Delivery Methods for Treatment of Genetic Disorders

Kunal Gupta; Nathan R. Selden

doi:10.1016/B978-0-12-813997-4.00022-0

Delivery Methods for Treatment of Genetic Disorders

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

Abstract

The effective delivery of gene therapy can be reduced to three essential components: (1) specific targeting of an anatomical region or cell population, (2) transfer of genetic material to the host, and (3) efficacious expression that alters the disease phenotype. The predominant delivery methods are viral-mediated and nonviral-mediated therapies. Each method has benefits and caveats that affect the targeting of specific regions, stable or unstable transfer of genetic material, and effect on the targeted disorder. In this chapter we will discuss viral- and nonviral-mediated therapies, challenges in applying these therapies in animal models and human patients, and ways to circumvent these delivery roadblocks. We will also discuss specific adaptations of these methods in attempting delivery to the central nervous system.

Original language	English (US)
Title of host publication	Nervous System Drug Delivery
Subtitle of host publication	Principles and Practice
Publisher	Elsevier
Pages	447-461
Number of pages	15
ISBN (Electronic)	9780128139981
ISBN (Print)	9780128139974
DOIs	https://doi.org/10.1016/B978-0-12-813997-4.00022-0
State	Published - Jun 25 2019

Keywords

CRISPR/Cas9
Central nervous system
Gene therapy
Stem cells
Stereotactic surgery
Virus

ASJC Scopus subject areas

General Medicine

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10.1016/B978-0-12-813997-4.00022-0

Cite this

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AB - The effective delivery of gene therapy can be reduced to three essential components: (1) specific targeting of an anatomical region or cell population, (2) transfer of genetic material to the host, and (3) efficacious expression that alters the disease phenotype. The predominant delivery methods are viral-mediated and nonviral-mediated therapies. Each method has benefits and caveats that affect the targeting of specific regions, stable or unstable transfer of genetic material, and effect on the targeted disorder. In this chapter we will discuss viral- and nonviral-mediated therapies, challenges in applying these therapies in animal models and human patients, and ways to circumvent these delivery roadblocks. We will also discuss specific adaptations of these methods in attempting delivery to the central nervous system.

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KW - Central nervous system

KW - Gene therapy

KW - Stem cells

KW - Stereotactic surgery

KW - Virus

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Delivery Methods for Treatment of Genetic Disorders

Abstract

Keywords

ASJC Scopus subject areas

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