Current Gene Therapy - Volume 15, Issue 3, 2015

Activation of hepatic stellate cells (HSCs) is a key event in pathogenesis of liver fibrosis and represents an orchestral interplay of inhibiting and activating transcription factors like forkhead box f1 (Foxf1), being described to stimulate pro-fibrogenic genes in HSCs. Here, we evaluated a lipidbased liver-specific delivery system (DBTC) suitable to transfer Foxf1 siRNA specifically to HSCs and examined its antifibrotic potentia Read More

New targets and therapeutic approaches for vascular targeted strategies in oncology are continuously explored. Endoglin, a co-receptor of TGF-β, is a known target, however, its silencing with vector-based RNA interference technology has not been evaluated yet. Therefore, in our study, we assembled plasmid DNA coding for shRNA against endoglin, and used gene electrotransfer as a delivery method to determine its antitu Read More

Wiskott-Aldrich syndrome (WAS) is a life-threatening disorder characterized by immunodeficiency, thrombocytopenia and eczema. Hematopoietic stem cell gene therapy can cure WAS but remains associated with the risk of leukemogenesis. In an effort to decrease the risk of gene-therapy induced leukemia associated with the use of first generation gamma-retroviral vectors, we have developed a series of codon-optimized Read More

Rare genetic diseases account for a considerable amount of fatalities and even their ‘mild’ or ‘non-lethal’ forms can produce drastic and undesirable discomfort to affected individuals. Various gene therapeutic approaches were tested for developing novel therapeutic concepts to treat these genetic diseases. Sleeping Beauty (SB) transposase represents one of these gene therapeutic systems which can be utilized for st Read More

Amyotrophic lateral sclerosis (ALS) is an incurable, chronic, fatal neuro-degenerative disease characterized by progressive loss of moto-neurons and paralysis of skeletal muscles. Reactivating dysfunctional areas is under earnest investigation utilizing various approaches. Here we present an innovative gene-cell construct aimed at reviving inert structure and function. Human umbilical cord blood cells (hUCBCs) transduced wit Read More

The feasibility of utilising bacteria as vectors for gene therapy is becoming increasingly recognised. This is primarily due to a number of intrinsic properties of bacteria such as their tumour targeting capabilities, their ability to carry large genetic or protein loads and the availability of wellestablished genetic engineering tools for a range of common lab strains. However, a number of issues relating to the use of bacteria Read More

The genetic engineering of T cells can lead to enhanced immune-mediated tumour destruction and harbors a great potential for the treatment of cancer. Recent efforts have centered on the design of receptors to re-direct the specificity of T cells towards tumour antigens by means of viral gene transfer. This strategy has shown great success in a number of phase one clinical trials. However, there are still challenges to over Read More

Epithelial Mesenchymal Transition (EMT) is an event where epithelial cells acquire mesenchymal- like phenotype. EMT can occur as a physiological phenomenon during tissue development and wound healing, but most importantly, EMT can confer highly invasive properties to epithelial carcinoma cells. The impairment of E-cadherin expression, an essential cell-cell adhesion protein, together with an increase in the expre Read More

Therapeutic revascularization had been considered as the most potential strategy for treating ischemic diseases. Reconstruction of mature blood vessels, which is the key for functional revascularization, is a complex process involving multiple angiogenesis factors. Attempts had been made to promote functional revascularization by delivering vectors or other macromolecules that could positively regulate angiogenesis; howeve Read More