Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.
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Delivery of RNAi Therapeutics to the Airways-From Bench to BedsideProgress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin DeficiencyLiver-targeted gene therapy: Approaches and challengesBarriers to inhaled gene therapy of obstructive lung diseases: A reviewNeuron-specific Sumo1-3 knockdown in mice impairs episodic and fear memories.Inhibition of hepatitis B virus replication by helper dependent adenoviral vectors expressing artificial anti-HBV pri-miRs from a liver-specific promoter.Gene Therapy 2017: Progress and Future Directions.The myeloid-binding peptide adenoviral vector enables multi-organ vascular endothelial gene targetingGene augmentation for adRP mutations in RHO.State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.Embedding siRNA sequences targeting apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacyDisorders of protein misfolding: alpha-1-antitrypsin deficiency as prototype.Multilineage transduction of resident lung cells in vivo by AAV2/8 for α1-antitrypsin gene therapy.Sustained knockdown of a disease-causing gene in patient-specific induced pluripotent stem cells using lentiviral vector-based gene therapy.Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders.Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin-related liver disease in mice.Mysteries of α1-antitrypsin deficiency: emerging therapeutic strategies for a challenging disease.Recombinant AAV as a platform for translating the therapeutic potential of RNA interference.Gene therapy in animal models of autosomal dominant retinitis pigmentosaGene-based therapy for alpha-1 antitrypsin deficiency.Recent advances in α-1-antitrypsin deficiency-related lung disease.Human RNAi pathway: crosstalk with organelles and cells.Broad spectrum of hepatocyte inclusions in humans, animals, and experimental models.Alpha-1 antitrypsin and liver disease: mechanisms of injury and novel interventions.Current status of gene therapy for α-1 antitrypsin deficiency.Gene therapy to treat cardiac arrhythmias.Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.Viral vectors for therapy of neurologic diseases.Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond.Stacking up CRISPR against RNAi for therapeutic gene inhibition.5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.Delivery technologies for genome editing.Production and discovery of novel recombinant adeno-associated viral vectors.Magnetic resonance elastography identifies fibrosis in adults with alpha-1 antitrypsin deficiency liver disease: a prospective study.Cellular Analysis of Silencing the Huntington's Disease Gene Using AAV9 Mediated Delivery of Artificial Micro RNA into the Striatum of Q140/Q140 Mice.Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency.Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.Construction and application of a novel hepatocyte-directed vector to simultaneous knockdown and overexpression of multiple genes.Design, Cloning, and In Vitro Screening of Artificial miRNAs to Silence Alpha-1 Antitrypsin.
P2860
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P2860
Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
2012年论文
@zh
2012年论文
@zh-cn
name
Sustained miRNA-mediated knock ...... n global liver miRNA profiles.
@en
Sustained miRNA-mediated knock ...... n global liver miRNA profiles.
@nl
type
label
Sustained miRNA-mediated knock ...... n global liver miRNA profiles.
@en
Sustained miRNA-mediated knock ...... n global liver miRNA profiles.
@nl
prefLabel
Sustained miRNA-mediated knock ...... n global liver miRNA profiles.
@en
Sustained miRNA-mediated knock ...... n global liver miRNA profiles.
@nl
P2093
P2860
P356
P1433
P1476
Sustained miRNA-mediated knock ...... on global liver miRNA profiles
@en
P2093
Alisha Gruntman
Christian Mueller
Keith Blomenkamp
Qiushi Tang
Terence R Flotte
P2860
P304
P356
10.1038/MT.2011.292
P577
2012-01-17T00:00:00Z