Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
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Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia BGene therapy for haemophiliaGene therapy for haemophiliaAllele-specific silencing of mutant Myh6 transcripts in mice suppresses hypertrophic cardiomyopathyA new generation of human artificial chromosomes for functional genomics and gene therapyClarifying lysosomal storage diseasesMicroRNAs in glioblastoma multiforme pathogenesis and therapeuticsTherapeutic Strategies for Neuropathic Pain: Potential Application of Pharmacosynthetics and OptogeneticsGene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translationImpaired vascular-mediated clearance of brain amyloid beta in Alzheimer's disease: the role, regulation and restoration of LRP1New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicalsThe gene therapy journey for hemophilia: are we there yet?State-of-the-art human gene therapy: part I. Gene delivery technologiesmiR-122 is a unique molecule with great potential in diagnosis, prognosis of liver disease, and therapy both as miRNA mimic and antimirAdeno-associated virus-mediated microRNA delivery and therapeuticsAdeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspectiveAnimal models of hemophiliaInducing brain regeneration from within: in vivo reprogramming of endogenous somatic cells into neuronsImpact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transductionNoninvasive and targeted gene delivery into the brain using microbubble-facilitated focused ultrasound.Gene delivery to adipose tissue using transcriptionally targeted rAAV8 vectorsAllele-specific silencing of mutant huntingtin in rodent brain and human stem cellsUltramicroscopy as a novel tool to unravel the tropism of AAV gene therapy vectors in the brainStructural Insights into Adeno-Associated Virus Serotype 5The structure of AAVrh32.33, a novel gene delivery vectorToward a therapy for mitochondrial diseaseGene therapy for hemophiliaCurrent prospects for RNA interference-based therapiesVEGF gene therapy: therapeutic angiogenesis in the clinic and beyondA universal system to select gene-modified hepatocytes in vivoSystemic Delivery of scAAV8-Encoded MiR-29a Ameliorates Hepatic Fibrosis in Carbon Tetrachloride-Treated MiceTerminal differentiation of cardiac and skeletal myocytes induces permissivity to AAV transduction by relieving inhibition imposed by DNA damage response proteinsProof of concept study with an HER-2 mimotope anticancer vaccine deduced from a novel AAV-mimotope library platformApplying gene silencing technology to contraceptionTargeted gene silencing to induce permanent sterilityCharacterization of a FGF19 variant with altered receptor specificity revealed a central role for FGFR1c in the regulation of glucose metabolismMassively parallel cis-regulatory analysis in the mammalian central nervous systemStructure of neurotropic adeno-associated virus AAVrh.8.The evolution of comprehensive haemophilia care in the United States: perspectives from the frontline.Enhancement of Blood-Brain Barrier Permeability and Delivery of Antisense Oligonucleotides or Plasmid DNA to the Brain by the Combination of Bubble Liposomes and High-Intensity Focused Ultrasound.
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Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
description
article científic
@ca
article scientifique
@fr
articolo scientifico
@it
artigo científico
@pt
bilimsel makale
@tr
scientific article published on May 2011
@en
vedecký článok
@sk
vetenskaplig artikel
@sv
videnskabelig artikel
@da
vědecký článek
@cs
name
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
@en
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
@nl
type
label
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
@en
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
@nl
prefLabel
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
@en
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
@nl
P356
P1476
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
@en
P2093
Federico Mingozzi
Katherine A High
P2888
P304
P356
10.1038/NRG2988
P577
2011-05-01T00:00:00Z