Board on Health Sciences Policy & Forum on Neuroscience and Nervous System Disorders 
Advancing Gene-Targeted Therapies for Central Nervous System Disorders [EPUB ebook] 
Proceedings of a Workshop

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غلاف Board on Health Sciences Policy & Forum on Neuroscience and Nervous System Disorders: Advancing Gene-Targeted Therapies for Central Nervous System Disorders (ePUB)

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.

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لغة الإنجليزية ● شكل EPUB ● صفحات 92 ● ISBN 9780309495875 ● محرر Clare Stroud & Lisa Bain ● الناشر National Academies Press ● نشرت 2019 ● للتحميل 3 مرات ● دقة EUR ● هوية شخصية 7282839 ● حماية النسخ Adobe DRM
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