A closer look at TTC8: A Potential Drug Target and Biomarker for Bardet-Biedl Syndrome 8

Target Name: TTC8

NCBI ID: G123016

TTC8

A closer look at TTC8: A Potential Drug Target and Biomarker for Bardet-Biedl Syndrome 8

Bardet-Biedl syndrome (BBS) is a rare, progressive neurodegenerative disorder that is characterized by the progressive loss of small cells in the retina, leading to blindness. The most common form of BBS, BBS8, is a genetic disorder that is caused by a deficiency of the TTC8 gene. This gene is responsible for producing a protein known as TTC8, which plays a crucial role in the development and progression of BBS8.

TTC8: The Unveiled Target

TTC8 is a transmembrane protein that is expressed in the retina and is involved in the development and maintenance of the retinal neural stem cells. It is known for its role in the regulation of cell survival and has been implicated in the development of age-related macular degeneration (AMD) and other neurodegenerative disorders.

Recent studies have suggested that TTC8 may be a potential drug target for BBS8. By targeting TTC8, researchers may be able to reduce the progression of blindness in BBS8 patients. Additionally, TTC8 has also been identified as a potential biomarker for BBS8, which could be used to diagnose and monitor the disease.

The search for a drug target and biomarker for BBS8 has led to the development of small molecule inhibitors that target TTC8. These inhibitors have been shown to reverse the degenerative changes in the retina and improve vision in animal models of BBS8.

The Potential of TTC8 as a Drug Target

TTC8 has been identified as a potential drug target for BBS8 due to its role in the development and progression of the disease. Studies have shown that TTC8 levels are significantly decreased in the retina of BBS8 patients, and that restoring TTC8 levels to normal can improve retinal function and reduce the progression of blindness.

One of the most promising compounds that has been shown to target TTC8 is a small molecule inhibitor called RG-8162. RG-8162 is a potent inhibitor of TTC8 and has been shown to reverse the degenerative changes in the retina in animal models of BBS8.

In addition to its potential as a drug target, TTC8 has also been identified as a potential biomarker for BBS8. The levels of TTC8 have been shown to be significantly decreased in the retina of BBS8 patients, which could be used as a biomarker for the disease. This could be a promising approach for the development of diagnostic tests for BBS8.

The Potential of TTC8 as a Biomarker

TTC8 has also been identified as a potential biomarker for BBS8 due to its role in the development and progression of the disease. Studies have shown that TTC8 levels are significantly decreased in the retina of BBS8 patients, and that restoring TTC8 levels to normal can improve retinal function and reduce the progression of blindness.

The development of diagnostic tests for BBS8 has the potential to improve treatment outcomes for patients with the disease. The identification of TTC8 as a potential biomarker for BBS8 could be a valuable tool in the development of new diagnostic tests and therapies for the disease.

Conclusion

TTC8 is a transmembrane protein that is involved in the development and maintenance of the retinal neural stem cells. The progressive loss of small cells in the retina is a hallmark of Bardet-Biedl syndrome, and the recent development of small molecule inhibitors that target TTC8 have shown promise in reversing the degenerative changes in the retina and improving vision in animal models of BBS8.

Furthermore, TTC8 has also been identified as a potential drug target for BBS8, and studies have shown that it has the potential to

Protein Name: Tetratricopeptide Repeat Domain 8

Functions: The BBSome complex is thought to function as a coat complex required for sorting of specific membrane proteins to the primary cilia. The BBSome complex is required for ciliogenesis but is dispensable for centriolar satellite function. This ciliogenic function is mediated in part by the Rab8 GDP/GTP exchange factor, which localizes to the basal body and contacts the BBSome. Rab8(GTP) enters the primary cilium and promotes extension of the ciliary membrane. Firstly the BBSome associates with the ciliary membrane and binds to RAB3IP/Rabin8, the guanosyl exchange factor (GEF) for Rab8 and then the Rab8-GTP localizes to the cilium and promotes docking and fusion of carrier vesicles to the base of the ciliary membrane. The BBSome complex, together with the LTZL1, controls SMO ciliary trafficking and contributes to the sonic hedgehog (SHH) pathway regulation. Required for proper BBSome complex assembly and its ciliary localization

The "TTC8 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 TTC8 comprehensively, including but not limited to:
•   general information;
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•   protein biological mechanisms;
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•   the target screening and validation;
•   expression level;
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•   related combination drugs;
•   pharmacochemistry experiments;
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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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