about
Advanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the BrainRecent advances and future prospects in choroideremiaCRISPR-Cas9: A Revolutionary Tool for Cancer ModellingGene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translationNeuroanatomy goes viral!Lipid Nanoparticles for Ocular Gene DeliveryThe adenovirus genome contributes to the structural stability of the virionGene therapy of inherited retinal degenerations: prospects and challengesA comprehensive review of retinal gene therapyHomologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transferStructural Insights into Adeno-Associated Virus Serotype 5Gene therapy for hemophiliaMybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in miceOptical control of mammalian endogenous transcription and epigenetic statesMassively parallel cis-regulatory analysis in the mammalian central nervous systemUsing injectoporation to deliver genes to mechanosensory hair cellsCRISPR-Cas9 knockin mice for genome editing and cancer modelingSomatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 SplicingMyosin7a deficiency results in reduced retinal activity which is improved by gene therapy.Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitationSynthesis and characterization of glycol chitosan DNA nanoparticles for retinal gene delivery.Lentiviral vectors can be used for full-length dystrophin gene therapyA viral over-expression system for the major malaria mosquito Anopheles gambiae.Harnessing the Potential of Human Pluripotent Stem Cells and Gene Editing for the Treatment of Retinal Degeneration.Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.Identification of postsynaptic phosphatidylinositol-4,5-bisphosphate (PIP2) roles for synaptic plasticity using chemically induced dimerizationIn vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuniEfficient production of dual recombinant adeno-associated viral vectors for factor VIII deliverySuppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosaNanoparticle-assisted targeted delivery of eye-specific genes to eyes significantly improves the vision of blind mice in vivo.Poly(β-amino ester)-nanoparticle mediated transfection of retinal pigment epithelial cells in vitro and in vivo.Modular dispensability of dysferlin C2 domains reveals rational design for mini-dysferlin molecules.Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application.Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6.Efficient transgene reconstitution with hybrid dual AAV vectors carrying the minimized bridging sequencesVector platforms for gene therapy of inherited retinopathies.Cystic fibrosis transmembrane conductance regulator with a shortened R domain rescues the intestinal phenotype of CFTR-/- mice.Random Insertion of mCherry Into VP3 Domain of Adeno-associated Virus Yields Fluorescent Capsids With no Loss of Infectivity.Capsid-expressing DNA in AAV vectors and its elimination by use of an oversize capsid gene for vector production.Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo.
P2860
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P2860
description
2009 nî lūn-bûn
@nan
2009 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած
@hyw
2009 թվականի նոյեմբերին հրատարակված գիտական հոդված
@hy
2009年の論文
@ja
2009年論文
@yue
2009年論文
@zh-hant
2009年論文
@zh-hk
2009年論文
@zh-mo
2009年論文
@zh-tw
2009年论文
@wuu
name
Effect of genome size on AAV vector packaging.
@ast
Effect of genome size on AAV vector packaging.
@en
type
label
Effect of genome size on AAV vector packaging.
@ast
Effect of genome size on AAV vector packaging.
@en
prefLabel
Effect of genome size on AAV vector packaging.
@ast
Effect of genome size on AAV vector packaging.
@en
P2093
P2860
P356
P1433
P1476
Effect of genome size on AAV vector packaging.
@en
P2093
Hongyan Yang
Peter Colosi
Zhijian Wu
P2860
P356
10.1038/MT.2009.255
P577
2009-11-10T00:00:00Z