BookClévio Nóbrega, Liliana Mendonça, Carlos A. Matos.
Summary: This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field. -- Provided by publisher.
Contents:
Intro
Acknowledgments
Contents
1: Gene and Cell Therapy
1.1 The Concepts of Gene and Cell Therapy
1.2 Types of Gene Therapy
1.3 Gene Therapy Strategies
1.4 Choice of the Therapeutic Target
1.5 Administration Routes
1.6 Delivery Systems
1.7 Expression and Persistence of the Therapy
1.8 Cell Targeting
1.9 Immune Response to the Therapy
1.10 Highlights in the History of Gene and Cell Therapy
1.11 Current Status of Gene Therapy
1.12 Ethical Questions and Concerns About Gene and Cell Therapy
This Chapter in a Nutshell
Review Questions 3.1 Lentiviral Vectors
3.1.1 Replicative Cycle
3.1.2 From Lentivirus to Lentiviral Vectors
3.1.3 Additional Improvements to Lentiviral Vectors
3.1.4 Lentiviral Vector Production
3.1.5 Lentiviral Vectors in Clinical Trials
3.2 Gamma Retrovirus
3.3 Adenoviral Vectors
3.3.1 Replicative Cycle
3.3.2 From Adenovirus to Adenoviral Vectors
3.3.3 Adenoviral Vector Modifications
3.3.4 Adenoviral Vector Production
3.3.5 Adenoviral Vectors in Clinical Trials
3.4 Adeno-associated Virus (AAV)
3.4.1 Replicative Cycle
3.4.2 From AAV to AAV Vectors 3.4.3 AAV Modifications
3.4.4 AAV Production
3.4.5 AAV in Clinical Trials
3.5 Herpes Simplex Virus
3.5.1 Replicative Cycle
3.5.2 From HSV to HSV Vectors
3.5.3 HSV Modifications
3.5.4 HSV Production
3.5.5 HSV in Clinical Trials
3.6 Vaccinia
3.6.1 Replicative Cycle
3.6.2 From Vaccinia Virus to Vaccinia Vectors
3.6.3 Vaccinia Modifications
3.6.4 Vaccinia Vector Production
3.6.5 Vaccinia in Clinical Trials
3.7 Baculovirus
3.7.1 Replicative Cycle
3.7.2 From Baculovirus to Baculovirus Vectors
3.7.3 Baculovirus Modifications 3.7.4 Baculovirus Production
3.7.5 Baculovirus in Clinical Trials
3.8 Choice of the Viral Vector
3.9 Oncolytic Virus Applications
This Chapter in a Nutshell
Review Questions
References and Further Reading
4: Barriers to Gene Delivery
4.1 Extracellular Barriers
4.1.1 Unspecific Interactions
4.1.2 Endothelial Barriers
4.1.3 Inflammatory and Immune Response
4.2 Intracellular Barriers
4.2.1 Cellular Uptake
4.2.2 Endosomal Escape
4.2.3 Intracellular Trafficking
4.2.4 Nuclear Delivery
4.3 Technical Barriers
4.3.1 Physical Restrictions
4.3.2 Cellular Targeting.