The Deubiquitinating Enzyme Ataxin-3 Regulates Ciliogenesis and Phagocytosis in the Retina.


Por: Toulis V, García-Monclús S, de la Peña-Ramírez C, Arenas-Galnares R, Abril JF, Todi SV, Khan N, Garanto A, Costa MDC and Marfany G

Publicada: 10 nov 2020
Categoría: Biochemistry, genetics and molecular biology (miscellaneous)

Resumen:
Expansion of a CAG repeat in ATXN3 causes the dominant polyglutamine disease spinocerebellar ataxia type 3 (SCA3), yet the physiological role of ATXN3 remains unclear. Here, we focus on unveiling the function of Ataxin-3 (ATXN3) in the retina, a neurological organ amenable to morphological and physiological studies. Depletion of Atxn3 in zebrafish and mice causes morphological and functional retinal alterations and, more precisely, photoreceptor cilium and outer segment elongation, cone opsin mislocalization, and cone hyperexcitation. ATXN3 localizes at the basal body and axoneme of the cilium, supporting its role in regulating ciliary length. Abrogation of Atxn3 expression causes decreased levels of the regulatory protein KEAP1 in the retina and delayed phagosome maturation in the retinal pigment epithelium. We propose that ATXN3 regulates two relevant biological processes in the retina, namely, ciliogenesis and phagocytosis, by modulating microtubule polymerization and microtubule-dependent retrograde transport, thus positing ATXN3 as a causative or modifier gene in retinal/macular dystrophies.

Filiaciones:
Toulis V:
 Departament de Genètica, Microbiologia i Estadística, Avda. Diagonal 643, Universitat de Barcelona, Barcelona 08028, Spain

 CIBERER, ISCIII, Universitat de Barcelona, Barcelona, Spain

 Department of Neurology, Michigan Medicine, University of Michigan, Ann Arbor, MI, USA

García-Monclús S:
 Departament de Genètica, Microbiologia i Estadística, Avda. Diagonal 643, Universitat de Barcelona, Barcelona 08028, Spain

de la Peña-Ramírez C:
 Departament de Genètica, Microbiologia i Estadística, Avda. Diagonal 643, Universitat de Barcelona, Barcelona 08028, Spain

Arenas-Galnares R:
 Departament de Genètica, Microbiologia i Estadística, Avda. Diagonal 643, Universitat de Barcelona, Barcelona 08028, Spain

 Institute of Biomedicine (IBUB, IBUB-IRSJD), Universitat de Barcelona, Barcelona, Spain

Abril JF:
 Departament de Genètica, Microbiologia i Estadística, Avda. Diagonal 643, Universitat de Barcelona, Barcelona 08028, Spain

 Institute of Biomedicine (IBUB, IBUB-IRSJD), Universitat de Barcelona, Barcelona, Spain

Todi SV:
 Department of Pharmacology, Wayne State University School of Medicine, Detroit, MI, USA

 Department of Neurology, Wayne State University School of Medicine, Detroit, MI, USA

Khan N:
 Department of Ophthalmology and Visual Sciences, W. K. Kellogg Eye Center, University of Michigan, Ann Arbor, MI, USA

Garanto A:
 Departament de Genètica, Microbiologia i Estadística, Avda. Diagonal 643, Universitat de Barcelona, Barcelona 08028, Spain

 Department of Human Genetics, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands

 Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, the Netherlands

Costa MDC:
 Department of Neurology, Michigan Medicine, University of Michigan, Ann Arbor, MI, USA

Marfany G:
 Departament de Genètica, Microbiologia i Estadística, Avda. Diagonal 643, Universitat de Barcelona, Barcelona 08028, Spain

 CIBERER, ISCIII, Universitat de Barcelona, Barcelona, Spain

 Institute of Biomedicine (IBUB, IBUB-IRSJD), Universitat de Barcelona, Barcelona, Spain
ISSN: 26391856





Cell Reports
Editorial
CELL PRESS, 50 HAMPSHIRE ST, FLOOR 5, CAMBRIDGE, MA 02139, Estados Unidos America
Tipo de documento: Article
Volumen: 33 Número: 6
Páginas: 108360-108360
WOS Id: 000590349200005
ID de PubMed: 33176149
imagen Green Accepted, gold, Green Published

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