Potential Drug Target for NM3 (G58)

Target Name: ACTA1

NCBI ID: G58

ACTA1

Potential Drug Target for NM3

ACTA1 (alpha-actinin-1) is a protein that is expressed in various tissues throughout the body, including muscle, heart, and brain. It is a member of the actinin family, which is a group of proteins that play a critical role in cell signaling and cytoskeletal organization.

ACTA1 is also known as nemaline myopathy type 3 (NM3), which is a genetic disorder that affects muscle strength and function. People with NM3 have progressive muscle weakness and wasting, which can lead to difficulty with daily activities and a decreased quality of life.

The exact cause of NM3 is not known, but it is thought to be related to a deficiency of dystrophin, a protein that helps keep muscle cells intact. Without dystrophin, muscle cells break down and are replaced with scar tissue, leading to progressive muscle weakness and wasting.

ACTA1 is a potential drug target for NM3 because it is involved in the regulation of dystrophin function. Studies have shown that ACTA1 levels are decreased in muscle cells from individuals with NM3, and that inhibiting ACTA1 activity can increase dystrophin levels in these cells. This suggests that ACTA1 could be a useful target for treating NM3 by increasing dystrophin levels and improving muscle strength and function.

In addition to its potential as a drug target, ACTA1 is also a potential biomarker for NM3. The loss of muscle mass and strength is a common symptom of NM3, and ACTA1 levels may be a useful indicator of the severity of this loss. This could make ACTA1 an attractive target for diagnostic tools, such as muscle biopsies or blood tests.

While further research is needed to fully understand the role of ACTA1 in NM3, its potential as a drug target and biomarker is an exciting area of study. If ACTA1 is found to be a useful target for treating NM3, it could lead to new treatments for this progressive muscle disorder that could improve the quality of life for those affected.

Protein Name: Actin Alpha 1, Skeletal Muscle

Functions: Actins are highly conserved proteins that are involved in various types of cell motility and are ubiquitously expressed in all eukaryotic cells

The "ACTA1 Target / Biomarker Review Report" is a customizable review of hundreds up to thousends of related scientific research literature by AI technology, covering specific information about ACTA1 comprehensively, including but not limited to:
•   general information;
•   protein structure and compound binding;
•   protein biological mechanisms;
•   its importance;
•   the target screening and validation;
•   expression level;
•   disease relevance;
•   drug resistance;
•   related combination drugs;
•   pharmacochemistry experiments;
•   related patent analysis;
•   advantages and risks of development, etc.
The report is helpful for project application, drug molecule design, research progress updates, publication of research papers, patent applications, etc. If you are interested to get a full version of this report, please feel free to contact us at BD@silexon.ai

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