Terkko Navigator / Gene Therapy

Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma

Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome

Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice

Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review

AAV-mediated delivery of actoxumab and bezlotoxumab results in serum and mucosal antibody concentrations that provide protection from C. difficile toxin challenge

Implications of hematopoietic stem cells heterogeneity for gene therapies

Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1

Gene delivery to the hypoglossal motor system: preclinical studies and translational potential

Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?

Comment on “Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine”

Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice

Liver targeting with rAAV7: balancing tropism with immune profiles

Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina

From macrophage biology to macrophage-based cellular immunotherapies

AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1

Correction: Targeted gene correction in the mdx mouse using short DNA fragments: towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle

Genome editing for Duchenne muscular dystrophy: a glimpse of the future?

Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells

Single-cell mapping of focused ultrasound-transfected brain

Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells

Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor

Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates

Double viral vector technology for selective manipulation of neural pathways with higher level of efficiency and safety

Correction: Conditional replication of oncolytic viruses based on detection of oncogenic mRNA

FEN1 inhibitor synergizes with low-dose camptothecin to induce increased cell killing via the mitochondria mediated apoptotic pathway

Restored microRNA-519a enhances the radiosensitivity of non-small cell lung cancer via suppressing EphA2

Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics

Host antibacterial defense of 6–10 Gy γ-irradiated mice subjected to lentiviral vector-based Gas5 gene therapy

Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse

Effects of oncolytic viruses and viral vectors on immunity in glioblastoma

Molecular targeting of vulnerable RNA sequences in SARS CoV-2: identifying clinical feasibility

Antitumor effects of IL-12 and GM-CSF co-expressed in an engineered oncolytic HSV-1

Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice

Self-amplifying RNA vaccines for infectious diseases

Intra-cisterna magna delivery of an AAV vector with the GLUT1 promoter in a pig recapitulates the physiological expression of SLC2A1

Long noncoding RNA CA3-AS1 suppresses gastric cancer migration and invasion by sponging miR-93-5p and targeting BTG3

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