Gene Therapy for Neurological Disorders Methods and Protocols [Hardcover]

This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results. Chapters in this book disseminate bits of unknown information that are important to consider during the course of experimentation and will answer questions such as: What delivery vehicle do you use?; How will you ensure that your vector retains stability?; What expression system best fits your needs?; What route will you choose to deliver your gene therapy agent?; How will you model the neurodegenerative disorder that you aim to investigate and what are the proven methods to treat these disorders in preclinical models? Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols and tips on troubleshooting and avoiding known pitfalls.

Authoritative and thorough, Gene Therapy for Neurological Disorders: Methods and Protocols, is a compilation of protocols and instructive chapters intended to give researchers, clinicians, and students of all levels, a foundation upon which future gene therapy experiments can be designed.

Part I Introduction

1. Introduction to Viral Vectors and Other Delivery Methods for Gene Therapy of the Nervous System
Frederic P. Manfredsson

Part II Expression Cassettes???????

2. Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors
Matthew L. Hirsch, Sonya J. Wolfe, and R. J. Samulski

3. Expression of Multiple Functional RNAs or Proteins from One Viral Vector
Tomas Bj?rklund

4. Regulatl#,