Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase (Q37730125)

22 August 2017

Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase (English)

1 reference

22 August 2017

Thomas J Conlon

1

1 reference

22 August 2017

Kirsten Erger

2

1 reference

22 August 2017

Stacy Porvasnik

3

1 reference

22 August 2017

Travis Cossette

4

1 reference

22 August 2017

Cheryl Roberts

5

1 reference

22 August 2017

Lynn Combee

6

1 reference

22 August 2017

Saleem Islam

7

1 reference

22 August 2017

Jeffry Kelley

8

1 reference

22 August 2017

Denise Cloutier

9

1 reference

22 August 2017

Nathalie Clément

10

1 reference

22 August 2017

Corinne R Abernathy

11

1 reference

22 August 2017

Barry J Byrne

12

1 reference

22 August 2017

1 September 2013

1 reference

Human gene therapy. Clinical development

22 August 2017

1 reference

22 August 2017

24

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22 August 2017

3

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22 August 2017

127-133

1 reference

Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease

22 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Pompe disease gene therapy.

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Vision 1 year after gene therapy for Leber's congenital amaurosis

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Neural deficits contribute to respiratory insufficiency in Pompe disease

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Different tissue reaction of oesophagus and diaphragm after mesh hiatoplasty. Results of an animal study

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

The role of the adeno-associated virus capsid in gene transfer

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Pompe disease diagnosis and management guideline.

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Production of recombinant adeno-associated virus vectors

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease).

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Gene therapy clinical trials worldwide to 2012 - an update.

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Repair of indirect inguinal hernias by "laparoscopic joining of the edges".

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Enzyme replacement therapy in the mouse model of Pompe disease

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003472

Case 15-2009: a man with coma after cardiac arrest

1 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24021025

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1089/HUMC.2013.147

1 reference

22 August 2017

1 reference

22 August 2017

1 reference