Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. (Q47301525)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29187643%20AND%20SRC:MED&resulttype=core&format=json

5 April 2020

title

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase (English)

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

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5 April 2020

1 reference

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5 April 2020

Patrice Vidal

1 reference

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5 April 2020

Fanny Collaud

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5 April 2020

Severine Charles

1 reference

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5 April 2020

Christian Leborgne

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5 April 2020

Mathilde Cohen-Tannoudji

1 reference

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5 April 2020

Stephanie Astord

1 reference

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5 April 2020

Bernard Gjata

1 reference

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5 April 2020

Pauline Sellier

1 reference

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5 April 2020

Laetitia van Wittenberghe

1 reference

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5 April 2020

Alban Vignaud

1 reference

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5 April 2020

Florence Boisgerault

1 reference

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5 April 2020

Martine Barkats

1 reference

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5 April 2020

Mark A Kay

1 reference

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5 April 2020

language of work or name

English

0 references

publication date

1 November 2017

1 reference

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Science Translational Medicine

5 April 2020

published in

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5 April 2020

volume

1 reference

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5 April 2020

issue

418

1 reference

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Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs

5 April 2020

cites work

1 reference

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Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

23 May 2018

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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

23 May 2018

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Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity

23 May 2018

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Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.

23 May 2018

1 reference

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Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice

23 May 2018

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Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

23 May 2018

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Gene Therapy for Duchenne muscular dystrophy

23 May 2018

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Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors.

23 May 2018

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Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

23 May 2018

1 reference

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Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice

23 May 2018

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Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system

23 May 2018

1 reference

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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

23 May 2018

1 reference

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

23 May 2018

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New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond.

23 May 2018

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Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease

23 May 2018

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Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome

23 May 2018

1 reference

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A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease

23 May 2018

1 reference

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

23 May 2018

1 reference

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Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

23 May 2018

1 reference

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

The role of autophagy in the pathogenesis of glycogen storage disease type II (GSDII).

23 May 2018

1 reference

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Enhanced protein synthesis and secretion using a rational signal-peptide library approach as a tailored tool

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice

23 May 2018

1 reference

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SignalP 4.0: discriminating signal peptides from transmembrane regions

23 May 2018

1 reference

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Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression

23 May 2018

1 reference

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Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

23 May 2018

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A randomized study of alglucosidase alfa in late-onset Pompe's disease

23 May 2018

1 reference

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

23 May 2018

1 reference

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Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease

23 May 2018

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Neural deficits contribute to respiratory insufficiency in Pompe disease

23 May 2018

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Large-scale proteomics and phosphoproteomics of urinary exosomes

23 May 2018

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23 May 2018

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Suppression of autophagy in skeletal muscle uncovers the accumulation of ubiquitinated proteins and their potential role in muscle damage in Pompe disease

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Safety and efficacy of gene transfer for Leber's congenital amaurosis

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Prediction of MHC class II binding affinity using SMM-align, a novel stabilization matrix alignment method

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Enzyme replacement and enhancement therapies for lysosomal diseases

23 May 2018

1 reference

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Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

23 May 2018

1 reference

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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

23 May 2018

1 reference

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Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Regulated secretion of conventional lysosomes

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

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 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Mannose 6-phosphate receptors in sorting and transport of lysosomal enzymes

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

23 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

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

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Lysosomal acid alpha-glucosidase consists of four different peptides processed from a single chain precursor

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

Packaging of an AAV vector encoding human acid α-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

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

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5826611

High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa

14 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/29187643

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Cognitive decline in classic infantile Pompe disease: An underacknowledged challenge

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/29187643

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1126/SCITRANSLMED.AAM6375

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29187643%20AND%20SRC:MED&resulttype=core&format=json

5 April 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29187643%20AND%20SRC:MED&resulttype=core&format=json

5 April 2020

PubMed publication ID

29187643

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29187643%20AND%20SRC:MED&resulttype=core&format=json