Efficient gene expression in human stem cell derived-cortical organoids using adeno associated virus

Ann-Na Cho; Fiona Bright; Nicolle Morey; Carol Au; Lars M. Ittner; Yazi D. Ke

doi:10.3390/cells11203194

Efficient gene expression in human stem cell derived-cortical organoids using adeno associated virus

Ann-Na Cho, Fiona Bright, Nicolle Morey, Carol Au, Lars M. Ittner, Yazi D. Ke

Research output: Contribution to journal › Article › peer-review

7 Citations (Scopus)

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Abstract

Cortical organoids are 3D structures derived either from human embryonic stem cells or human induced pluripotent stem cells with their use exploding in recent years due to their ability to better recapitulate the human brain in vivo in respect to organization; differentiation; and polarity. Adeno-associated viruses (AAVs) have emerged in recent years as the vectors of choice for CNS-targeted gene therapy. Here; we compare the use of AAVs as a mode of gene expression in cortical organoids; over traditional methods such as lipofectamine and electroporation and demonstrate its ease-of-use in generating quick disease models through expression of different variants of the central gene—TDP-43—implicated in amyotrophic lateral sclerosis and frontotemporal dementia.

Original language	English
Article number	3194
Pages (from-to)	1-13
Number of pages	13
Journal	Cells
Volume	11
Issue number	20
DOIs	https://doi.org/10.3390/cells11203194
Publication status	Published - 11 Oct 2022

Bibliographical note

Copyright the Author(s) 2022. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.

Keywords

cortical organoids
Adeno-associated viruses (AAVs)
TDP-43
gene delivery
neurodegenerative disease

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10.3390/cells11203194Licence: CC BY

Publisher version (open access)Final published version, 4.05 MBLicence: CC BY

Cite this

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title = "Efficient gene expression in human stem cell derived-cortical organoids using adeno associated virus",

abstract = "Cortical organoids are 3D structures derived either from human embryonic stem cells or human induced pluripotent stem cells with their use exploding in recent years due to their ability to better recapitulate the human brain in vivo in respect to organization; differentiation; and polarity. Adeno-associated viruses (AAVs) have emerged in recent years as the vectors of choice for CNS-targeted gene therapy. Here; we compare the use of AAVs as a mode of gene expression in cortical organoids; over traditional methods such as lipofectamine and electroporation and demonstrate its ease-of-use in generating quick disease models through expression of different variants of the central gene—TDP-43—implicated in amyotrophic lateral sclerosis and frontotemporal dementia.",

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doi = "10.3390/cells11203194",

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AU - Cho, Ann-Na

AU - Bright, Fiona

AU - Morey, Nicolle

AU - Au, Carol

AU - Ittner, Lars M.

AU - Ke, Yazi D.

N1 - Copyright the Author(s) 2022. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.

PY - 2022/10/11

Y1 - 2022/10/11

N2 - Cortical organoids are 3D structures derived either from human embryonic stem cells or human induced pluripotent stem cells with their use exploding in recent years due to their ability to better recapitulate the human brain in vivo in respect to organization; differentiation; and polarity. Adeno-associated viruses (AAVs) have emerged in recent years as the vectors of choice for CNS-targeted gene therapy. Here; we compare the use of AAVs as a mode of gene expression in cortical organoids; over traditional methods such as lipofectamine and electroporation and demonstrate its ease-of-use in generating quick disease models through expression of different variants of the central gene—TDP-43—implicated in amyotrophic lateral sclerosis and frontotemporal dementia.

AB - Cortical organoids are 3D structures derived either from human embryonic stem cells or human induced pluripotent stem cells with their use exploding in recent years due to their ability to better recapitulate the human brain in vivo in respect to organization; differentiation; and polarity. Adeno-associated viruses (AAVs) have emerged in recent years as the vectors of choice for CNS-targeted gene therapy. Here; we compare the use of AAVs as a mode of gene expression in cortical organoids; over traditional methods such as lipofectamine and electroporation and demonstrate its ease-of-use in generating quick disease models through expression of different variants of the central gene—TDP-43—implicated in amyotrophic lateral sclerosis and frontotemporal dementia.

KW - cortical organoids

KW - Adeno-associated viruses (AAVs)

KW - TDP-43

KW - gene delivery

KW - neurodegenerative disease

UR - https://purl.org/au-research/grants/arc/DP170100781

UR - https://purl.org/au-research/grants/arc/DP210101957

UR - https://purl.org/au-research/grants/nhmrc/1123564

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UR - https://purl.org/au-research/grants/nhmrc/1143848

UR - https://purl.org/au-research/grants/nhmrc/2001572

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U2 - 10.3390/cells11203194

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JO - Cells

JF - Cells

IS - 20

M1 - 3194

ER -

Efficient gene expression in human stem cell derived-cortical organoids using adeno associated virus

Abstract

Bibliographical note

Keywords

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Single-session Introduction of Mutations in Parallel Lines (SIMPL)

The tau interactome – novel functions in neuronal physiology

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Efficient gene expression in human stem cell derived-cortical organoids using adeno associated virus

Abstract

Bibliographical note

Keywords

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Projects

Single-session Introduction of Mutations in Parallel Lines (SIMPL)

The tau interactome – novel functions in neuronal physiology

Cite this