(Q38470242)

3 September 2017

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title

Current developments in gene therapy for amyotrophic lateral sclerosis (English)

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amyotrophic lateral sclerosis

3 September 2017

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author

Pamela Shaw

4

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3 September 2017

Aziza R Alrafiah

3

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Mimoun Azzouz

object named as

Mimoun Azzouz

5

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author name string

Joseph M Scarrott

1

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3 September 2017

Saúl Herranz-Martín

2

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3 September 2017

language of work or name

English

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publication date

10 May 2015

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Expert Opinion on Biological Therapy

3 September 2017

published in

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3 September 2017

volume

15

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3 September 2017

issue

7

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3 September 2017

page(s)

935-947

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

3 September 2017

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State of play in amyotrophic lateral sclerosis genetics

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

21 January 2018

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Pathogenesis/genetics of frontotemporal dementia and how it relates to ALS.

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

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Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved?

21 January 2018

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Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

21 January 2018

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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

21 January 2018

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A phase I study of OncoVEXGM-CSF, a second-generation oncolytic herpes simplex virus expressing granulocyte macrophage colony-stimulating factor.

21 January 2018

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Gene therapy for amyotrophic lateral sclerosis

21 January 2018

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Research advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis

21 January 2018

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Sporadic and hereditary amyotrophic lateral sclerosis (ALS).

21 January 2018

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Converging mechanisms in ALS and FTD: disrupted RNA and protein homeostasis

21 January 2018

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The product of C9orf72, a gene strongly implicated in neurodegeneration, is structurally related to DENN Rab-GEFs

21 January 2018

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Frequency of the C9orf72 hexanucleotide repeat expansion in patients with amyotrophic lateral sclerosis and frontotemporal dementia: a cross-sectional study

21 January 2018

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RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention

21 January 2018

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The C9orf72 GGGGCC repeat is translated into aggregating dipeptide-repeat proteins in FTLD/ALS.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

RAN proteins and RNA foci from antisense transcripts in C9ORF72 ALS and frontotemporal dementia

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

C9orf72 nucleotide repeat structures initiate molecular cascades of disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

The disease-associated r(GGGGCC)n repeat from the C9orf72 gene forms tract length-dependent uni- and multimolecular RNA G-quadruplex structures

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Unconventional translation of C9ORF72 GGGGCC expansion generates insoluble polypeptides specific to c9FTD/ALS

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Antisense proline-arginine RAN dipeptides linked to C9ORF72-ALS/FTD form toxic nuclear aggregates that initiate in vitro and in vivo neuronal death

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Aggregation-prone c9FTD/ALS poly(GA) RAN-translated proteins cause neurotoxicity by inducing ER stress

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Poly-dipeptides encoded by the C9orf72 repeats bind nucleoli, impede RNA biogenesis, and kill cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Amyotrophic lateral sclerosis associated with mutations in the CuZn superoxide dismutase gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Current hypotheses for the underlying biology of amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Is SOD1 loss of function involved in amyotrophic lateral sclerosis?

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

S[+] Apomorphine is a CNS penetrating activator of the Nrf2-ARE pathway with activity in mouse and patient fibroblast models of amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Histological evidence of protein aggregation in mutant SOD1 transgenic mice and in amyotrophic lateral sclerosis neural tissues

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Aggregation and Motor Neuron Toxicity of an ALS-Linked SOD1 Mutant Independent from Wild-Type SOD1

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Formation of high molecular weight complexes of mutant Cu, Zn-superoxide dismutase in a mouse model for familial amyotrophic lateral sclerosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Glial nuclear aggregates of superoxide dismutase-1 are regularly present in patients with amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Novel antibodies reveal inclusions containing non-native SOD1 in sporadic ALS patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Aberrant localization of FUS and TDP43 is associated with misfolding of SOD1 in amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Role of mutant SOD1 disulfide oxidation and aggregation in the pathogenesis of familial ALS

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Protein aggregation can inhibit clathrin-mediated endocytosis by chaperone competition

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

CSF and plasma amino acid levels in motor neuron disease: elevation of CSF glutamate in a subset of patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Riluzole, neuroprotection and amyotrophic lateral sclerosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

SOD1 mutants linked to amyotrophic lateral sclerosis selectively inactivate a glial glutamate transporter.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Abnormal exocytotic release of glutamate in a mouse model of amyotrophic lateral sclerosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Group I metabotropic glutamate autoreceptors induce abnormal glutamate exocytosis in a mouse model of amyotrophic lateral sclerosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Knocking down metabotropic glutamate receptor 1 improves survival and disease progression in the SOD1(G93A) mouse model of amyotrophic lateral sclerosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

An adverse property of a familial ALS-linked SOD1 mutation causes motor neuron disease characterized by vacuolar degeneration of mitochondria

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Mitochondrial damage revealed by immunoselection for ALS-linked misfolded SOD1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Amyotrophic lateral sclerosis-associated SOD1 mutant proteins bind and aggregate with Bcl-2 in spinal cord mitochondria

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

SOD1 mutation is associated with accumulation of neurofilaments in amyotrophic lateral sclerosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Transgenic mice carrying a human mutant superoxide dismutase transgene develop neuronal cytoskeletal pathology resembling human amyotrophic lateral sclerosis lesions

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Slowing of axonal transport is a very early event in the toxicity of ALS-linked SOD1 mutants to motor neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Partial loss of TDP-43 function causes phenotypes of amyotrophic lateral sclerosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

TDP-43 mutations in familial and sporadic amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

TARDBP mutations in individuals with sporadic and familial amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Mutations in the FUS/TLS gene on chromosome 16 cause familial amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Mutations in FUS, an RNA processing protein, cause familial amyotrophic lateral sclerosis type 6

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

ALS mutant FUS disrupts nuclear localization and sequesters wild-type FUS within cytoplasmic stress granules

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Antisense oligonucleotide therapy for neurodegenerative disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Motor neurone acetylcholinesterase release precedes neurotoxicity caused by systemic administration of excitatory amino acids and strychnine

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Presymptomatic treatment with acetylcholinesterase antisense oligonucleotides prolongs survival in ALS (G93A-SOD1) mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Acetylcholinesterase-transgenic mice display embryonic modulations in spinal cord choline acetyltransferase and neurexin Ibeta gene expression followed by late-onset neuromotor deterioration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Invited review: decoding the pathophysiological mechanisms that underlie RNA dysregulation in neurodegenerative disorders: a review of the current state of the art.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Neuronal dark matter: the emerging role of microRNAs in neurodegeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Viral vectors for gene delivery to the central nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Vascular endothelial growth factor overexpression delays neurodegeneration and prolongs survival in amyotrophic lateral sclerosis mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Treatment of motoneuron degeneration by intracerebroventricular delivery of VEGF in a rat model of ALS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Synergistic effects of GDNF and VEGF on lifespan and disease progression in a familial ALS rat model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Hepatocyte growth factor (HGF) attenuates gliosis and motoneuronal degeneration in the brainstem motor nuclei of a transgenic mouse model of ALS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Direct muscle delivery of GDNF with human mesenchymal stem cells improves motor neuron survival and function in a rat model of familial ALS

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

CNS-targeted viral delivery of G-CSF in an animal model for ALS: improved efficacy and preservation of the neuromuscular unit

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Granulocyte-colony stimulating factor improves outcome in a mouse model of amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Intraparenchymal spinal cord delivery of adeno-associated virus IGF-1 is protective in the SOD1G93A model of ALS

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Intraspinal cord delivery of IGF-I mediated by adeno-associated virus 2 is neuroprotective in a rat model of familial ALS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Therapeutic applications of mesenchymal stem cells for amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Viral delivery of antioxidant genes as a therapeutic strategy in experimental models of amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Adeno-associated virus-mediated delivery of a recombinant single-chain antibody against misfolded superoxide dismutase for treatment of amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Widespread and efficient transduction of spinal cord and brain following neonatal AAV injection and potential disease modifying effect in ALS mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

TDP-43 pathology in sporadic ALS occurs in motor neurons lacking the RNA editing enzyme ADAR2.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

A role for calpain-dependent cleavage of TDP-43 in amyotrophic lateral sclerosis pathology

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1044894

10.1517/14712598.2015.1044894

21 January 2018

Identifiers

DOI

1 reference

3 September 2017

1 reference