Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors.
about
Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studiesManufacturing of recombinant adeno-associated viral vectors for clinical trialsNew insights into therapeutic options for Pompe diseaseAdipocytes as a vehicle for ex vivo gene therapy: Novel replacement therapy for diabetes and other metabolic diseasesDisturbance of cardiac gene expression and cardiomyocyte structure predisposes Mecp2-null mice to arrhythmiasRetrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established diseaseImmunomodulatory gene therapy in lysosomal storage disordersAdeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia.Pompe disease gene therapy.Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockadeHypoglossal neuropathology and respiratory activity in pompe miceAAV vectors for cardiac gene transfer: experimental tools and clinical opportunities.Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe miceSystemic gene transfer to skeletal muscle using reengineered AAV vectorsSpinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice.Preclinical Development of New Therapy for Glycogen Storage Diseases.Correcting Neuromuscular Deficits With Gene Therapy in Pompe DiseaseGene therapy approaches for lysosomal storage disease: next-generation treatment.A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease.Gene therapy for cardiovascular manifestations of lysosomal storage diseases.Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice.Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease.Neural deficits contribute to respiratory insufficiency in Pompe disease.Adeno-associated virus-mediated gene therapy for metabolic myopathy.Therapeutic approaches in glycogen storage disease type II/Pompe Disease.Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase.The respiratory neuromuscular system in Pompe disease.Myocardial glycogen dynamics: new perspectives on disease mechanisms.Systemic delivery of adeno-associated viral vectors.RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1).Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice.Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction.Airway smooth muscle dysfunction in Pompe (Gaa-/- ) mice.
P2860
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P2860
Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors.
description
2007 nî lūn-bûn
@nan
2007年の論文
@ja
2007年学术文章
@wuu
2007年学术文章
@zh
2007年学术文章
@zh-cn
2007年学术文章
@zh-hans
2007年学术文章
@zh-my
2007年学术文章
@zh-sg
2007年學術文章
@yue
2007年學術文章
@zh-hant
name
Physiological correction of Po ...... ated virus serotype 1 vectors.
@en
Physiological correction of Po ...... ated virus serotype 1 vectors.
@nl
type
label
Physiological correction of Po ...... ated virus serotype 1 vectors.
@en
Physiological correction of Po ...... ated virus serotype 1 vectors.
@nl
prefLabel
Physiological correction of Po ...... ated virus serotype 1 vectors.
@en
Physiological correction of Po ...... ated virus serotype 1 vectors.
@nl
P2093
P356
P1433
P1476
Physiological correction of Po ...... ated virus serotype 1 vectors.
@en
P2093
Barry J Byrne
Cathryn Mah
Christina A Pacak
David D Fuller
Denise A Cloutier
Kerry O Cresawn
Lara R Deruisseau
Sean Germain
P304
P356
10.1038/SJ.MT.6300100
P577
2007-01-23T00:00:00Z