In vivo genome editing improves motor function and extends survival in a mouse model of ALS. (Q47153099)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

title

In vivo genome editing improves motor function and extends survival in a mouse model of ALS (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

amyotrophic lateral sclerosis

8 April 2020

main subject

1 reference

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

Freja K Ekman

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

Leah C Byrne

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

David Schaffer

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

author name string

David S Ojala

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

Prajit Limsirichai

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

publication date

20 December 2017

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

volume

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

issue

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

page(s)

eaar3952

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

describes a project that uses

8 April 2020

ImageJ

1 reference

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC5738228/fullTextXML

inferred from PubMed Central ID database lookup

cites work

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Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model

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Morpholino-mediated SOD1 reduction ameliorates an amyotrophic lateral sclerosis disease phenotype

23 May 2018

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A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

23 May 2018

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In vivo gene editing in dystrophic mouse muscle and muscle stem cells

23 May 2018

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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

23 May 2018

1 reference

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Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

23 May 2018

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Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.

23 May 2018

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Genome-wide inactivation of porcine endogenous retroviruses (PERVs).

23 May 2018

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Engineered CRISPR-Cas9 nucleases with altered PAM specificities

23 May 2018

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In vivo genome editing using Staphylococcus aureus Cas9

23 May 2018

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SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

23 May 2018

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Quantifying on- and off-target genome editing

23 May 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

23 May 2018

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Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

23 May 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

23 May 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

23 May 2018

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23 May 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Microglia induce motor neuron death via the classical NF-κB pathway in amyotrophic lateral sclerosis

23 May 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Necroptosis drives motor neuron death in models of both sporadic and familial ALS.

23 May 2018

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23 May 2018

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23 May 2018

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23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Degeneration and impaired regeneration of gray matter oligodendrocytes in amyotrophic lateral sclerosis.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

RNA-programmed genome editing in human cells

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

RNA-guided human genome engineering via Cas9

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Multiplex genome engineering using CRISPR/Cas systems

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Astrocytes from familial and sporadic ALS patients are toxic to motor neurons

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

In vivo genome editing restores haemostasis in a mouse model of haemophilia

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Wild-type and mutant SOD1 share an aberrant conformation and a common pathogenic pathway in ALS

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

A rapid and general assay for monitoring endogenous gene modification

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Modification of superoxide dismutase 1 (SOD1) properties by a GFP tag--implications for research into amyotrophic lateral sclerosis (ALS)

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

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

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

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

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

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

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Non-cell-autonomous effect of human SOD1 G37R astrocytes on motor neurons derived from human embryonic stem cells

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Effect of gene therapy on visual function in Leber's congenital amaurosis

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Non-cell autonomous effect of glia on motor neurons in an embryonic stem cell-based ALS model

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Wild-type microglia extend survival in PU.1 knockout mice with familial amyotrophic lateral sclerosis

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Antisense oligonucleotide therapy for neurodegenerative disease.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Onset and progression in inherited ALS determined by motor neurons and microglia

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

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

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Directed differentiation of embryonic stem cells into motor neurons

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Amyotrophic lateral sclerosis

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

The use of transgenic mouse models of amyotrophic lateral sclerosis in preclinical drug studies

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

ALS-linked SOD1 mutant G85R mediates damage to astrocytes and promotes rapidly progressive disease with SOD1-containing inclusions.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Neuroblastoma x spinal cord (NSC) hybrid cell lines resemble developing motor neurons

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

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

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS.

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Wild-Type Nonneuronal Cells Extend Survival of SOD1 Mutant Motor Neurons in ALS Mice

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5738228

Erratum: Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis

15 August 2018

1 reference

Crossref

https://api.crossref.org/works/10.1126/SCIADV.AAR3952

A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/29279867

12 December 2020

inferred from PubMed ID database lookup

Optimized protocols for isolation of primary motor neurons, astrocytes and microglia from embryonic mouse spinal cord.

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/29279867

12 December 2020

inferred from PubMed ID database lookup

Effect of alipogene tiparvovec (AAV1-LPL(S447X)) on postprandial chylomicron metabolism in lipoprotein lipase-deficient patients

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/29279867

12 December 2020

inferred from PubMed ID database lookup

Identifiers

DOI

10.1126/SCIADV.AAR3952

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29279867 AND SRC:MED&resulttype=core&format=json

8 April 2020

PubMed publication ID

29279867

1 reference