Name: Advancing Gene-Targeted Therapies for Central Nervous System Disorders. Proceedings of a Workshop
SKU: 64847692
Price: 1126.23 MXN
Availability: InStock
Author: Board on Health Sciences Policy;Lisa Bain;Clare Stroud;Health and Medicine Division;Forum on Neuroscience and Nervous System Disorders;Engineering, and Medicine National Academies of Sciences
ISBN: 9780309495844

portada Advancing Gene-Targeted Therapies for Central Nervous System Disorders. Proceedings of a Workshop

Advancing Gene-Targeted Therapies for Central Nervous System Disorders. Proceedings of a Workshop

Formato

Libro Físico

Editorial

National Academies Press

Autor

Board on Health Sciences Policy;Lisa Bain;Clare Stroud;Health and Medicine Division;Forum on Neuroscience and Nervous System Disorders;Engineering, and Medicine National Academies of Sciences

Año

2019

N° páginas

Encuadernación

Tapa Blanda

Dimensiones

22.9x15.2x2.5 cm

ISBN13

9780309495844

Advancing Gene-Targeted Therapies for Central Nervous System Disorders. Proceedings of a Workshop

Board On Health Sciences Policy;Lisa Bain;Clare Stroud;Health And Medicine Division;Forum On Neuroscience And Nervous System Disorders;Engineering, And Medicine National Academies Of Sciences (Autor) · National Academies Press · Tapa Blanda

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Reseña del libro "Advancing Gene-Targeted Therapies for Central Nervous System Disorders. Proceedings of a Workshop"

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 ContentsFront Matter1 Introduction and Overview2 Exploring the Current Landscape of Central Nervous System Gene-Targeted Therapies3 Gene-Targeted Therapy Approaches for Central Nervous System Disorders: Opportunities and Challenges4 Translating Gene-Targeted Therapies from Bench to Bedside5 Meaningful Engagement of Patients and Families6 Future Directions in the Development of Gene-Targeted TherapiesAppendix A: ReferencesAppendix B: Workshop AgendaAppendix C: Registered Attendees