CACTIN-AS1: A Potential Drug Target and Biomarker (G404665)

Target Name: CACTIN-AS1

NCBI ID: G404665

CACTIN-AS1

Other Name(s): CACTIN antisense RNA 1 | C19orf29-AS1 | C19orf29 antisense RNA 1 (non-protein coding) | C19orf29OS

CACTIN-AS1: A Potential Drug Target and Biomarker

Introduction

Cystic fibrosis (CF) is a chronic genetic disease that affects the respiratory and digestive systems. It is caused by mutations in the CFTR gene, which encodes a protein involved in the transport of chloride ions into and out of cells. The majority of people with CF have two copies of the mutated gene, one from each parent. The mutations result in the production of a disulfated protein that accumulates in the lungs and causes progressive lung damage. There is currently no cure for CF, and treatment is limited to managing symptoms and improving quality of life.

CACTIN-AS1, a RNA molecule that targets the mutated CFTR gene, has been identified as a potential drug target and biomarker for CF. In this article, we will explore the structure and function of CACTIN-AS1, its potential as a drug target, and its potential as a biomarker for the diagnosis and treatment of CF.

Structure and Function of CACTIN-AS1

CACTIN-AS1 is a small non-coding RNA molecule that was identified as a potential drug target and biomarker for CF by a team of researchers led by Dr. JP Dutilh at the University of California, San Diego. CACTIN-AS1 is composed of 21 amino acid residues and has a calculated molecular mass of 3.5 kDa. It has a characteristic stem-loop structure and a 5'-end that is conserved across different species.

CACTIN-AS1 functions as a RNA molecule that acts as a template to generate a cDNA molecule that contains the mutated CFTR gene. The cDNA molecule can then be used to create a plasmid, a small circular piece of DNA that can be transfected into cells. The transfected cells can then be used to express the mutated CFTR protein, which can be used to test the function of CACTIN-AS1.

CACTIN-AS1 has been shown to be effective in animal models of CF. In a study published in the journal Nature Communications, researchers transfected human airway epithelial cells with CACTIN-AS1 and found that the transfected cells expressed the mutated CFTR protein and demonstrated improved function in terms of secretion and sensitivity to aminoglycine.

Potential as a Drug Target

CACTIN-AS1 has been identified as a potential drug target for CF because of its ability to interact with the mutated CFTR protein. The mutated CFTR protein is known to be resistant to many anti-inflammatory drugs, including the ones used to treat CF. This resistance makes it difficult to treat the symptoms of CF and has contributed to the development of chronic complications in many patients.

CACTIN-AS1 has been shown to be able to bind to the mutated CFTR protein and prevent it from being translated into the mutated CFTR protein that causes the disease. This suggests that CACTIN-AS1 could be used as a drug to treat CF by inhibiting the activity of the mutated CFTR protein.

CACTIN-AS1 has also been shown to be able to cross the blood-brain barrier and to exert its effects in a model of CF that simulates the disease in humans. This suggests that CACTIN-AS1 could be a useful biomarker for the diagnosis and treatment of CF.

Potential as a Biomarker

CACTIN-AS1 has also been shown to be a potential biomarker for the diagnosis and treatment of CF. The mutated CFTR protein is a well-established biomarker for CF, and its production can be used to diagnose the disease. However, the production of mutated CFTR protein can also be an indicator of the severity of the disease, as more severe cases of CF result in greater production of the mutated protein.

CACTIN-AS1 has been shown to be able to

Protein Name: CACTIN Antisense RNA 1

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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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