Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders (Q42428990)

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scientific article published on September 2010

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English	Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders	scientific article published on September 2010

Statements

scholarly article

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders (English)

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

0 references

author name string

E Aguilar Salegio

1

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Europe PubMed Central

PMC publication ID

31 October 2017

A P Kells

2

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

R M Richardson

3

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

P Hadaczek

4

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

J Forsayeth

5

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

J Bringas

6

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

S P Sardi

7

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

M A Passini

8

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

L S Shihabuddin

9

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

S H Cheng

10

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

M S Fiandaca

11

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

K S Bankiewicz

12

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

publication date

1 September 2010

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Europe PubMed Central

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31 October 2017

Human Gene Therapy

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

21

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Europe PubMed Central

PMC publication ID

31 October 2017

9

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

1093-1103

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Optimal region of the putamen for image-guided convection-enhanced delivery of therapeutics in human and non-human primates

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Controlled dissemination of AAV vectors in the primate brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Real-time MR imaging of adeno-associated viral vector delivery to the primate brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Detection of infusate leakage in the brain using real-time imaging of convection-enhanced delivery.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Results from a phase I safety trial of hAADC gene therapy for Parkinson disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Safety of real-time convection-enhanced delivery of liposomes to primate brain: a long-term retrospective

1 reference

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28 May 2018

Image-guided convection-enhanced delivery platform in the treatment of neurological diseases.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Current status of gene therapy trials for Parkinson's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

The "perivascular pump" driven by arterial pulsation is a powerful mechanism for the distribution of therapeutic molecules within the brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Expression and secretion of human glucocerebrosidase mediated by recombinant lentivirus vectors in vitro and in vivo: implications for gene therapy of Gaucher disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Recombinant adeno-associated virus: formulation challenges and strategies for a gene therapy vector.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Critical issues in gene therapy for neurologic disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Adeno-associated virus vectors can be efficiently produced without helper virus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Selective and rapid uptake of adeno-associated virus type 2 in brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Accurate differentiation of neuronopathic and nonneuronopathic forms of Niemann-Pick disease by evaluation of the effective residual lysosomal sphingomyelinase activity in intact cells.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Acid sphingomyelinase-deficient mice mimic the neurovisceral form of human lysosomal storage disease (Niemann-Pick disease)

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Sphingosylphosphocholine, a signaling molecule which accumulates in Niemann-Pick disease type A, stimulates DNA-binding activity of the transcription activator protein AP-1

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

28 May 2018

Cannula placement for effective convection-enhanced delivery in the nonhuman primate thalamus and brainstem: implications for clinical delivery of therapeutics.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Real-time imaging of convection-enhanced delivery of viruses and virus-sized particles

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Real-time image-guided direct convective perfusion of intrinsic brainstem lesions. Technical note

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Real-time visualization and characterization of liposomal delivery into the monkey brain by magnetic resonance imaging

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Lentiviral vectors for gene transfer to the central nervous system. Applications in lysosomal storage disease animal models

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Effect of hyperosmolar mannitol on convection-enhanced delivery into the rat brain stem

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Variables affecting convection-enhanced delivery to the striatum: a systematic examination of rate of infusion, cannula size, infusate concentration, and tissue-cannula sealing time

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Adenovirus-mediated gene transfer results in decreased lysosomal storage in brain and total correction in liver of aspartylglucosaminuria (AGU) mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2936496

16 August 2018

Gene therapy for lysosomal storage disorders

1 reference

https://pubmed.ncbi.nlm.nih.gov/20408734

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/20408734

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/20408734

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Distribution of AAV2-hAADC-transduced cells after 3 years in Parkinsonian monkeys

1 reference

https://pubmed.ncbi.nlm.nih.gov/20408734

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Creation of a mouse model for non-neurological (type B) Niemann-Pick disease by stable, low level expression of lysosomal sphingomyelinase in the absence of secretory sphingomyelinase: relationship between brain intra-lysosomal enzyme activity and c

1 reference

https://pubmed.ncbi.nlm.nih.gov/20408734

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1089/HUM.2010.040

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

31 October 2017

ResearchGate publication ID

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