Disease Modeling and Gene Therapy of Copper Storage Disease in Canine Hepatic Organoids

Sathidpak Nantasanti; Bart Spee; Hedwig S Kruitwagen; Chen Chen; Niels Geijsen; Loes A Oosterhoff; Monique E van Wolferen; Nicolas Pelaez; Hille Fieten; Richard W Wubbolts; Guy C Grinwis; Jefferson Chan; Meritxell Huch; Robert R G Vries; Hans Clevers; Alain de Bruin; Jan Rothuizen; Louis C Penning; Baukje A Schotanus

doi:10.1016/j.stemcr.2015.09.002

Disease Modeling and Gene Therapy of Copper Storage Disease in Canine Hepatic Organoids

Sathidpak Nantasanti, Bart Spee, Hedwig S Kruitwagen, Chen Chen, Niels Geijsen, Loes A Oosterhoff, Monique E van Wolferen, Nicolas Pelaez, Hille Fieten, Richard W Wubbolts, Guy C Grinwis, Jefferson Chan, Meritxell Huch, Robert R G Vries, Hans Clevers, Alain de Bruin, Jan Rothuizen, Louis C Penning, Baukje A Schotanus

Hubrecht Institute

Onderzoeksoutput: Bijdrage aan wetenschappelijk tijdschrift/periodieke uitgave › Artikel › Wetenschappelijk › peer review

82 Citaten (Scopus)

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The recent development of 3D-liver stem cell cultures (hepatic organoids) opens up new avenues for gene and/or stem cell therapy to treat liver disease. To test safety and efficacy, a relevant large animal model is essential but not yet established. Because of its shared pathologies and disease pathways, the dog is considered the best model for human liver disease. Here we report the establishment of a long-term canine hepatic organoid culture allowing undifferentiated expansion of progenitor cells that can be differentiated toward functional hepatocytes. We show that cultures can be initiated from fresh and frozen liver tissues using Tru-Cut or fine-needle biopsies. The use of Wnt agonists proved important for canine organoid proliferation and inhibition of differentiation. Finally, we demonstrate that successful gene supplementation in hepatic organoids of COMMD1-deficient dogs restores function and can be an effective means to cure copper storage disease.

Originele taal-2	Engels
Tijdschrift	Stem Cell Reports
DOI's	https://doi.org/10.1016/j.stemcr.2015.09.002
Status	Gepubliceerd - 06 okt. 2015

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10.1016/j.stemcr.2015.09.002

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Nantasanti, S., Spee, B., Kruitwagen, H. S., Chen, C., Geijsen, N., Oosterhoff, L. A., van Wolferen, M. E., Pelaez, N., Fieten, H., Wubbolts, R. W., Grinwis, G. C., Chan, J., Huch, M., Vries, R. R. G., Clevers, H., de Bruin, A., Rothuizen, J., Penning, L. C., & Schotanus, B. A. (2015). Disease Modeling and Gene Therapy of Copper Storage Disease in Canine Hepatic Organoids. Stem Cell Reports. https://doi.org/10.1016/j.stemcr.2015.09.002

@article{76087ead4e494e33b51c4c733e9827c2,

title = "Disease Modeling and Gene Therapy of Copper Storage Disease in Canine Hepatic Organoids",

abstract = "The recent development of 3D-liver stem cell cultures (hepatic organoids) opens up new avenues for gene and/or stem cell therapy to treat liver disease. To test safety and efficacy, a relevant large animal model is essential but not yet established. Because of its shared pathologies and disease pathways, the dog is considered the best model for human liver disease. Here we report the establishment of a long-term canine hepatic organoid culture allowing undifferentiated expansion of progenitor cells that can be differentiated toward functional hepatocytes. We show that cultures can be initiated from fresh and frozen liver tissues using Tru-Cut or fine-needle biopsies. The use of Wnt agonists proved important for canine organoid proliferation and inhibition of differentiation. Finally, we demonstrate that successful gene supplementation in hepatic organoids of COMMD1-deficient dogs restores function and can be an effective means to cure copper storage disease.",

author = "Sathidpak Nantasanti and Bart Spee and Kruitwagen, {Hedwig S} and Chen Chen and Niels Geijsen and Oosterhoff, {Loes A} and {van Wolferen}, {Monique E} and Nicolas Pelaez and Hille Fieten and Wubbolts, {Richard W} and Grinwis, {Guy C} and Jefferson Chan and Meritxell Huch and Vries, {Robert R G} and Hans Clevers and {de Bruin}, Alain and Jan Rothuizen and Penning, {Louis C} and Schotanus, {Baukje A}",

year = "2015",

month = oct,

day = "6",

doi = "10.1016/j.stemcr.2015.09.002",

language = "English",

journal = "Stem Cell Reports",

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publisher = "Cell Press",

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Nantasanti, S, Spee, B, Kruitwagen, HS, Chen, C, Geijsen, N, Oosterhoff, LA, van Wolferen, ME, Pelaez, N, Fieten, H, Wubbolts, RW, Grinwis, GC, Chan, J, Huch, M, Vries, RRG, Clevers, H, de Bruin, A, Rothuizen, J, Penning, LC & Schotanus, BA 2015, 'Disease Modeling and Gene Therapy of Copper Storage Disease in Canine Hepatic Organoids', Stem Cell Reports. https://doi.org/10.1016/j.stemcr.2015.09.002

TY - JOUR

T1 - Disease Modeling and Gene Therapy of Copper Storage Disease in Canine Hepatic Organoids

AU - Nantasanti, Sathidpak

AU - Spee, Bart

AU - Kruitwagen, Hedwig S

AU - Chen, Chen

AU - Geijsen, Niels

AU - Oosterhoff, Loes A

AU - van Wolferen, Monique E

AU - Pelaez, Nicolas

AU - Fieten, Hille

AU - Wubbolts, Richard W

AU - Grinwis, Guy C

AU - Chan, Jefferson

AU - Huch, Meritxell

AU - Vries, Robert R G

AU - Clevers, Hans

AU - de Bruin, Alain

AU - Rothuizen, Jan

AU - Penning, Louis C

AU - Schotanus, Baukje A

PY - 2015/10/6

Y1 - 2015/10/6

N2 - The recent development of 3D-liver stem cell cultures (hepatic organoids) opens up new avenues for gene and/or stem cell therapy to treat liver disease. To test safety and efficacy, a relevant large animal model is essential but not yet established. Because of its shared pathologies and disease pathways, the dog is considered the best model for human liver disease. Here we report the establishment of a long-term canine hepatic organoid culture allowing undifferentiated expansion of progenitor cells that can be differentiated toward functional hepatocytes. We show that cultures can be initiated from fresh and frozen liver tissues using Tru-Cut or fine-needle biopsies. The use of Wnt agonists proved important for canine organoid proliferation and inhibition of differentiation. Finally, we demonstrate that successful gene supplementation in hepatic organoids of COMMD1-deficient dogs restores function and can be an effective means to cure copper storage disease.

AB - The recent development of 3D-liver stem cell cultures (hepatic organoids) opens up new avenues for gene and/or stem cell therapy to treat liver disease. To test safety and efficacy, a relevant large animal model is essential but not yet established. Because of its shared pathologies and disease pathways, the dog is considered the best model for human liver disease. Here we report the establishment of a long-term canine hepatic organoid culture allowing undifferentiated expansion of progenitor cells that can be differentiated toward functional hepatocytes. We show that cultures can be initiated from fresh and frozen liver tissues using Tru-Cut or fine-needle biopsies. The use of Wnt agonists proved important for canine organoid proliferation and inhibition of differentiation. Finally, we demonstrate that successful gene supplementation in hepatic organoids of COMMD1-deficient dogs restores function and can be an effective means to cure copper storage disease.

U2 - 10.1016/j.stemcr.2015.09.002

DO - 10.1016/j.stemcr.2015.09.002

M3 - Article

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SN - 2213-6711

JO - Stem Cell Reports

JF - Stem Cell Reports

ER -

Disease Modeling and Gene Therapy of Copper Storage Disease in Canine Hepatic Organoids

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