Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches (Q35796587)

26123042

10 August 2017

title

Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches (English)

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26123042

10 August 2017

main subject

spinal muscular atrophy

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1 reference

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1

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14 June 2020

Serena Pagliarani

6

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14 June 2020

Federica Rizzo

7

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14 June 2020

Franco Pagani

9

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14 June 2020

Nereo Bresolin

10

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14 June 2020

Giacomo P Comi

11

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14 June 2020

Stefania Corti

12

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14 June 2020

author name string

Chiara Simone

2

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14 June 2020

Sara Dametti

3

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14 June 2020

Sabrina Salani

4

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14 June 2020

Gianna Ulzi

5

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14 June 2020

Emanuele Frattini

8

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14 June 2020

language of work or name

English

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publication date

30 June 2015

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10 August 2017

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14 June 2020

volume

5

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14 June 2020

page(s)

11746

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Creative Commons Attribution 4.0 International

14 June 2020

copyright license

start time

30 June 2015

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April 2022 Public Data File from Crossref

copyright status

copyrighted

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cites work

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1 reference

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Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates

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11 July 2018

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

11 July 2018

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A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

11 July 2018

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Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

11 July 2018

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RNA-based therapeutic approaches for coagulation factor deficiencies

11 July 2018

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

11 July 2018

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A positive modifier of spinal muscular atrophy in the SMN2 gene

11 July 2018

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

11 July 2018

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11 July 2018

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Induced pluripotent stem cells from a spinal muscular atrophy patient

11 July 2018

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Refined characterization of the expression and stability of the SMN gene products.

11 July 2018

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SMN mRNA and protein levels in peripheral blood: biomarkers for SMA clinical trials

11 July 2018

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Characterization of functional domains of the SMN protein in vivo

11 July 2018

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11 July 2018

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Identifiers

DOI

10.1038/SREP11746

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14 June 2020

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14 June 2020

26123042

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