دانلود کتاب توسعه و برنامه های کاربردی سیستم های تحویل ژن

Human gene therapy was in a conceptual stage in the 20th century, but come the 21st century, it has become a reality. The recombinant adeno-associated virus (rAAV) has become a major gene therapy vector and spawned a multibillion dollar industry. With the advent of the COVID-19 pandemic in the last two years, gene therapy has become a great tool to treat new virus infections. Most of the COVID-19 virus vaccines are using messenger RNA (mRNA) technology as a treatment tool. The possibilities of rAAV gene therapy are continuing to grow. In addition, advances in structural biology provide easier access to the molecular details respon­sible for cell entry and escape from neutralizing antibodies. This is helping investiga­tors design increasingly sophisticated capsid modification schemes to improve rAAV vector properties. Newer challenges are forthcoming in developing drug delivery systems to deliver various gene-based treatment, including mRNA, small interfer­ing RNA (siRNA) and other gene therapies for treatment of various diseases. These treatments are providing incredible solutions, especially for the treatment of chronic diseases, including cancers and infectious diseases. There is a lot to be understood about the key molecular interactions of capsids with host factors, and one would anticipate continuing feedback into vector design over the coming years. There are also areas in which the salient molecular interactions remain largely uncharacterized, such as cellular immune responses, and so prog­ress in vector delivery now involves empirical mitigation strategies. It is important to emphasize that identification of the most critical host factors for entry is recent, their characterization is ongoing and exploitation of the emerging understanding for improved vector delivery is only just starting.