Tools and Applications in Gene Therapy

Gene therapy is a powerful therapeutic approach involving modifications at the genetic level. Different techniques can be applied for several purposes. For example, a mutated gene causing a disease can be replaced with a healthy copy of the gene, can be inactivated or "knocked out", or a new gene can be introduced to help fight a disease. In this book, recent advances in gene therapy tools and applications are proposed, according to four main aspects: 1. Introduction on the gene therapy, discussing recent progresses and predictions about these approaches of gene delivery and modification. The first clinical gene delivery was published 25 years ago: since then, enormous progresses have been made to improve human life. Moreover, the use of non-viral and viral vectors, including alp ha retroviral vectors, for gene therapy delivery in the clinic are discussed. 2. The role of CRISPR-Cas9 system for gene therapy. CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats (CRISPR) associated nuclease 9) is a recent easy-to-use technique with multiplexing nature for editing the genome. This approach is revolutionary in several medical research areas, with tremendous gene therapy potentials. Many works have demonstrated the therapeutic potentials of CRISPR-Cas9 in vitro or in vivo in animal germlines. Here, several recent reports evaluating the role of CRISPR-Cas9 in gene therapy for cystic fibrosis, hemophilia B, Wilson disease in a rabbit model, among others, are included. 3. Recent applications of gene therapy, with emphasis on physiological processes such as histone deacetylation in melanoma disease investigated by targeted gene delivery by using a bacteriophage viral vector. Moreover, the genetic abrogation of immune checkpoints in antigen-specific cytotoxic T-lymphocyte is evaluated as potential alternative approach. 4. Innovative tools for gene therapy. Several purposes are discussed, including the use of baculovirus Bombyx mori nucleopolyhedrovirus vector for gene delivery and expression in vertebrate. Book jacket.