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2,500 followers

6mo

✨ The B6-HATXN3 mouse model is a significant advancement in SCA3 research, offering crucial insights into the genetic mutations causing spinocerebellar ataxia type 3. ✅ This model closely mirrors SCA3's pathology, aiding in preclinical studies, biomarker identification, and therapy evaluation. ✅ The TurboKnockout technology enhances its versatility for genetic diversity representation in patients, supporting drug screening and pharmacological studies. Cyagen's introduction of the B6-HATXN3 model highlights their commitment to medical research progress in SCA3 treatment. Learn more about this mouse model here: https://lnkd.in/d9iRFQmr #mousemodels #preclinicalresearch #research #science #biotech #biotechnology #genetherapy

Transcript

Hello, I'm Doctor Reza Rezai from Seigen. Today I'm excited to introduce a significant breakthrough in neurodegenerative disease research, the B66N3 mouse model. This model is engineered to advance our understanding and development of treatments for spinal cerebellar ataxia type 3, also known as Machado Joseph disease. Join me as we explore this models development, its pivotal role in current research and how it can facilitate the discovery of effective therapies. Spinal Cerebellar ataxia type 3 for SCA Three is the most common form of SCA worldwide. It's a neurodegenerative disorder that primarily affects motor control, with symptoms worsening overtime. The disease is marked by a progressive decline in muscle coordination and balance, severely impacting patients quality of life. That's a three results from genetic mutations that lead to abnormal expansions of CHG trinucleotide repeats in the ATXN 3 gene. These expansions caused the encoded attacks in three proteins to misfold an aggregate, disrupting normal cellular functions and leading to neuronal death, particularly in the cerebellum and other parts of the brain responsible for motor functions. Studying SCP-3 is challenging due to the complex nature of its pathogenesis and the variability in symptom severity and progression among patients. These factors complicate the development of models that accurately reflect the human condition and test potential therapies effectively. Arby's 6H TX N3 mouse model is specifically designed to overcome these challenges. By exclusively expressing the human ATX and three gene, it closely mimics the diseases human pathology, providing a more reliable platform for studying the molecular mechanisms and testing interventions that could potentially modify the diseases course. To be 6 HTX N3 model is instrumental in preclinical studies aimed at understanding the diseases, Natural History, identifying biomarkers for early diagnosis and evaluating the efficacy of potential therapies including gene therapy, RNA interference and small molecule inhibitors that target protein aggregation. Using our proprietary Turbo Knockout technology, we can tailor the B6 Hixon 3 model to include specific pathogenic mutations or mimic particular patient genetic profiles. This customization facilitates precise, targeted research that reflects the genetic diversity scene and ESCA three patients, enhancing the Modell's utility for drug screening and pharmacological studies. To be 6 Pitx and three model is not just a tool for basic research, it's a pivotal resource in the translational pathway that bridges bench research in clinical application. It helps researchers develop and refine therapeutic strategies that could one day alleviate or even reverse the effects of ESCA 3. Thank you for joining me today to explore the B6 Atxn 3 mouse model and it's significant impact on SCA 3 research. At Siegen, we are committed to providing innovative solutions that help push the boundaries of medical research. For more information or to discuss how we can support your research needs, please don't hesitate to reach out to us.

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Cyagen

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