Gene Therapy

Viral delivery and therapeutic strategies: retroviral vectors for gene therapy; adenovirus vectors for gene therapy; adeno-associated virus and adeno-associated virus vectors for gene delivery; ex vivo gene therapy using myoblasts and regulatable retroviral vectors; design and use of herpes simplex viral vectors for gene therapy; alphavirus-based vectors for vaccine and gene therapy applications. Nonviral delivery and therapeutic strategies: gene delivery with polyethylenimine; receptor-mediated gene transfer; modification of melanoma cells via ballistic gene therapy for vaccination; polymer-encapsulated cells for gene therapy; molecular interactions in lipids, DNA, and DNA-lipid complexes; bio-organic colloids - macromolecules, DNA, self-assembled particles and their complexes; synthetic virus systems for systemic gene therapy. Other therapeutic strategies and regulatory aspects: gene therapy applications of ribozymes; antisense oligonucleotide-based therapeutics; selectable markers for gene therapy; suicide gene therapy; regulatory aspects of gene therapy. Disease targets and therapeutic strategies: gene transfer into haematopoietic stem cells; gene therapy for haematopoietic disorders; gene therapy for cardiovascular disease and vascular grafts; gene therapy for cancer; barriers to efficient airway epithelial gene transfer -lessons from cystic fibrosis; gene transfer to the central nervous system; principles of gene therapy for inborn errors of metabolism; gene therapy and HIV-1 infection; gene delivery to the skin; DNA vaccines.