Activation of the sigma-1 receptor chaperone alleviates symptoms of Wolfram syndrome in preclinical models.


Journal

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

Informations de publication

Date de publication:
09 02 2022
Historique:
entrez: 9 2 2022
pubmed: 10 2 2022
medline: 27 4 2022
Statut: ppublish

Résumé

The Wolfram syndrome is a rare autosomal recessive disease affecting many organs with life-threatening consequences; currently, no treatment is available. The disease is caused by mutations in the

Identifiants

pubmed: 35138910
doi: 10.1126/scitranslmed.abh3763
pmc: PMC9516885
mid: NIHMS1831267
doi:

Substances chimiques

Receptors, sigma 0
Calcium SY7Q814VUP

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

Pagination

eabh3763

Subventions

Organisme : Intramural NIH HHS
ID : Z01 DA000206
Pays : United States
Organisme : Intramural NIH HHS
ID : ZIA DA000549
Pays : United States

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Auteurs

Lucie Crouzier (L)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

Alberto Danese (A)

Department of Medical Sciences, Laboratory for Technologies of Advanced Therapies (LTTA), University of Ferrara, 44121 Ferrara, Italy.

Yuko Yasui (Y)

Cellular Pathobiology Section, Integrative Neuroscience Research Branch, Intramural Research Program, National Institute on Drug Abuse, NIH, 333 Cassell Drive, Baltimore, MD 21224, USA.

Elodie M Richard (EM)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

Jean-Charles Liévens (JC)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

Simone Patergnani (S)

Department of Medical Sciences, Laboratory for Technologies of Advanced Therapies (LTTA), University of Ferrara, 44121 Ferrara, Italy.

Simon Couly (S)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.
Cellular Pathobiology Section, Integrative Neuroscience Research Branch, Intramural Research Program, National Institute on Drug Abuse, NIH, 333 Cassell Drive, Baltimore, MD 21224, USA.

Camille Diez (C)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

Morgane Denus (M)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

Nicolas Cubedo (N)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

Mireille Rossel (M)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

Marc Thiry (M)

Laboratoire de Biologie Cellulaire, Université de Liège, GIGA-Neurosciences, Quartier Hopital, Avenue Hippocrate 15, 4000 Liege 1, Belgium.

Tsung-Ping Su (TP)

Cellular Pathobiology Section, Integrative Neuroscience Research Branch, Intramural Research Program, National Institute on Drug Abuse, NIH, 333 Cassell Drive, Baltimore, MD 21224, USA.

Paolo Pinton (P)

Department of Medical Sciences, Laboratory for Technologies of Advanced Therapies (LTTA), University of Ferrara, 44121 Ferrara, Italy.

Tangui Maurice (T)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

Benjamin Delprat (B)

MMDN, Univ Montpellier, EPHE, INSERM, Montpellier, France.

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Classifications MeSH