(Q40219437)

English

Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.

scientific article published on 2 May 2017

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scholarly article

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Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I. (English)

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

mucopolysaccharidosis I

1 reference

based on heuristic

inferred from title

12

object named as

R Scott McIvor

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5 July 2018

http://europepmc.org/abstract/PMC/5549804

Lalitha R Belur

1

object named as

Lalitha R Belur

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Kelly M Podetz-Pedersen

3

object named as

Kelly M Podetz-Pedersen

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Maureen S Riedl

4

object named as

Maureen Riedl

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Karen F Kozarsky

8

object named as

Karen F Kozarsky

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Lucy Vulchanova

5

object named as

Lucy Vulchanova

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

author name string

Alexa Temme

2

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Nicholas Robinson

6

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Leah R Hanson

7

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Paul J Orchard

9

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

William H Frey

10

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Walter C Low

11

1 reference

Europe PubMed Central

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http://europepmc.org/abstract/PMC/5549804

publication date

20 April 2017

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Human Gene Therapy

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

28

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

7

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Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

576-587

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

Intranasal Delivery of Recombinant NT4-NAP/AAV Exerts Potential Antidepressant Effect.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Gene therapy for neurologic manifestations of mucopolysaccharidoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Rapid transport within cerebral perivascular spaces underlies widespread tracer distribution in the brain after intranasal administration.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Mucopolysaccharidosis type I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Intranasal administration of plasmid DNA nanoparticles yields successful transfection and expression of a reporter protein in rat brain.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Platelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Analysis of transduction efficiency, tropism and axonal transport of AAV serotypes 1, 2, 5, 6, 8 and 9 in the mouse brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Overview of the mucopolysaccharidoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Intranasal delivery of biologics to the central nervous system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Transplant outcomes in mucopolysaccharidoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Intranasal delivery to the central nervous system: mechanisms and experimental considerations

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Intranasal delivery bypasses the blood-brain barrier to target therapeutic agents to the central nervous system and treat neurodegenerative disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Lysosomal storage diseases and the blood-brain barrier

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Delivery of interferon-beta to the monkey nervous system following intranasal administration

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Hematopoietic cell therapy for metabolic disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Gene therapy for mucopolysaccharidosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Enhanced mucosal immunoglobulin A response of intranasal adenoviral vector human immunodeficiency virus vaccine and localization in the central nervous system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Molecular genetics of mucopolysaccharidosis type I: diagnostic, clinical, and biological implications.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

The hippocampus as a spatial map. Preliminary evidence from unit activity in the freely-moving rat

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Trafficking of lysosomal enzymes.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

10 July 2018

Place units in the hippocampus of the freely moving rat

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

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Memory deficits associated with senescence: A neurophysiological and behavioral study in the rat

1 reference

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Intranasal delivery of siRNA to the olfactory bulbs of mice via the olfactory nerve pathway.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Endogenous anxiety and stress responses in water maze and Barnes maze spatial memory tasks

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Prevention of neuropathology in the mouse model of Hurler syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Hematopoietic origin of microglial and perivascular cells in brain.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Long-term outcome of Hurler syndrome following bone marrow transplantation.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5549804

20 August 2018

Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I

1 reference

https://pubmed.ncbi.nlm.nih.gov/28462595

12 December 2020

based on heuristic

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Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression

1 reference

https://pubmed.ncbi.nlm.nih.gov/28462595

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts

1 reference

https://pubmed.ncbi.nlm.nih.gov/28462595

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Male donor-derived cells in the brains of female sex-mismatched bone marrow transplant recipients: a Y-chromosome specific in situ hybridization study

1 reference

https://pubmed.ncbi.nlm.nih.gov/28462595

12 December 2020

based on heuristic

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Delivery of insulin-like growth factor-I to the rat brain and spinal cord along olfactory and trigeminal pathways following intranasal administration

1 reference

https://pubmed.ncbi.nlm.nih.gov/28462595

12 December 2020

based on heuristic

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Identifiers

10.1089/HUM.2017.187

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

1 reference

Europe PubMed Central

5 July 2018

http://europepmc.org/abstract/PMC/5549804

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