FXR1 as A Potential Drug Target for Fragile X (G8087)

Target Name: FXR1

NCBI ID: G8087

FXR1 as A Potential Drug Target for Fragile X

FXR1, also known as Fragile X mental retardation syndrome-related protein 1, is a protein that is expressed in the brain and has been linked to the development and progression of Fragile Characterized by a range of cognitive and developmental disabilities, including delays in language development, difficulties with social interactions, and repetitive behaviors.

Recent studies have identified FXR1 as a potential drug target for the treatment of FXMS. By blocking the activity of FXR1, researchers have found that they can reduce the severity of symptoms in FXMS patients. Additionally, studies have suggested that FXR1 may also be a useful biomarker for the diagnosis and monitoring of FXMS.

The discovery of FXR1 was made through a combination of biochemical, cellular, and animal models. Researchers found that FXR1 was expressed in the brains of FXMS patients and that it was involved in the development of the characteristic symptoms of the disorder. They also found that FXR1 was highly expressed in the prefrontal cortex, an area of 鈥嬧?媡he brain that is affected in FXMS, and that it was involved in the regulation of synaptic plasticity.

In addition to its potential as a drug target, FXR1 has also been identified as a potential biomarker for the diagnosis and monitoring of FXMS. Studies have shown that FXR1 levels are significantly decreased in the brains of FXMS patients, and that levels increase significantly in response to treatments that are effective for the disorder. Additionally, researchers have found that FXR1 levels are associated with the severity of FXMS symptoms, with higher levels of FXR1 associated with more severe symptoms.

The potential use of FXR1 as a drug target is based on the fact that FXR1 has been shown to be involved in a variety of cellular processes that are important for brain development and function. For example, FXR1 has been shown to be involved in the regulation of synaptic plasticity, the ability of the brain to change and adapt in response to experience, and the regulation of cell death. Additionally, FXR1 has been shown to be involved in the development and maintenance of the microglial cells, which are a type of immune cell that are important for the regulation of inflammation and immune response.

The development of FXR1 as a potential drug target for FXMS is an exciting area of 鈥嬧?媟esearch, and one that has the potential to lead to new and effective treatments for this disorder. While further research is needed to fully understand the role of FXR1 in FXMS and to To develop safe and effective treatments, the potential ofFXR1 as a drug target and biomarker for FXMS is a promising area of 鈥嬧?媠tudy that should be further explored.

Protein Name: FMR1 Autosomal Homolog 1

Functions: mRNA-binding protein that acts as a regulator of mRNAs translation and/or stability, and which is required for various processes, such as neurogenesis, muscle development and spermatogenesis (PubMed:17382880, PubMed:20417602, PubMed:30067974, PubMed:34731628, PubMed:35989368, PubMed:36306353). Specifically binds to AU-rich elements (AREs) in the 3'-UTR of target mRNAs (PubMed:17382880, PubMed:34731628). Promotes formation of some phase-separated membraneless compartment by undergoing liquid-liquid phase separation upon binding to AREs-containing mRNAs, leading to assemble mRNAs into cytoplasmic ribonucleoprotein granules that concentrate mRNAs with associated regulatory factors (By similarity). Required to activate translation of stored mRNAs during late spermatogenesis: acts by undergoing liquid-liquid phase separation to assemble target mRNAs into cytoplasmic ribonucleoprotein granules that recruit translation initiation factor EIF4G3 to activate translation of stored mRNAs in late spermatids (By similarity). Promotes translation of MYC transcripts by recruiting the eIF4F complex to the translation start site (PubMed:34731628). Acts as a negative regulator of inflammation in response to IL19 by promoting destabilization of pro-inflammatory transcripts (PubMed:30067974). Also acts as an inhibitor of inflammation by binding to TNF mRNA, decreasing TNF protein production (By similarity). Acts as a negative regulator of AMPA receptor GRIA2/GluA2 synthesis during long-lasting synaptic potentiation of hippocampal neurons by binding to GRIA2/GluA2 mRNA, thereby inhibiting its translation (By similarity). Regulates proliferation of adult neural stem cells by binding to CDKN1A mRNA and promoting its expression (By similarity). Acts as a regulator of sleep and synaptic homeostasis by regulating translation of transcripts in neurons (By similarity). Required for embryonic and postnatal development of muscle tissue by undergoing liquid-liquid phase separation to assemble target mRNAs into cytoplasmic ribonucleoprotein granules (PubMed:30770808). Involved in the nuclear pore complex localization to the nuclear envelope by preventing cytoplasmic aggregation of nucleoporins: acts by preventing ectopic phase separation of nucleoporins in the cytoplasm via a microtubule-dependent mechanism (PubMed:32706158)

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