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/Manipulating Golgi Ions For Treatment Of Neurodegenerative Disease And Cutaneous Disorders
Abstract

The invention provides that the expression and/or activity of metal ion transporter, such as a Ca2+/Mn2+ transporter and secretory pathway calcium ATPase 1 (SPCA1) or SPCA2 is associated with the restoration and conservation of Golgi integrity in cells, as well as with the restoration of Golgi ion concentrations. Provided herein are methods of restoring or maintaining Golgi integrity in a cell and methods of modulating Golgi ion concentration is a cell including increasing the expression and/or activity of a metal ion transporter such as SPCA1 or SPCA2 protein in the cell. The invention further provides methods of treating neurodegenerative disease, such as amyotrophic lateral sclerosis (ALS). The methods of treatment include increasing the expression and/or activity of a metal ion transporter such as SPCA1 or SPCA2 protein in cells to restore or maintain Golgi integrity and Golgi ion concentration in the cell.

Full Text

What is claimed is:

The invention provides that the expression and/or activity of metal ion transporter, such as a Ca2+/Mn2+ transporter and secretory pathway calcium ATPase 1 (SPCA1) or SPCA2 is associated with the restoration and conservation of Golgi integrity in cells, as well as with the restoration of Golgi ion concentrations. Provided herein are methods of restoring or maintaining Golgi integrity in a cell and methods of modulating Golgi ion concentration is a cell including increasing the expression and/or activity of a metal ion transporter such as SPCA1 or SPCA2 protein in the cell. The invention further provides methods of treating neurodegenerative disease, such as amyotrophic lateral sclerosis (ALS). The methods of treatment include increasing the expression and/or activity of a metal ion transporter such as SPCA1 or SPCA2 protein in cells to restore or maintain Golgi integrity and Golgi ion concentration in the cell.
Timeline
Filed
02/18/2026
Published
06/18/2026
Granted
Not Available
IPC Codes(4)
A61K 48/00:Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 38/43:Enzymes; Proenzymes; Derivatives thereof
A61P 25/28:for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia