(Q36013060)

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Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

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scholarly article

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Europe PubMed Central

PMC publication ID

11 August 2017

Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis (English)

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Europe PubMed Central

PMC publication ID

11 August 2017

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Infantile neuronal ceroid lipofuscinosis

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1 reference

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inferred from title

Shannon L Macauley

object named as

Shannon L Macauley

1

0 references

object named as

Mark S Sands

7

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Jonathan D. Cooper

object named as

Jonathan D Cooper

6

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author name string

Marie S Roberts

2

1 reference

Europe PubMed Central

PMC publication ID

11 August 2017

Andrew M Wong

3

1 reference

Europe PubMed Central

PMC publication ID

11 August 2017

Francesca McSloy

4

1 reference

Europe PubMed Central

PMC publication ID

11 August 2017

Adarsh S Reddy

5

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Europe PubMed Central

PMC publication ID

11 August 2017

publication date

24 February 2012

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Europe PubMed Central

PMC publication ID

11 August 2017

Annals of Neurology

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Europe PubMed Central

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11 August 2017

71

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Europe PubMed Central

PMC publication ID

11 August 2017

6

1 reference

Europe PubMed Central

PMC publication ID

11 August 2017

797-804

1 reference

Europe PubMed Central

PMC publication ID

11 August 2017

Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy

1 reference

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Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

1 reference

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Disruption of adaptive energy metabolism and elevated ribosomal p-S6K1 levels contribute to INCL pathogenesis: partial rescue by resveratrol

1 reference

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2 October 2017

Assessing the potential for AAV vector genotoxicity in a murine model

1 reference

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Molybdenum cofactor deficiency: Mutations in GPHN, MOCS1, and MOCS2.

1 reference

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2 October 2017

Palmitoyl:protein thioesterase (PPT1) inhibitors can act as pharmacological chaperones in infantile Batten disease

1 reference

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Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB

1 reference

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Cerebellar pathology and motor deficits in the palmitoyl protein thioesterase 1-deficient mouse

1 reference

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2 October 2017

A murine model of infantile neuronal ceroid lipofuscinosis-ultrastructural evaluation of storage in the central nervous system and viscera.

1 reference

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2 October 2017

Successive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis

1 reference

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Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

1 reference

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2 October 2017

Disruption of PPT1 or PPT2 causes neuronal ceroid lipofuscinosis in knockout mice

1 reference

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2 October 2017

Hematopoietic stem cell transplantation in infantile neuronal ceroid lipofuscinosis

1 reference

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2 October 2017

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

1 reference

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2 October 2017

DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors.

1 reference

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2 October 2017

DNA amplification of adeno-associated virus as a response to cellular genotoxic stress

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3369009

2 October 2017

Regional and cellular neuropathology in the palmitoyl protein thioesterase-1 null mutant mouse model of infantile neuronal ceroid lipofuscinosis

1 reference

https://api.crossref.org/works/10.1002%2FANA.23545

21 January 2018

Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis

1 reference

https://api.crossref.org/works/10.1002%2FANA.23545

21 January 2018

CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis.

1 reference

https://api.crossref.org/works/10.1002%2FANA.23545

21 January 2018

AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis.

1 reference

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21 January 2018

Neuroprotection of host cells by human central nervous system stem cells in a mouse model of infantile neuronal ceroid lipofuscinosis

1 reference

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21 January 2018

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

1 reference

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21 January 2018

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

1 reference

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21 January 2018

Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy

1 reference

https://api.crossref.org/works/10.1002%2FANA.23545

21 January 2018

Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver.

1 reference

https://api.crossref.org/works/10.1002%2FANA.23545

21 January 2018

Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII.

1 reference

https://api.crossref.org/works/10.1002%2FANA.23545

21 January 2018

AAV vector integration sites in mouse hepatocellular carcinoma.

1 reference

https://api.crossref.org/works/10.1002%2FANA.23545

21 January 2018

Percutaneous intravenous injection in neonatal mice

1 reference

https://pubmed.ncbi.nlm.nih.gov/22368049

12 December 2020

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Identifiers

10.1002/ANA.23545

1 reference

Europe PubMed Central

PMC publication ID

11 August 2017

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

11 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

11 August 2017

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