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The Resource Advancing gene-targeted therapies for central nervous system disorders : proceedings of a workshop, Lisa Bain and Clare Stroud, rapporteurs ; Forum on Neuroscience and Nervous System Disorders, Board on Health Sciences Policy, Health and Medicine Division, the National Academies of Sciences, Engineering, Medicine

Advancing gene-targeted therapies for central nervous system disorders : proceedings of a workshop, Lisa Bain and Clare Stroud, rapporteurs ; Forum on Neuroscience and Nervous System Disorders, Board on Health Sciences Policy, Health and Medicine Division, the National Academies of Sciences, Engineering, Medicine

Creator
Contributor
Summary
"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"--Publisher's description
Language
eng
Work
Publication
Extent
1 online resource (1 PDF file (xii, 80 pages))
Contents
  • Introduction and overview
  • Exploring the current landscape of central nervous system gene-targeted therapies
  • Gene-targeted therapy approaches for central nervous system disorders : opportunities and challenges
  • Translating gene-targeted therapies from bench to bedside
  • Meaningful engagement of patients and families
  • Future directions in the development of gene-targeted therapies
  • Appendixes
Isbn
9780309495875
Instance
Label
Advancing gene-targeted therapies for central nervous system disorders : proceedings of a workshop
Title
Advancing gene-targeted therapies for central nervous system disorders
Title remainder
proceedings of a workshop
Statement of responsibility
Lisa Bain and Clare Stroud, rapporteurs ; Forum on Neuroscience and Nervous System Disorders, Board on Health Sciences Policy, Health and Medicine Division, the National Academies of Sciences, Engineering, Medicine
Creator
Contributor
Issuing body
Subject
Genre
Language
eng
Summary
"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"--Publisher's description
Cataloging source
NLM
http://library.link/vocab/creatorName
Bain, Lisa J
Dewey number
616.80442
Illustrations
illustrations
Index
no index present
LC call number
RC350.G45
Literary form
non fiction
Nature of contents
  • dictionaries
  • bibliography
NLM call number
WL 301
http://bibfra.me/vocab/relation/rapporteur
  • ONV1dHp5J00
  • j6A4gshEj3Y
http://library.link/vocab/relatedWorkOrContributorDate
2019
http://library.link/vocab/relatedWorkOrContributorName
  • Stroud, Clare
  • National Academies of Sciences, Engineering, and Medicine (U.S.)
  • Advancing Gene-Targeted Therapies for Central Nervous System Disorders (Workshop)
http://library.link/vocab/subjectName
  • Nervous system
  • Gene therapy
  • Central Nervous System Diseases
  • Genetic Therapy
  • Central Nervous System Diseases
  • Gene therapy
Label
Advancing gene-targeted therapies for central nervous system disorders : proceedings of a workshop, Lisa Bain and Clare Stroud, rapporteurs ; Forum on Neuroscience and Nervous System Disorders, Board on Health Sciences Policy, Health and Medicine Division, the National Academies of Sciences, Engineering, Medicine
Instantiates
Publication
Copyright
Bibliography note
Includes bibliographical references (pages 59-64)
Carrier category
online resource
Carrier category code
  • cr
Carrier MARC source
rdacarrier
Color
multicolored
Content category
text
Content type code
  • txt
Content type MARC source
rdacontent
Contents
Introduction and overview -- Exploring the current landscape of central nervous system gene-targeted therapies -- Gene-targeted therapy approaches for central nervous system disorders : opportunities and challenges -- Translating gene-targeted therapies from bench to bedside -- Meaningful engagement of patients and families -- Future directions in the development of gene-targeted therapies -- Appendixes
Control code
on1127178869
Dimensions
unknown
Extent
1 online resource (1 PDF file (xii, 80 pages))
Form of item
online
Isbn
9780309495875
Media category
computer
Media MARC source
rdamedia
Media type code
  • c
Other physical details
color illustrations
Specific material designation
remote
System control number
(OCoLC)1127178869
Label
Advancing gene-targeted therapies for central nervous system disorders : proceedings of a workshop, Lisa Bain and Clare Stroud, rapporteurs ; Forum on Neuroscience and Nervous System Disorders, Board on Health Sciences Policy, Health and Medicine Division, the National Academies of Sciences, Engineering, Medicine
Publication
Copyright
Bibliography note
Includes bibliographical references (pages 59-64)
Carrier category
online resource
Carrier category code
  • cr
Carrier MARC source
rdacarrier
Color
multicolored
Content category
text
Content type code
  • txt
Content type MARC source
rdacontent
Contents
Introduction and overview -- Exploring the current landscape of central nervous system gene-targeted therapies -- Gene-targeted therapy approaches for central nervous system disorders : opportunities and challenges -- Translating gene-targeted therapies from bench to bedside -- Meaningful engagement of patients and families -- Future directions in the development of gene-targeted therapies -- Appendixes
Control code
on1127178869
Dimensions
unknown
Extent
1 online resource (1 PDF file (xii, 80 pages))
Form of item
online
Isbn
9780309495875
Media category
computer
Media MARC source
rdamedia
Media type code
  • c
Other physical details
color illustrations
Specific material designation
remote
System control number
(OCoLC)1127178869

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