Gene Therapy

Abstract

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.