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Advancing Gene-Targeted Therapies for Central Nervous System Disorders Proceedings of a Workshop (2019) / Chapter Skim
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1 Introduction and Overview
Pages 1-4

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From page 1...
... After decades of scientific, clinical, and manufacturing advances, genetargeted therapies have recently been approved for two rare monogenic neurological disorders: an inherited retinal disease caused by biallelic mutations in the RPE65 gene (known clinically as some types of Leber con­ genital amaurosis or retinitis pigmentosa) and the progressive and often fatal neuro­ uscular disorder called spinal muscular atrophy.
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From page 2...
... 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 diseases, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism.
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From page 3...
... Invited presentations and discussions will be designed to: • Provide an overview of the current landscape of gene-targeted therapy approaches for CNS disorders. • Discuss lessons learned from recent advances in gene therapy and antisense oligonucleotide development for retinal dystrophy and spinal muscular atrophy.
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From page 4...
... 4 ADVANCING GENE-TARGETED THERAPIES FOR CNS DISORDERS patients and families, as well as ethical issues related to the development and marketing of gene-targeted therapies, including issues related to data sharing and access. Advancing the development of gene-targeted therapies through further innovation and collaboration is discussed in Chapter 6.
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Key Terms

  • therapy approaches
  • approved therapies
  • targeted therapy
  • clinical development
  • gene therapy

  • rare genetic
  • explore approaches
  • rare monogenic
  • nonprofit organizations
  • viral vectors

  • central nervous
  • genetic disorders
  • auditory disorders
  • disorder called
  • compare features

  • potential collaborative
  • cal findings
  • oligonucleotide development
  • retinal dystrophy
  • disorders convened

  • workshop brought
  • addressing issues
  • clinical trials
  • trial design
  • proceedings chapter

  • generation viral
  • recent advances
  • potential solutions
  • including potential
  • gene products


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