Advancing Gene-Targeted Therapies for Central Nervous System Disorders - Proceedings of a Workshop (Paperback)

On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop. Table of Contents Front Matter 1 Introduction and Overview 2 Exploring the Current Landscape of Central Nervous System Gene-Targeted Therapies 3 Gene-Targeted Therapy Approaches for Central Nervous System Disorders: Opportunities and Challenges 4 Translating Gene-Targeted Therapies from Bench to Bedside 5 Meaningful Engagement of Patients and Families 6 Future Directions in the Development of Gene-Targeted Therapies Appendix A: References Appendix B: Workshop Agenda Appendix C: Registered Attendees

General

Imprint:	National Academies Press
Country of origin:	United States
Release date:	December 2019
First published:	2019
Authors:	National Academies of Sciences, Engineering, and Medicine • Health and Medicine Division • Board on Health Sciences Policy • Forum on Neuroscience and Nervous System Disorders
Editors:	Clare Stroud • Lisa Bain
Dimensions:	229 x 152mm (L x W)
Format:	Paperback
Pages:	92
ISBN-13:	978-0-309-49584-4
Categories:	Books > Medicine > General
LSN:	0-309-49584-9
Barcode:	9780309495844

Is the information for this product incomplete, wrong or inappropriate? Let us know about it.

Does this product have an incorrect or missing image? Send us a new image.

Is this product missing categories? Add more categories.

Review This Product

No reviews yet - be the first to create one!