(Q40318313)

http://europepmc.org/abstract/PMC/5383631

22 May 2018

title

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

1 reference

http://europepmc.org/abstract/PMC/5383631

22 May 2018

0 references

23

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Jean-Yves Hogrel

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Valerie E. Kelly

19

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Fulvio Mavilio

22

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

18

Nikhil Gopal

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

author name string

David L Mack

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Karine Poulard

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Melissa A Goddard

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Virginie Latournerie

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Jessica M Snyder

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Robert W Grange

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Matthew R Elverman

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Jérôme Denard

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Philippe Veron

9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Laurine Buscara

10

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Christine Le Bec

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Annie G Brezovec

13

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Hui Meng

14

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Lin Yang

15

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Fujun Liu

16

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Michael O'Callaghan

17

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Barbara K Smith

20

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Jennifer L Strande

21

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Federico Mingozzi

24

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Michael W Lawlor

25

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Ana Buj-Bello

26

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

Martin K Childers

27

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

publication date

22 February 2017

1 reference

http://europepmc.org/abstract/PMC/5383631

22 May 2018

published in

Molecular Therapy

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

volume

25

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

issue

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

page(s)

839-854

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

Novel findings associated with MTM1 suggest a higher number of female symptomatic carriers

13 June 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myotubular myopathy

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Gene therapy in monogenic congenital myopathies

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Common Data Elements for Muscle Biopsy Reporting

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Validity of a Neurological Scoring System for Canine X-Linked Myotubular Myopathy

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

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

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Recommendations for cardiac chamber quantification by echocardiography in adults: an update from the American Society of Echocardiography and the European Association of Cardiovascular Imaging.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Our journey to successful gene therapy for hemophilia B.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Gait characteristics in a canine model of X-linked myotubular myopathy

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Tissue triage and freezing for models of skeletal muscle disease.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Differential muscle hypertrophy is associated with satellite cell numbers and Akt pathway activation following activin type IIB receptor inhibition in Mtm1 p.R69C mice

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Automated fiber-type-specific cross-sectional area assessment and myonuclei counting in skeletal muscle

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Enzyme replacement therapy rescues weakness and improves muscle pathology in mice with X-linked myotubular myopathy.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Muscle function in a canine model of X-linked myotubular myopathy

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

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

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

D³: Data-Driven Documents

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

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

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

In vivo canine muscle function assay

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

MTM1 mutation associated with X-linked myotubular myopathy in Labrador Retrievers

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Centronuclear myopathies: a widening concept

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Centronuclear (myotubular) myopathy

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Characterization of the myotubularin dual specificity phosphatase gene family from yeast to human

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Changes in linear dimensions of the heart, relative to body weight, as measured by M-mode echocardiography in growing dogs

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Medical complications in long-term survivors with X-linked myotubular myopathy.

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5383631

Establishing clinical end points of respiratory function in large animals for clinical translation

20 August 2018

1 reference

Crossref

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2017.02.004

Contraction force generated by tarsal joint flexion and extension in dogs with golden retriever muscular dystrophy

7 January 2021

1 reference

Crossref

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2017.02.004

Validity of a neurological scoring system for canine X-linked myotubular myopathy.

7 January 2021

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/28237839

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1016/J.YMTHE.2017.02.004

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28237839%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

PubMed publication ID

28237839

1 reference