Adeno-associated virus-mediated gene therapy in central nervous system genetic disorders

Widler Casy; Qinglan Ling; Frances C. Shaffo; Sarah E. Sinnett; Steven J. Gray

doi:10.1016/B978-0-12-813955-4.00010-6

Adeno-associated virus-mediated gene therapy in central nervous system genetic disorders

Widler Casy, Qinglan Ling, Frances C. Shaffo, Sarah E. Sinnett, Steven J. Gray

Research output: Chapter in Book/Report/Conference proceeding › Chapter

1 Scopus citations

Abstract

Gene therapy has become a promising strategy to treat the genetic disorders of central nervous system (CNS). In the last five decades the development and characterization of recombinant adeno-associated virus (rAAV) as a safe and effective vector for gene delivery has revolutionized this field. Development of rAAV with better efficacy and selectivity for the CNS has resulted in increasingly successful preclinical and clinical applications. This chapter provides an overview of AAV-mediated CNS gene therapy, including discovery and engineering of novel AAV capsids, advances in preclinical models of genetic disorders, and clinical translation of AAV technology.

Original language	English (US)
Title of host publication	Rosenberg’s Molecular and Genetic Basis of Neurological and Psychiatric Disease
Subtitle of host publication	Volume 1
Publisher	Elsevier
Pages	129-144
Number of pages	16
ISBN (Electronic)	9780128139554
DOIs	https://doi.org/10.1016/B978-0-12-813955-4.00010-6
State	Published - Jan 1 2020

Keywords

AAV
Capsid engineering
Central nervous system
Gene therapy
Rare genetic disorders

ASJC Scopus subject areas

General Medicine

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10.1016/B978-0-12-813955-4.00010-6

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abstract = "Gene therapy has become a promising strategy to treat the genetic disorders of central nervous system (CNS). In the last five decades the development and characterization of recombinant adeno-associated virus (rAAV) as a safe and effective vector for gene delivery has revolutionized this field. Development of rAAV with better efficacy and selectivity for the CNS has resulted in increasingly successful preclinical and clinical applications. This chapter provides an overview of AAV-mediated CNS gene therapy, including discovery and engineering of novel AAV capsids, advances in preclinical models of genetic disorders, and clinical translation of AAV technology.",

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AU - Casy, Widler

AU - Ling, Qinglan

AU - Shaffo, Frances C.

AU - Sinnett, Sarah E.

AU - Gray, Steven J.

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AB - Gene therapy has become a promising strategy to treat the genetic disorders of central nervous system (CNS). In the last five decades the development and characterization of recombinant adeno-associated virus (rAAV) as a safe and effective vector for gene delivery has revolutionized this field. Development of rAAV with better efficacy and selectivity for the CNS has resulted in increasingly successful preclinical and clinical applications. This chapter provides an overview of AAV-mediated CNS gene therapy, including discovery and engineering of novel AAV capsids, advances in preclinical models of genetic disorders, and clinical translation of AAV technology.

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KW - Rare genetic disorders

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BT - Rosenberg’s Molecular and Genetic Basis of Neurological and Psychiatric Disease

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ER -

Adeno-associated virus-mediated gene therapy in central nervous system genetic disorders

Abstract

Keywords

ASJC Scopus subject areas

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