Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles. - Wikidata - awgldk - TriplyDB

Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.

Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.

http://www.wikidata.org/entity/Q42029205

about

Delivery of RNAi Therapeutics to the Airways-From Bench to Bedside Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency Liver-targeted gene therapy: Approaches and challenges Barriers to inhaled gene therapy of obstructive lung diseases: A review Neuron-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 targeting Gene 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 efficacy Disorders 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 pigmentosa Gene-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.

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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.

http://www.wikidata.org/entity/Q42029205

description

2012 nî lūn-bûn

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name

Sustained miRNA-mediated knock ...... n global liver miRNA profiles.

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Sustained miRNA-mediated knock ...... n global liver miRNA profiles.

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type

label

Sustained miRNA-mediated knock ...... n global liver miRNA profiles.

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Sustained miRNA-mediated knock ...... n global liver miRNA profiles.

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prefLabel

Sustained miRNA-mediated knock ...... n global liver miRNA profiles.

@en

Sustained miRNA-mediated knock ...... n global liver miRNA profiles.

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Molecular Therapy

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Sustained miRNA-mediated knock ...... on global liver miRNA profiles

@en

P2093

Alisha Gruntman

http://www.w3.org/2001/XMLSchema#string

Christian Mueller

http://www.w3.org/2001/XMLSchema#string

Keith Blomenkamp

http://www.w3.org/2001/XMLSchema#string

Lina Song

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Qiushi Tang

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Terence R Flotte

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P2860

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

A comparison of normalization methods for high density oligonucleotide array data based on variance and bias

Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

Processing of primary microRNAs by the Microprocessor complex

A dicer-independent miRNA biogenesis pathway that requires Ago catalysis

Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs

Collapse of Germline piRNAs in the Absence of Argonaute3 Reveals Somatic piRNAs in Flies

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver

Argonaute2 suppresses Drosophila fragile X expression preventing neurogenesis and oogenesis defects.

Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo.

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590-600

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10.1038/MT.2011.292

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3

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20

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Jeffrey Teckman

Phillip D. Zamore

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2012-01-17T00:00:00Z

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221754418

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22252449

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3293602

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