ATXN3: A Potential Drug Target and Biomarker for Neurodegenerative Diseases

Target Name: ATXN3

NCBI ID: G4287

ATXN3: A Potential Drug Target and Biomarker for Neurodegenerative Diseases

ATXN3 (also known as Josephin) is a protein that is expressed in the brain and is known for its role in the development and progression of neurodegenerative diseases, such as Alzheimer's disease and Parkinson's disease. The protein is composed of 120 amino acids and has a calculated molecular weight of 13.9 kDa.

One of the unique features of ATXN3 is its ability to interact with other proteins, particularly with the protein known as Tau. Tau is a protein that is involved in the formation of neurofibrillary tangles, which are a hallmark of neurodegenerative diseases. By interacting with Tau, ATXN3 has been shown to contribute to the development of neurofibrillary tangles and to increase the risk of neurodegeneration.

In addition to its role in neurodegenerative diseases, ATXN3 has also been shown to have potential as a drug target. One reason for this is the fact that ATXN3 is a protein that is expressed in the brain and is involved in the development and progression of neurodegenerative diseases. This means that any drug that targets ATXN3 may have specific effects on the brain and may be more effective than drugs that do not target this protein.

Another reason for ATXN3's potential as a drug target is its structure. The amino acid sequence of ATXN3 is highly conserved and is similar to that of other proteins that are involved in the development and progression of neurodegenerative diseases. This suggests that any drug that targets ATXN3 may have similar effects to drugs that target other proteins that are involved in the same process.

In addition to its potential as a drug target, ATXN3 is also a potential biomarker for neurodegenerative diseases. The fact that ATXN3 is a protein that is expressed in the brain and is involved in the development and progression of neurodegenerative diseases makes it an attractive candidate for use as a biomarker. This is because any changes in the level of ATXN3 in the brain may be an indication of the early stages of neurodegeneration that is developing.

Overall, ATXN3 is a protein that has the potential to be a drug target and a biomarker for neurodegenerative diseases. Further research is needed to fully understand the role of ATXN3 in these processes and to develop effective treatments for neurodegenerative diseases.

Protein Name: Ataxin 3

Functions: Deubiquitinating enzyme involved in protein homeostasis maintenance, transcription, cytoskeleton regulation, myogenesis and degradation of misfolded chaperone substrates (PubMed:12297501, PubMed:17696782, PubMed:23625928, PubMed:28445460, PubMed:16118278). Binds long polyubiquitin chains and trims them, while it has weak or no activity against chains of 4 or less ubiquitins (PubMed:17696782). Involved in degradation of misfolded chaperone substrates via its interaction with STUB1/CHIP: recruited to monoubiquitinated STUB1/CHIP, and restricts the length of ubiquitin chain attached to STUB1/CHIP substrates and preventing further chain extension (By similarity). Interacts with key regulators of transcription and represses transcription: acts as a histone-binding protein that regulates transcription (PubMed:12297501). Regulates autophagy via the deubiquitination of 'Lys-402' of BECN1 leading to the stabilization of BECN1 (PubMed:28445460)

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