Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease
about
In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical applicationTargeted approaches to induce immune tolerance for Pompe disease therapyNew insights into therapeutic options for Pompe diseaseIn vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory function.Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established diseaseGenetic modification of airway progenitors after lentiviral gene delivery to the amniotic fluid of murine fetuses.Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene.Pompe disease gene therapy.Peptide-based inhibition of NF-κB rescues diaphragm muscle contractile dysfunction in a murine model of Duchenne muscular dystrophy.mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx MiceSpinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice.AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.Gene therapy approaches for lysosomal storage disease: next-generation treatment.The force-temperature relationship in healthy and dystrophic mouse diaphragm; implications for translational study design.Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Frequency and spectrum of genomic integration of recombinant adeno-associated virus serotype 8 vector in neonatal mouse liverIntrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease.Adeno-associated virus-mediated gene therapy for metabolic myopathy.Organ targeted prenatal gene therapy--how far are we?Fetal gene therapy: recent advances and current challenges.Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors.Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors.Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart.Prenatal gene therapy for the early treatment of genetic disorders
P2860
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P2860
Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease
description
2004 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2004 թվականի հունիսին հրատարակված գիտական հոդված
@hy
artículu científicu espublizáu en 2004
@ast
im Juni 2004 veröffentlichter wissenschaftlicher Artikel
@de
scientific journal article
@en
vedecký článok (publikovaný 2004/06/01)
@sk
vědecký článek publikovaný v roce 2004
@cs
wetenschappelijk artikel (gepubliceerd op 2004/06/01)
@nl
наукова стаття, опублікована в червні 2004
@uk
مقالة علمية (نشرت في يونيو 2004)
@ar
name
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@ast
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@en
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@nl
type
label
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@ast
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@en
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@nl
prefLabel
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@ast
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@en
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@nl
P2093
P3181
P356
P1433
P1476
Rescue of enzyme deficiency in ...... abolic myopathy: Pompe disease
@en
P2093
Barry J. Byrne
Denise A. Cloutier
Irene Zolotukhin
Mary Rucker
Stacy L. Porvasnik
Thomas J. Fraites
P304
P3181
P356
10.1242/DEV.01169
P407
P577
2004-06-01T00:00:00Z