gene therapy – TechConnect Briefs

Dendrigraft Poly-L-lysine (d-PLL) Coated Gold Nanoparticles in Water for siRNA Delivery to Prostate Cancer Cells

Rahme K., Minassian G., Ghanem E., Souaid E., Guo J., O’Driscoll C.M., Holmes J.D., Notre Dame University (Louaize), LB

In this study we used dendrigraft poly-L-lysine (d-PLL) as a stabilising ligand for gold nanoparticles (AuNPs) in water. The d-PLL generation two (P2) with narrow molecular weight distribution of about 7 KD was obtained by [...]

HA-decorated nanoparticles as tumour-targeting nucleic acids carriers

Tirella A., Rios J.M., Tirelli N., University of Manchester, UK

The design of tools that allow for a targeted delivery of therapeutic cargos have a great potential in the treatment of many pathological conditions (e.g. cancer and autoimmune diseases). In the context of nucleic acids [...]

Gene Delivery and Transfection in Human Pancreatic Cancer Cells using Epidermal Growth Factor Receptor-Targeted Gelatin Nanoparticles

Xu J., Amiji M., Northeastern University, US

More than 32,000 patients are diagnosed with pancreatic cancer in the United States per year and the disease is associated with very high mortality. Although gene therapy has tremendous promise, the major challenge has been [...]

Labile Catalytic Packaging and Delivering of Short Interference RNA to Cancer Cells: Control of Gold Nanoparticles “out” of RNA Complexes

Chen A.M., Taratula O., Wei D., Minko T., He H., Rutgers, The State of New Jersey, US

We report a novel approach to efficiently package and deliver siRNAs with low generation dendrimers by using Au nanoparticles as a “labile catalytic” packaging agent. The Au nanoparticles helped low generation dendrimers to package siRNA [...]

Establishing a GMP manufacturing site for nanoparticles

Sepulveda C.A., Roth J.A., Lu C., Ji L., Ramesh R., O’Connor S., Sadeghi T., Flagge F., Baez O., Shen A.C., Mondesir E., Jenkins J., Shpall E., McMannis J.D., University Texas MD Anderson Cancer Center, US

An active clinical trial at MD Anderson Cancer Center uses DOTAP:Chol liposomes as a non-viral vector for gene therapy. This therapeutic product needs to be produced fresh for each administration. Originally, the manufacture of the [...]

Dicationic gemini surfactant gene delivery complexes contain cubic-lamellar mixed polymorphic phase

Foldvari M., Wettig S., Badea I., Verrall R., Bagonluri M., University of Saskatchewan, CA

The characterization of structural properties of novel dicationic (gemini) surfactant-based DNA complexes as micro/nano-scale self-assembling delivery systems for cutaneous gene therapy is described and discussed as related to measured transfection efficiencies. Previous small-angle x-ray scattering [...]

Cluster assembled nanostructured TiO2 film mediates efficient and safe retroviral gene transduction in primary adult human melanocytes for ex-vivo gene therapy

Carbone R., Marangi I., Zanardi A., Lanfrancone L., Minucci S., Pelicci P.G., Giorgetti L., Clerici E., Bongiorno G., Piseri P., Milani P., European Institute of Oncology, IT

Primary human melanocytes represent the precursor cells to melanoma and also specific targets of inherited and acquired pigmentation disorders (1,3). The possibility to genetically manipulate these cells for biological studies or therapy has not been [...]