Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease

Research output: Contribution to journal › Journal article › Research › peer-review

Standard

Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease. / Grealish, Shane; Diguet, Elsa; Kirkeby, Agnete; Mattsson, Bengt; Heuer, Andreas; Bramoulle, Yann; Van Camp, Nadja; Perrier, Anselme L.; Hantraye, Philippe; Björklund, Anders; Parmar, Malin.

In: Cell Stem Cell, Vol. 15, No. 5, 01.01.2014, p. 653-665.

Research output: Contribution to journal › Journal article › Research › peer-review

Harvard

Grealish, S, Diguet, E, Kirkeby, A, Mattsson, B, Heuer, A, Bramoulle, Y, Van Camp, N, Perrier, AL, Hantraye, P, Björklund, A & Parmar, M 2014, 'Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease', Cell Stem Cell, vol. 15, no. 5, pp. 653-665. https://doi.org/10.1016/j.stem.2014.09.017

APA

Grealish, S., Diguet, E., Kirkeby, A., Mattsson, B., Heuer, A., Bramoulle, Y., Van Camp, N., Perrier, A. L., Hantraye, P., Björklund, A., & Parmar, M. (2014). Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease. Cell Stem Cell, 15(5), 653-665. https://doi.org/10.1016/j.stem.2014.09.017

Vancouver

Grealish S, Diguet E, Kirkeby A, Mattsson B, Heuer A, Bramoulle Y et al. Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease. Cell Stem Cell. 2014 Jan 1;15(5):653-665. https://doi.org/10.1016/j.stem.2014.09.017

Author

Grealish, Shane ; Diguet, Elsa ; Kirkeby, Agnete ; Mattsson, Bengt ; Heuer, Andreas ; Bramoulle, Yann ; Van Camp, Nadja ; Perrier, Anselme L. ; Hantraye, Philippe ; Björklund, Anders ; Parmar, Malin. / Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease. In: Cell Stem Cell. 2014 ; Vol. 15, No. 5. pp. 653-665.

Bibtex

@article{3303e46b2ea245ce8797bdaf16e1b64a,
title = "Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease",
abstract = "Considerable progress has been made in generating fully functional and transplantable dopamine neurons from human embryonic stem cells (hESCs). Before these cells can be used for cell replacement therapy in Parkinson's disease (PD), it is important to verify their functional properties and efficacy in animal models. Here we provide a comprehensive preclinical assessment of hESC-derived midbrain dopamine neurons in a rat model of PD. We show long-term survival and functionality using clinically relevant MRI and PET imaging techniques and demonstrate efficacy in restoration of motor function with a potency comparable to that seen with human fetal dopamine neurons. Furthermore, we show that hESC-derived dopamine neurons can project sufficiently long distances for use in humans, fully regenerate midbrain-to-forebrain projections, and innervate correct target structures. This provides strong preclinical support for clinical translation of hESC-derived dopamine neurons using approaches similar to those established with fetal cells for the treatment of Parkinson's disease.",
author = "Shane Grealish and Elsa Diguet and Agnete Kirkeby and Bengt Mattsson and Andreas Heuer and Yann Bramoulle and {Van Camp}, Nadja and Perrier, {Anselme L.} and Philippe Hantraye and Anders Bj{\"o}rklund and Malin Parmar",
year = "2014",
month = jan,
day = "1",
doi = "10.1016/j.stem.2014.09.017",
language = "English",
volume = "15",
pages = "653--665",
journal = "Cell Stem Cell",
issn = "1934-5909",
publisher = "Cell Press",
number = "5",

}

RIS

TY - JOUR

T1 - Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease

AU - Grealish, Shane

AU - Diguet, Elsa

AU - Kirkeby, Agnete

AU - Mattsson, Bengt

AU - Heuer, Andreas

AU - Bramoulle, Yann

AU - Van Camp, Nadja

AU - Perrier, Anselme L.

AU - Hantraye, Philippe

AU - Björklund, Anders

AU - Parmar, Malin

PY - 2014/1/1

Y1 - 2014/1/1

N2 - Considerable progress has been made in generating fully functional and transplantable dopamine neurons from human embryonic stem cells (hESCs). Before these cells can be used for cell replacement therapy in Parkinson's disease (PD), it is important to verify their functional properties and efficacy in animal models. Here we provide a comprehensive preclinical assessment of hESC-derived midbrain dopamine neurons in a rat model of PD. We show long-term survival and functionality using clinically relevant MRI and PET imaging techniques and demonstrate efficacy in restoration of motor function with a potency comparable to that seen with human fetal dopamine neurons. Furthermore, we show that hESC-derived dopamine neurons can project sufficiently long distances for use in humans, fully regenerate midbrain-to-forebrain projections, and innervate correct target structures. This provides strong preclinical support for clinical translation of hESC-derived dopamine neurons using approaches similar to those established with fetal cells for the treatment of Parkinson's disease.

AB - Considerable progress has been made in generating fully functional and transplantable dopamine neurons from human embryonic stem cells (hESCs). Before these cells can be used for cell replacement therapy in Parkinson's disease (PD), it is important to verify their functional properties and efficacy in animal models. Here we provide a comprehensive preclinical assessment of hESC-derived midbrain dopamine neurons in a rat model of PD. We show long-term survival and functionality using clinically relevant MRI and PET imaging techniques and demonstrate efficacy in restoration of motor function with a potency comparable to that seen with human fetal dopamine neurons. Furthermore, we show that hESC-derived dopamine neurons can project sufficiently long distances for use in humans, fully regenerate midbrain-to-forebrain projections, and innervate correct target structures. This provides strong preclinical support for clinical translation of hESC-derived dopamine neurons using approaches similar to those established with fetal cells for the treatment of Parkinson's disease.

UR - http://www.scopus.com/inward/record.url?scp=84922662454&partnerID=8YFLogxK

U2 - 10.1016/j.stem.2014.09.017

DO - 10.1016/j.stem.2014.09.017

M3 - Journal article

C2 - 25517469

AN - SCOPUS:84922662454

VL - 15

SP - 653

EP - 665

JO - Cell Stem Cell

JF - Cell Stem Cell

SN - 1934-5909

IS - 5

ER -

ID: 228505568