Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

Josse A. Depla; Marina Sogorb-Gonzalez; Lance A. Mulder; Vivi M. Heine; Pavlina Konstantinova; Sander J. van Deventer; Katja C. Wolthers; Dasja Pajkrt; Adithya Sridhar; Melvin M. Evers

doi:https://doi.org/10.1016/j.omtm.2020.05.028

Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

Josse A. Depla, Marina Sogorb-Gonzalez, Lance A. Mulder, Vivi M. Heine, Pavlina Konstantinova, Sander J. van Deventer, Katja C. Wolthers, Dasja Pajkrt, Adithya Sridhar, Melvin M. Evers

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

19 Citations (Scopus)

Abstract

Induced pluripotent stem cell-derived cerebral organoids are an innovative human model of the brain that could improve translation of gene therapy to the clinic. Depla et al. demonstrate the application of cerebral organoids as a preclinical model for AAV capsid selection and testing efficacy of therapeutic transgenes.

Original language	English
Pages (from-to)	167-175
Number of pages	9
Journal	Molecular Therapy - Methods and Clinical Development
Volume	18
Early online date	1 Jun 2020
DOIs	https://doi.org/10.1016/j.omtm.2020.05.028
Publication status	Published - 11 Sept 2020

Keywords

AAV
CNS
Cerebral organoids
SCA3
Transduction
adeno-associated virus
central nervous system
gene therapy
iPSC
induced pluripotent stem cells
miRNA
micro RNA
organoids
spinocerebellar ataxia 3

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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https://doi.org/10.1016/j.omtm.2020.05.028

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Depla, J. A., Sogorb-Gonzalez, M., Mulder, L. A., Heine, V. M., Konstantinova, P., van Deventer, S. J., Wolthers, K. C., Pajkrt, D., Sridhar, A., & Evers, M. M. (2020). Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain. Molecular Therapy - Methods and Clinical Development, 18, 167-175. https://doi.org/10.1016/j.omtm.2020.05.028

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abstract = "Induced pluripotent stem cell-derived cerebral organoids are an innovative human model of the brain that could improve translation of gene therapy to the clinic. Depla et al. demonstrate the application of cerebral organoids as a preclinical model for AAV capsid selection and testing efficacy of therapeutic transgenes.",

keywords = "AAV, CNS, Cerebral organoids, SCA3, Transduction, adeno-associated virus, central nervous system, gene therapy, iPSC, induced pluripotent stem cells, miRNA, micro RNA, organoids, spinocerebellar ataxia 3",

author = "Depla, {Josse A.} and Marina Sogorb-Gonzalez and Mulder, {Lance A.} and Heine, {Vivi M.} and Pavlina Konstantinova and {van Deventer}, {Sander J.} and Wolthers, {Katja C.} and Dasja Pajkrt and Adithya Sridhar and Evers, {Melvin M.}",

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doi = "https://doi.org/10.1016/j.omtm.2020.05.028",

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Depla, JA, Sogorb-Gonzalez, M, Mulder, LA , Heine, VM, Konstantinova, P, van Deventer, SJ, Wolthers, KC , Pajkrt, D , Sridhar, A & Evers, MM 2020, 'Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain', Molecular Therapy - Methods and Clinical Development, vol. 18, pp. 167-175. https://doi.org/10.1016/j.omtm.2020.05.028

TY - JOUR

T1 - Cerebral Organoids

T2 - A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

AU - Depla, Josse A.

AU - Sogorb-Gonzalez, Marina

AU - Mulder, Lance A.

AU - Heine, Vivi M.

AU - Konstantinova, Pavlina

AU - van Deventer, Sander J.

AU - Wolthers, Katja C.

AU - Pajkrt, Dasja

AU - Sridhar, Adithya

AU - Evers, Melvin M.

PY - 2020/9/11

Y1 - 2020/9/11

N2 - Induced pluripotent stem cell-derived cerebral organoids are an innovative human model of the brain that could improve translation of gene therapy to the clinic. Depla et al. demonstrate the application of cerebral organoids as a preclinical model for AAV capsid selection and testing efficacy of therapeutic transgenes.

AB - Induced pluripotent stem cell-derived cerebral organoids are an innovative human model of the brain that could improve translation of gene therapy to the clinic. Depla et al. demonstrate the application of cerebral organoids as a preclinical model for AAV capsid selection and testing efficacy of therapeutic transgenes.

KW - AAV

KW - CNS

KW - Cerebral organoids

KW - SCA3

KW - Transduction

KW - adeno-associated virus

KW - central nervous system

KW - gene therapy

KW - iPSC

KW - induced pluripotent stem cells

KW - miRNA

KW - micro RNA

KW - organoids

KW - spinocerebellar ataxia 3

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U2 - https://doi.org/10.1016/j.omtm.2020.05.028

DO - https://doi.org/10.1016/j.omtm.2020.05.028

M3 - Article

C2 - 32637448

SN - 2329-0501

VL - 18

SP - 167

EP - 175

JO - Molecular Therapy - Methods and Clinical Development

JF - Molecular Therapy - Methods and Clinical Development

ER -

Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

Abstract

Keywords

UN SDGs

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