Concepts in gene therapy

This comprehensive work explores various methods of gene delivery, including retroviral, adenoviral, and adeno-associated viral vectors, as well as herpes simplex virus-mediated gene transfer to neurons. It discusses targeted gene delivery systems, particularly for hepatocytes, and particle-mediated gene delivery in cancer research. The book also addresses gene inactivation techniques through homologous recombination, potential therapies using antisense oligonucleotides for neurodegenerative diseases, and the application of synthetic ribozymes in gene therapy. Cellular target systems are examined, with a focus on gene transfer in hematopoietic cells and the liver's role in gene therapy. Specific case studies include gene therapy for Gaucher disease using genetically engineered primary myoblasts and the in vivo secretion of therapeutic proteins from neo-organs. The text delves into genetic diseases, presenting strategies and challenges in gene therapy for cystic fibrosis, dystrophin deficiency, familial hypercholesterolemia, and lysosomal storage disorders. It also covers non-genetic and infectious diseases, including gene therapy approaches for arthritis and HIV-1. In the cancer section, the book discusses suicide genes, tumor cell vaccines utilizing genetically modified cells, the impact of dosage on immunotherapy with cytokine-gene modified vaccines, and tumor suppressor gene therapy focusing on growth arrest and programm