(Q27328843)

26 June 2017

title

Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy (English)

1 reference

Human Induced Pluripotent Stem Cells

26 June 2017

main subject

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inferred from title

spinal muscular atrophy

1 reference

1 reference

10

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Stefania Corti

1

object named as

Stefania Corti

1 reference

26 June 2017

14

Nereo Bresolin

1 reference

26 June 2017

Monica Nizzardo

2

1 reference

26 June 2017

Chiara Simone

3

1 reference

26 June 2017

Marianna Falcone

4

1 reference

26 June 2017

Martina Nardini

5

1 reference

26 June 2017

Dario Ronchi

6

1 reference

26 June 2017

Chiara Donadoni

7

1 reference

26 June 2017

Sabrina Salani

8

1 reference

26 June 2017

Giulietta Riboldi

9

1 reference

26 June 2017

Giorgia Menozzi

11

1 reference

26 June 2017

Clara Bonaglia

12

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26 June 2017

Federica Rizzo

13

1 reference

26 June 2017

Giacomo P Comi

15

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26 June 2017

Francesca Magri

10

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26 June 2017

language of work or name

English

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publication date

1 December 2012

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Science Translational Medicine

26 June 2017

published in

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26 June 2017

volume

4

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26 June 2017

issue

165

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26 June 2017

page(s)

165ra162

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Alternative splicing events are a late feature of pathology in a mouse model of spinal muscular atrophy

26 June 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

The gene encoding p44, a subunit of the transcription factor TFIIH, is involved in large-scale deletions associated with Werdnig-Hoffmann disease

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Induced pluripotent stem cell lines derived from human somatic cells

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Linear models and empirical bayes methods for assessing differential expression in microarray experiments

21 March 2017

1 reference

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Exploration, normalization, and summaries of high density oligonucleotide array probe level data

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Summaries of Affymetrix GeneChip probe level data

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Generation of germline-competent induced pluripotent stem cells

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

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

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Human induced pluripotent stem cells free of vector and transgene sequences

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Identification and characterization of a spinal muscular atrophy-determining gene

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Induced pluripotent stem cells from a spinal muscular atrophy patient

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Intramyocardial transplantation of undifferentiated rat induced pluripotent stem cells causes tumorigenesis in the heart

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Spinal muscular atrophy disease: a literature review for therapeutic strategies

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Embryonic stem cell-derived neural stem cells improve spinal muscular atrophy phenotype in mice.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Highly efficient differentiation and enrichment of spinal motor neurons derived from human and monkey embryonic stem cells

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Differentiation of spinal motor neurons from pluripotent human stem cells

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

A functional screen implicates microRNA-138-dependent regulation of the depalmitoylation enzyme APT1 in dendritic spine morphogenesis

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Neural stem cell transplantation can ameliorate the phenotype of a mouse model of spinal muscular atrophy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

FIRMA: a method for detection of alternative splicing from exon array data

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Genetic conversion of an SMN2 gene to SMN1: a novel approach to the treatment of spinal muscular atrophy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Mutation of Vps54 causes motor neuron disease and defective spermiogenesis in the wobbler mouse

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Stathmin and its phosphoprotein family: general properties, biochemical and functional interaction with tubulin.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

The survival motor neuron protein in spinal muscular atrophy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Correlation between severity and SMN protein level in spinal muscular atrophy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

The neurobiology of childhood spinal muscular atrophy.

2 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4722730

Impaired minor tri-snRNP assembly generates differential splicing defects of U12-type introns in lymphoblasts derived from a type I SMA patient.

2 June 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004108

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004108

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004108

Tumorigenic development of induced pluripotent stem cells in ischemic mouse brain.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004108

Neural stem cells LewisX+ CXCR4+ modify disease progression in an amyotrophic lateral sclerosis model.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004108

Soma-restricted products of the myelin proteolipid gene are expressed primarily in neurons in the developing mouse nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004108

Mutant SOD1(G93A) microglia are more neurotoxic relative to wild-type microglia

21 January 2018

1 reference

12 December 2020

International SMA consortium meeting. (26-28 June 1992, Bonn, Germany)

1 reference

12 December 2020

ALS pathogenesis: recent insights from genetics and mouse models

1 reference

12 December 2020

Motoneuron Transplantation Rescues the Phenotype of SMARD1 (Spinal Muscular Atrophy with Respiratory Distress Type 1)

1 reference

12 December 2020

SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN

1 reference

12 December 2020

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

1 reference

12 December 2020

10.1126/SCITRANSLMED.3004108

Identifiers

DOI

1 reference

OpenCitations bibliographic resource ID

26 June 2017

587243

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

587243

1 reference