Viral Vectors for Gene Therapy

This book offers methodologies for virus production, transferring protocols, and evaluating the efficacy of gene products. Covers the basic principles of popular and less common viral vector systems, using the successful Methods in Molecular Biology(TM) format.

The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular Biology™ series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls.

Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective.

Focuses on the many applications of viral vectors, widely considered more efficient than DNA transfection Contains practical laboratory protocols using a wide array of common and uncommon viruses Features expert tips and key implementation advice Includes supplementary material: sn.pub/extras

Inhalt

Introduction to Viral Vectors.- Introduction to Gene Therapy: A Clinical Aftermath.- Host Cells and Cell Banking.- Overview of Current Scalable Methods for Purification of Viral Vectors.- Methods to Construct Recombinant Adenovirus Vectors.- Manufacturing of Adenoviruses Vectors: Production and Purification of Helper Dependent Adenovirus.- Manufacturing of Retroviruses.- Lentiviral Vectors.- Adeno-Associated Viruses.- Manufacturing of Adeno-Associated Viruses, for Example: AAV2.- Vector Characterization Methods for Quality Control Testing of Recombinant Adeno-Associated Viruses.- Baculoviruses Mediate Efficient Gene Expression in a Wide Range of Vertebrate Cells.- Herpes Simplex Virus Type 1 (HSV-1)-Derived Recombinant and Amplicon Vectors.- Manufacture of Measles Viruses.- In Vivo Gene Delivery into hCD34+ Cells in a Humanized Mouse Model.- In Vivo Evaluation of Gene Transfer into Mesenchymal Cells (in View of Cartilage Repair).- Ethical Consideration.- Clinical Trials of GMP Products in the Gene Therapy Field.