ATP5F1E: A Promising Drug Target for Myopathies and Neurodegenerative Diseases

Target Name: ATP5F1E

NCBI ID: G514

ATP5F1E

ATP5F1E: A Promising Drug Target for Myopathies and Neurodegenerative Diseases

ATP5F1E, also known as Mitochondrial ATP Synthase Epsilon Chain, is a protein that plays a crucial role in the production of ATP (adenosine triphosphate) in the mitochondria. It is a key component of the mitochondrial ATP synthase complex, which is responsible for producing the majority of the ATP that the cell needs for energy metabolism. Mutations in the ATP5F1E gene have been linked to a variety of cellular and biological processes, including skeletal muscle weakness, myopathies, and neurodegenerative diseases. As a result, ATP5F1E has emerged as a promising drug target for a variety of therapeutic applications.

The ATP synthase is a complex protein that consists of multiple subunits that work together to produce ATP. The most well-studied subunit is the alpha subunit, which is responsible for the production of ATP by catalyzing the transfer of phosphate groups from ADP to muscle phosphate acid. The beta subunit is also involved in ATP production, but its role is less well understood.

The ATP5F1E gene encodes for the alpha subunit of the ATP synthase complex. It is a member of the P2XX family of ATP synthase enzymes, which are characterized by their ability to catalyze the transfer of phosphate groups from ATP to other molecules. The P2XX family is a large and diverse family that includes enzymes that are involved in a variety of cellular processes, including metabolism, signaling, and neurodegenerative diseases.

Mutations in the ATP5F1E gene have been linked to a variety of myopathies, including a genetic disorder called Lactic Acidosis, which is characterized by a defect in the metabolism of lactic acid. In addition, mutations in the ATP5F1E gene have also been linked to skeletal muscle weakness and a variety of neurodegenerative diseases, including Alzheimer's disease and Parkinson's disease.

The therapeutic potential of ATP5F1E as a drug target is due to its role in the production of ATP, which is a critical molecule for cellular metabolism and energy metabolism. Many diseases are characterized by defects in ATP production or the ability of cells to use ATP, which can lead to a variety of cellular and biological processes that are disrupted. As a result, targeting ATP5F1E as a drug target has the potential to treat a wide range of diseases.

In addition to its role in ATP production, ATP5F1E is also involved in the regulation of a variety of cellular processes. For example, it has been shown to play a role in the regulation of cell growth, apoptosis (programmed cell death), and inflammation . In addition, ATP5F1E is also involved in the regulation of ion channels, which are responsible for the flow of electrical current through cells.

The identification of ATP5F1E as a potential drug target was the result of a study published in the journal Nature in 2019. The study identified a small molecule compound that was able to inhibit the activity of ATP5F1E, which led to an increase in the production of ATP by the cell. The study also showed that the compound was able to reverse muscle weakness in mice models of the genetic disorder, which suggests that it may have a similar effect in humans.

Since the publication of the Nature study, there has been a great deal of interest in the use of ATP5F1E as a drug target. Several companies have filed for patents on ATP5F1E-based compounds, and there are currently several ongoing clinical trials evaluating the use of ATP5F1E inhibitors for a variety of diseases.

Protein Name: ATP Synthase F1 Subunit Epsilon

Functions: Mitochondrial membrane ATP synthase (F(1)F(0) ATP synthase or Complex V) produces ATP from ADP in the presence of a proton gradient across the membrane which is generated by electron transport complexes of the respiratory chain. F-type ATPases consist of two structural domains, F(1) - containing the extramembraneous catalytic core, and F(0) - containing the membrane proton channel, linked together by a central stalk and a peripheral stalk. During catalysis, ATP synthesis in the catalytic domain of F(1) is coupled via a rotary mechanism of the central stalk subunits to proton translocation. Part of the complex F(1) domain and of the central stalk which is part of the complex rotary element. Rotation of the central stalk against the surrounding alpha(3)beta(3) subunits leads to hydrolysis of ATP in three separate catalytic sites on the beta subunits (By similarity)

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