Gene Therapy

Gene Therapy

基因治疗

  • 3区 中科院分区
  • Q1 JCR分区

高引用文章

文章名称 引用次数
Understanding the molecular biology of intervertebral disc degeneration and potential gene therapy strategies for regeneration: a review 22
LncRNA FTX sponges miR-215 and inhibits phosphorylation of vimentin for promoting colorectal cancer progression 19
Long noncoding RNA Meg3 regulates cardiomyocyte apoptosis in myocardial infarction 19
Optimization of lentiviral vector production for scale-up in fixed-bed bioreactor 18
Inside out: optimization of lipid nanoparticle formulations for exterior complexation and in vivo delivery of saRNA 15
Inhibition of microRNA-495 suppresses chondrocyte apoptosis through activation of the NF-kappa B signaling pathway by regulating CCL4 in osteoarthritis 13
Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9 13
Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis 12
Improved therapeutic potential of MSCs by genetic modification 12
AAV-mediated NT-3 overexpression protects cochleae against noise-induced synaptopathy 11
Astrocyte-selective AAV gene therapy through the endogenous GFAP promoter results in robust transduction in the rat spinal cord following injury 9
Serotype survey of AAV gene delivery via subconjunctival injection in mice 9
Nanosphere-mediated co-delivery of VEGF-A and PDGF-B genes for accelerating diabetic foot ulcers healing in rats 9
Characterization of abscopal effects of intratumoral electroporation-mediated IL-12 gene therapy 8
A next-generation sequencing method for gene doping detection that distinguishes low levels of plasmid DNA against a background of genomic DNA 8
Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain 7
Effect of PAK1 gene silencing on proliferation and apoptosis in hepatocellular carcinoma cell lines MHCC97-H and HepG2 and cells in xenograft tumor 7
Feasibility of using NF1-GRD and AAV for gene replacement therapy in NF1-associated tumors 6
GDNF-mediated rescue of the nigrostriatal system depends on the degree of degeneration 6
HIV infection alters the human epigenetic landscape 6
Downreguation of FoxM1 by miR-214 inhibits proliferation and migration in hepatocellular carcinoma 6
More expansive gene transfer to the rat CNS: AAV PHP.EB vector dose-response and comparison to AAV PHP.B 5
Superior human hepatocyte transduction with adeno-associated virus vector serotype 7 5
Immunosuppression overcomes insulin- and vector-specific immune responses that limit efficacy of AAV2/8-mediated insulin gene therapy in NOD mice 5
Scaffold-mediated delivery for non-viral mRNA vaccines 5
MR-guided delivery of AAV2-BDNF into the entorhinal cortex of non-human primates 5
CCAT1 stimulation of the symmetric division of NSCLC stem cells through activation of the Wnt signalling cascade 5
AAV-encoded Ca(V)2.2 peptide aptamer CBD3A6K for primary sensory neuron-targeted treatment of established neuropathic pain 5
Ectopic BAT mUCP-1 overexpression in SKM by delivering a BMP7/PRDM16/PGC-1a gene cocktail or single PRMD16 using non-viral UTMD gene therapy 4
Characterization of neural stem cells modified with hypoxia/neuron-specific VEGF expression system for spinal cord injury 4
AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration 4
Polarized AAVR expression determines infectivity by AAV gene therapy vectors 4
Anti-angiogenic treatment of endometriosis via anti-VEGFA siRNA delivery by means of peptide-based carrier in a rat subcutaneous model 4
Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector 4
Improving therapeutic efficacy of IL-12 intratumoral gene electrotransfer through novel plasmid design and modified parameters 4
AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model 4
ILK promotes survival and self-renewal of hypoxic MSCs via the activation of lncTCF7-Wnt pathway induced by IL-6/STAT3 signaling 4
Effects of microRNA-292-5p on myocardial ischemia-reperfusion injury through the peroxisome proliferator-activated receptor-alpha/-gamma signaling pathway 4
Retina transduction by rAAV2 after intravitreal injection: comparison between mouse and rat 4
MiR-21 derived from the exosomes of MSCs regulates the death and differentiation of neurons in patients with spinal cord injury 3
Evaluation of the Glypican 3 promoter for transcriptional targeting of hepatocellular carcinoma 3
Primary T cells for mRNA-mediated immunotoxin delivery 3
Dual-targeted NIS polyplexes-a theranostic strategy toward tumors with heterogeneous receptor expression 3
Factors governing the transduction efficiency of adeno-associated virus in the retinal ganglion cells following intravitreal injection 3
Optimization of viral protein ratios for production of rAAV serotype 5 in the baculovirus system 3
Bat adeno-associated viruses as gene therapy vectors with the potential to evade human neutralizing antibodies 3
Safety and efficacy evaluations of an adeno-associated virus variant for preparing IL10-secreting human neural stem cell-based therapeutics 3
In vitro evaluation of a lentiviral two-step transcriptional amplification system using GAL4FF transactivator for gene therapy applications in bone repair 3
AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy 3
Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization 2