Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates (Q35153080)

25358252

6 August 2017

title

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

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6 August 2017

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2

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7 June 2020

Alessandra Govoni

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Arthur Burghes

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7 June 2020

author name string

Kathrin Meyer

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7 June 2020

Leah Schmelzer

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Lyndsey Braun

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Vicki McGovern

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Shibi Likhite

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Olivia Michels

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Julie Fitzgerald

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Pablo Morales

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7 June 2020

Kevin D Foust

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Jerry R Mendell

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Brian K Kaspar

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language of work or name

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31 October 2014

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6 August 2017

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volume

23

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7 June 2020

issue

3

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7 June 2020

page(s)

477-487

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Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I

7 June 2020

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Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain

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Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates

28 July 2018

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Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

28 July 2018

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Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.

28 July 2018

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Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates

28 July 2018

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

28 July 2018

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Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy

28 July 2018

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

28 July 2018

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Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy

28 July 2018

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Delivery of Therapeutic Agents Through Intracerebroventricular (ICV) and Intravenous (IV) Injection in Mice

28 July 2018

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28 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

28 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

28 July 2018

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Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors

28 July 2018

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

28 July 2018

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

28 July 2018

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28 July 2018

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28 July 2018

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A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.

28 July 2018

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28 July 2018

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28 July 2018

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Identification of proximal spinal muscular atrophy carriers and patients by analysis of SMNT and SMNC gene copy number

28 July 2018

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The neurobiology of childhood spinal muscular atrophy.

28 July 2018

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The genetic component in child mortality

28 July 2018

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28 July 2018

1 reference

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28 July 2018

1 reference

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Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type-specific transgene expression in the spinal cord

27 September 2018

1 reference

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27 September 2018

1 reference

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Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

Comparison of Adeno-Associated Viral Vector Serotypes for Spinal Cord and Motor Neuron Gene Delivery

27 September 2018

1 reference

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

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

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

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

Regulation of SMN protein stability

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

The Trendelenburg position increases the spread and accelerates the onset of epidural anesthesia for Cesarean section

27 September 2018

1 reference

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Natural history in proximal spinal muscular atrophy. Clinical analysis of 445 patients and suggestions for a modification of existing classifications

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

Reversible molecular pathology of skeletal muscle in spinal muscular atrophy

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

SMN oligomerization defect correlates with spinal muscular atrophy severity

16 November 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25358252

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

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

PubMed

https://pubmed.ncbi.nlm.nih.gov/25358252

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Newborn and carrier screening for spinal muscular atrophy

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25358252

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2014.210

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25358252 AND SRC:MED&resulttype=core&format=json

7 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25358252 AND SRC:MED&resulttype=core&format=json

7 June 2020

25358252

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25358252 AND SRC:MED&resulttype=core&format=json