AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease (Q27324755)

26560358

26 June 2017

title

AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease (English)

1 reference

26 June 2017

4

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7 June 2020

author name string

Martin L Katz

1

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7 June 2020

Luis Tecedor

2

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7 June 2020

Yonghong Chen

3

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7 June 2020

Elena Lysenko

5

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7 June 2020

Fred A Wininger

6

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7 June 2020

Whitney M Young

7

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7 June 2020

Gayle C Johnson

8

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7 June 2020

Rebecca E H Whiting

9

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7 June 2020

Joan R Coates

10

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7 June 2020

Beverly L Davidson

11

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7 June 2020

language of work or name

English

0 references

publication date

1 November 2015

1 reference

26560358

Science Translational Medicine

26 June 2017

published in

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7 June 2020

volume

7

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issue

313

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7 June 2020

page(s)

313ra180

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Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease

7 June 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

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

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Loss of Dishevelleds disrupts planar polarity in ependymal motile cilia and results in hydrocephalus

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease).

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Lysosomal membrane permeability stimulates protein aggregate formation in neurons of a lysosomal disease

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

A reversal learning task detects cognitive deficits in a Dachshund model of late-infantile neuronal ceroid lipofuscinosis.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Immune responses to AAV in clinical trials

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Cilia organize ependymal planar polarity

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

A frame shift mutation in canine TPP1 (the ortholog of human CLN2) in a juvenile Dachshund with neuronal ceroid lipofuscinosis

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Gene therapy for lysosomal storage diseases.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Determination of the substrate specificity of tripeptidyl-peptidase I using combinatorial peptide libraries and development of improved fluorogenic substrates

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Biochemical characterization of a lysosomal protease deficient in classical late infantile neuronal ceroid lipofuscinosis (LINCL) and development of an enzyme-based assay for diagnosis and exclusion of LINCL in human specimens and animal models

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

The ependyma: a protective barrier between brain and cerebrospinal fluid.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Distribution of extracellular tracers in perivascular spaces of the rat brain

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Targeted gene transfer into ependymal cells through intraventricular injection of AAV1 vector and long-term enzyme replacement via the CSF.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Ciliary motility in brain ventricular system and trachea of hamsters

2 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4968409

Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis

2 June 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

Adult ependymal cells are postmitotic and are derived from radial glial cells during embryogenesis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

Retinal pathology in a canine model of late infantile neuronal ceroid lipofuscinosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

Distribution of tripeptidyl peptidase I in human tissues under normal and pathological conditions

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

Progressive conduction defects and cardiac death in late infantile neuronal ceroid lipofuscinosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAC6191

Cardiac involvement in juvenile neuronal ceroid lipofuscinosis (Batten disease)

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26560358

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12 December 2020

based on heuristic

Identifiers

DOI

10.1126/SCITRANSLMED.AAC6191

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26560358%20AND%20SRC:MED&resulttype=core&format=json

OpenCitations bibliographic resource ID

7 June 2020

1334571

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26560358%20AND%20SRC:MED&resulttype=core&format=json

7 June 2020

26560358

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26560358%20AND%20SRC:MED&resulttype=core&format=json