muscular atrophy (type II and III SMA): 569 patients

28 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21811247

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Present techniques for antibody removal

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21811247

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2011.157

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21811247%20AND%20SRC:MED&resulttype=core&format=json

9 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21811247%20AND%20SRC:MED&resulttype=core&format=json

9 February 2020

1 reference