Gene-edited human stem cell-derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice.

Animals Cell Differentiation Diabetes Mellitus / metabolism Gene Editing Humans Induced Pluripotent Stem Cells / metabolism Insulin / metabolism Insulin Secretion Insulin-Secreting Cells / metabolism Mice

Journal

Science translational medicine
ISSN: 1946-6242
Titre abrégé: Sci Transl Med
Pays: United States
ID NLM: 101505086

Informations de publication

Date de publication:
22 04 2020
Historique:
received: 03 05 2019
revised: 11 10 2019
accepted: 29 03 2020
entrez: 24 4 2020
pubmed: 24 4 2020
medline: 24 6 2021
Statut: ppublish

Résumé

Differentiation of insulin-producing pancreatic β cells from induced pluripotent stem cells (iPSCs) derived from patients with diabetes promises to provide autologous cells for diabetes cell replacement therapy. However, current approaches produce patient iPSC-derived β (SC-β) cells with poor function in vitro and in vivo. Here, we used CRISPR-Cas9 to correct a diabetes-causing pathogenic variant in Wolfram syndrome 1 (

Identifiants

DOI: 10.1126/scitranslmed.aax9106 PMID: 32321868 PMC: PMC7233417
pubmed: 32321868
pii: 12/540/eaax9106
doi: 10.1126/scitranslmed.aax9106
pmc: PMC7233417
mid: NIHMS1585432
pii:
doi:

Substances chimiques

Insulin 0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : NCATS NIH HHS
ID : UH3 TR002065
Pays : United States
Organisme : NIDDK NIH HHS
ID : P30 DK056341
Pays : United States
Organisme : NIDDK NIH HHS
ID : T32 DK108742
Pays : United States
Organisme : NIGMS NIH HHS
ID : R25 GM103757
Pays : United States
Organisme : NIDDK NIH HHS
ID : R01 DK114233
Pays : United States
Organisme : NCI NIH HHS
ID : P30 CA091842
Pays : United States
Organisme : NIDDK NIH HHS
ID : P30 DK020579
Pays : United States
Organisme : NIDDK NIH HHS
ID : R01 DK112921
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR000448
Pays : United States
Organisme : NIDDK NIH HHS
ID : T32 DK007120
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR002345
Pays : United States
Organisme : NCATS NIH HHS
ID : UH2 TR002065
Pays : United States

Informations de copyright

Copyright © 2020 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.

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Auteurs

Kristina G Maxwell (KG)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
Department of Biomedical Engineering, Washington University in St. Louis, 1 Brookings Drive, St. Louis, MO 63130, USA.
ORCID: 0000-0003-0869-1662

Punn Augsornworawat (P)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
Department of Biomedical Engineering, Washington University in St. Louis, 1 Brookings Drive, St. Louis, MO 63130, USA.
ORCID: 0000-0001-8602-5433

Leonardo Velazco-Cruz (L)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
ORCID: 0000-0003-4664-3936

Michelle H Kim (MH)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
ORCID: 0000-0003-0096-8276

Rie Asada (R)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
ORCID: 0000-0001-8347-6909

Nathaniel J Hogrebe (NJ)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
ORCID: 0000-0002-6282-2056

Shuntaro Morikawa (S)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
ORCID: 0000-0001-5547-9491

Fumihiko Urano (F)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA. jmillman@wustl.edu urano@wustl.edu.
Department of Pathology and Immunology, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
ORCID: 0000-0002-9944-6293

Jeffrey R Millman (JR)

Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA. jmillman@wustl.edu urano@wustl.edu.
Department of Biomedical Engineering, Washington University in St. Louis, 1 Brookings Drive, St. Louis, MO 63130, USA.
ORCID: 0000-0003-0426-3492

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Phénomènes physiologiques cellulaires › Différenciation cellulaire › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Maladies endocriniennes › Diabète › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Édition de gène › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Cellules › Cellules souches › Cellules souches pluripotentes › Cellules souches pluripotentes induites › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Acides aminés, peptides et protéines › Peptides › Hormones peptidiques › Hormones pancréatiques › Insulines › Insuline › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Phénomènes physiologiques › Sécrétion d'insuline › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Cellules › Cellules épithéliales › Cellules entéroendocrines › Cellules à insuline › Gene-edited human stem cell-derived β cells...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Gene-edited human stem cell-derived β cells...