1.2 Abbreviations. Expert opinion: Gene therapy may revolutionize the treatment of neurological disorders in the coming decades but there are still great challenges ahead. 2 Executive Summary. The book begins with general reviews of gene therapy strategies with a focus on neurological disorders. WHAT IS GENE THERAPY ?• Definiton: an experimental technique for correcting defective genes that are responsible for disease development• The most … In preclinical studies, transgenes encoding therapeutic proteins, microRNAs, antibodies or gene-editing machinery have been successfully delivered to the central nervous system with natural or engineered viral capsids via various routes of administration. EXPERT OPINION: Gene therapy may revolutionize the treatment of neurological disorders in the coming decades but there are still great challenges ahead. The strength of gene therapy has been emphasized in the overexpression of therapeutic genes. 1.3 Related Reports. 28, No. Roundtable: Gene Therapy in Neurological Diseases Wednesday, Oct. 23, 8:30 - 11:00 a.m., Room N230B Gene Therapy Press Conference Summary. 2, pp. Journal of Drug Targeting: Vol. 1.1 Table of Contents. Gene therapy ppt 1. The remainder of the chapters present approaches to specific neurological disorders. (2020). Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the treatment of neurological diseases. Gene Therapy in Neurological Disorders thoroughly reviews currently available gene therapy tools and presents examples of their application in a variety of neurological diseases. Course DescriptionParticipants will gain an understanding of the rapidly evolving landscape of gene therapy for neurological disorders of childhood. 1 Table of Contents (PowerPoint Deck) 1 Preface. 2.1 Key Findings. Gene therapy for neurological disorders: challenges and recent advancements. 2.2 KOL Insights on Competitive Landscape for Gene Therapy for Neurology Indications. 3 Overview – Gene Therapy in Neurology. GENE THERAPY Presented by:Nurul Miza Shasheiha binti Abdul Mutalib -5089- Wan Fathiah Nasuha binti Wan Nudri -5117- Nurul Husna binti Muryadi -5088- Nur Amalin Shafirah binti Nur Farzana Sahirah binti Ariffuddin 2. 3.2 Gene Therapy Versus Conventional Therapies. 3.1 Objectives of Gene Therapy. We will discuss the basic principles of gene vector development and delivery, and describe specific diseases for which gene therapy is currently approved for clinical use or available as an experimental treatment in clinical trials. 111-128.