Terkko Navigator / Gene Therapy

A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies

Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1

Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors

Transduction optimization of AAV vectors for human gene therapy of glaucoma and their reversed cell entry characteristics

MiR-21 derived from the exosomes of MSCs regulates the death and differentiation of neurons in patients with spinal cord injury

Retina transduction by rAAV2 after intravitreal injection: comparison between mouse and rat

Estrogen administration reduces the risk of pulmonary arterial hypertension by modulating the miR-133a signaling pathways in rats

Human umbilical cord perivascular cells-derived extracellular vesicles mediate the transfer of IGF-I to the liver and ameliorate hepatic fibrogenesis in mice

Analysis of recombinant adeno-associated viral vector shedding in sheep following intracoronary delivery

Downregulation of the human peripheral myelin protein 22 gene by miR-29a in cellular models of Charcot–Marie–Tooth disease

Inferring novel genes related to oral cancer with a network embedding method and one-class learning algorithms

Bicistronic transfer of CDKN2A and p53 culminates in collaborative killing of human lung cancer cells in vitro and in vivo

AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration

Inferring novel genes related to colorectal cancer via random walk with restart algorithm

Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis

Inside out: optimization of lipid nanoparticle formulations for exterior complexation and in vivo delivery of saRNA

Gene therapy of hematological disorders: current challenges

A next-generation sequencing method for gene doping detection that distinguishes low levels of plasmid DNA against a background of genomic DNA

Gene therapy for refractory angina and cell therapy for heart failure: experience of a Brazilian research group

Genetic therapies, human genetic enhancement, and … eugenics?

Design and applications of gene therapy vectors for mucopolysaccharidosis in Colombia

Effects of a combinatorial treatment with gene and cell therapy on retinal ganglion cell survival and axonal outgrowth after optic nerve injury

AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy

Safety profiling of genetically engineered Pim-1 kinase overexpression for oncogenicity risk in human c-kit+ cardiac interstitial cells

Bringing balance to the force–regulatable gene therapy for epilepsy

Feasibility of using NF1-GRD and AAV for gene replacement therapy in NF1-associated tumors

AAV-encoded CaV2.2 peptide aptamer CBD3A6K for primary sensory neuron-targeted treatment of established neuropathic pain

Bat adeno-associated viruses as gene therapy vectors with the potential to evade human neutralizing antibodies

Comparative efficacy of dermal fibroblast-mediated and direct adenoviral bone morphogenetic protein-2 gene therapy for bone regeneration in an equine rib model

Gene therapy progress and prospects: synthetic polymer-based systems

The radiation-inducible pE9 promoter driving inducible nitric oxide synthase radiosensitizes hypoxic tumour cells to radiation

Correction of a genetic defect in multipotent germline stem cells using a human artificial chromosome

Expression and maturation of Sendai virus vector-derived CFTR protein: functional and biochemical evidence using a GFP-CFTR fusion protein

Topical matrix-based siRNA silences local gene expression in a murine wound model

Complete knockdown of CCR5 by lentiviral vector-expressed siRNAs and protection of transgenic macrophages against HIV-1 infection

Human neural stem cells target and deliver therapeutic gene to experimental leptomeningeal medulloblastoma

Biocompatible micellar nanovectors achieve efficient gene transfer to vascular lesions without cytotoxicity and thrombus formation

In vivo gene silencing in solid tumors by targeted electrically mediated siRNA delivery