A novel alpha-synuclein G14R missense variant is associated with atypical neuropathological features.

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Tác giả: Aishwarya Agarwal, Mohammed Al-Azzani, Leslie Amaral, Christof Brücke, Fiamma Buratti, Ruben Fernandez Busnadiego, Aswathy Chandran, Susana R Chaves, Ulf Dettmer, Claudio O Fernández, Ellen Gelpi, Janin Lautenschläger, Tiago Fleming Outeiro, Nagendran Ramalingam, Maria Ramón, Kevin Sicking, Rita L Sousa, Michael Zech, Alexander Zimprich, Markus Zweckstetter

Ngôn ngữ: eng

Ký hiệu phân loại: 594.38 *Pulmonata

Thông tin xuất bản: United States : medRxiv : the preprint server for health sciences , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 215503

BACKGROUND: Parkinson's disease (PD) affects millions of people worldwide, but only 5-10% of patients suffer from a monogenic form of the disease with Mendelian inheritance. AIM AND METHODS: A patient carrying aSyn missense mutation and his family members were studied. We present the clinical features, genetic testing - whole exome sequencing (WES), and neuropathological findings. The functional consequences of this aSyn variant were extensively investigated using biochemical, biophysical, and cellular assays. RESULTS: The patient exhibited a complex neurodegenerative disease that included generalized myocloni, bradykinesia, dystonia of the left arm and apraxia. WES identified a novel heterozygous SUMMARY: The atypical neuropathological features observed, which are reminiscent of those observed for the G51D aSyn variant, suggest a causal role of the
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