Terkko Navigator / Gene Therapy

Potent CAR-T cells engineered with Sleeping Beauty transposon vectors display a central memory phenotype

Particulate mediators of the bystander effect linked to suicide and interferon-β transgene expression in melanoma cells

Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa

Retraction Note: Targeted suicide gene therapy for glioma using human embryonic stem cell-derived neural stem cells genetically modified by baculoviral vectors

Nanoparticle-based delivery of self-amplifying RNA

Mesenchymal stem cells as new perspective for the treatment of alcohol use disorder

Spotlight edition on South America

Reviewer recognition

Therapeutic potential of adenovirus-encoding brain-derived neurotrophic factor for spina bifida aperta by intra-amniotic delivery in a rat model

Utility of microminipigs for evaluating liver-mediated gene expression in the presence of neutralizing antibody against vector capsid

Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient-induced pluripotent stem cells

LncRNA OIP5-AS1 interacts with miR-363-3p to contribute to hepatocellular carcinoma progression through up-regulation of SOX4

Cancer gene therapy by NF-κB-activated cancer cell-specific expression of CRISPR/Cas9 targeting telomeres

CRISPR/Cas9 increases mitotic gene conversion in human cells

Adeno-associated virus vector enables safe and efficient Cas9 activation in neonatal and adult Cas9 knockin murine cochleae

miR-19a/19b improves the therapeutic potential of mesenchymal stem cells in a mouse model of myocardial infarction

Transposon-mediated generation of CAR-T cells shows efficient anti B-cell leukemia response after ex vivo expansion

Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components

Suppression of miR-203-3p inhibits lipopolysaccharide induced human intervertebral disc inflammation and degeneration through upregulating estrogen receptor α

A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases

Inhibition of miR-296-5p protects the heart from cardiac hypertrophy by targeting CACNG6

Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model

An engineered serum albumin-binding AAV9 capsid achieves improved liver transduction after intravenous delivery in mice

MicroRNA-204 silencing relieves pain of cervical spondylotic radiculopathy by targeting GDNF

AAV shedding after intracoronary delivery: just a safety concern?

Hsa_circ_0000285 functions as a competitive endogenous RNA to promote osteosarcoma progression by sponging hsa-miRNA-599

Treating genes and patients

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

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