SMN Deficiency Destabilizes ABCA1 Expression in Human Fibroblasts: Novel Insights in Pathophysiology of Spinal Muscular Atrophy.


Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
02 Feb 2023
Historique:
received: 28 12 2022
revised: 20 01 2023
accepted: 31 01 2023
entrez: 11 2 2023
pubmed: 12 2 2023
medline: 15 2 2023
Statut: epublish

Résumé

The deficiency of survival motor neuron protein (SMN) causes spinal muscular atrophy (SMA), a rare neuromuscular disease that affects different organs. SMN is a key player in RNA metabolism regulation. An intriguing aspect of SMN function is its relationship with plasma membrane-associated proteins. Here, we provide a first demonstration that SMN affects the ATP-binding cassette transporter A1, (ABCA1), a membrane protein critically involved in cholesterol homeostasis. In human fibroblasts, we showed that SMN associates to ABCA1 mRNA, and impacts its subcellular distribution. Consistent with the central role of ABCA1 in the efflux of free cholesterol from cells, we observed a cholesterol accumulation in SMN-depleted human fibroblasts. These results were also confirmed in SMA type I patient-derived fibroblasts. These findings not only validate the intimate connection between SMN and plasma membrane-associated proteins, but also highlight a contribution of dysregulated cholesterol efflux in SMA pathophysiology.

Identifiants

pubmed: 36769246
pii: ijms24032916
doi: 10.3390/ijms24032916
pmc: PMC9917534
pii:
doi:

Substances chimiques

Transcription Factors 0
Membrane Proteins 0
Survival of Motor Neuron 1 Protein 0
ABCA1 protein, human 0
ATP Binding Cassette Transporter 1 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

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Auteurs

Francesca Gabanella (F)

CNR-Institute of Biochemistry and Cell Biology, Department of Sense Organs, Sapienza University of Rome, Viale del Policlinico 155, 00161 Rome, Italy.

Annalisa Onori (A)

CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University of Rome, Viale Regina Elena 291, 00161 Rome, Italy.

Cinzia Pisani (C)

CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University of Rome, Viale Regina Elena 291, 00161 Rome, Italy.

Marco Fiore (M)

CNR-Institute of Biochemistry and Cell Biology, Department of Sense Organs, Sapienza University of Rome, Viale del Policlinico 155, 00161 Rome, Italy.

Giampiero Ferraguti (G)

Department of Experimental Medicine, Sapienza University of Rome, Viale del Policlinico 155, 00161 Rome, Italy.

Andrea Colizza (A)

Department of Sense Organs, Sapienza University of Rome, Viale del Policlinico 155, 00161 Rome, Italy.

Marco de Vincentiis (M)

Department of Sense Organs, Sapienza University of Rome, Viale del Policlinico 155, 00161 Rome, Italy.

Marco Ceccanti (M)

Center for Rare Neuromuscular Diseases, Department of Human Neuroscience, Policlinico Umberto I, Sapienza University of Rome, 00185 Rome, Italy.

Maurizio Inghilleri (M)

Center for Rare Neuromuscular Diseases, Department of Human Neuroscience, Policlinico Umberto I, Sapienza University of Rome, 00185 Rome, Italy.

Nicoletta Corbi (N)

CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University of Rome, Viale Regina Elena 291, 00161 Rome, Italy.

Claudio Passananti (C)

CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University of Rome, Viale Regina Elena 291, 00161 Rome, Italy.

Maria Grazia Di Certo (MG)

CNR-Institute of Biochemistry and Cell Biology, Department of Sense Organs, Sapienza University of Rome, Viale del Policlinico 155, 00161 Rome, Italy.

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Classifications MeSH