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RNU4-2 Gene-Engineered Cell Models Products

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RNU4-2, a key non-coding RNA in the spliceosome, is crucial for RNA splicing and brain function. Studies link its variants to neurodevelopmental disorders via splicing errors and dysfunctional proteins, causing conditions like intellectual disability and seizures. Creative Biolabs offers RNU4-2 cell models to expedite research into these diseases and new treatments. To know more about our RNU4-2 cell models, please contact us.

You can explore our RNU4-2 model categories below, or view our Product List.

Featured RNU4-2 Gene-Engineered Cell Model Categories

Our RNU4-2 gene-engineered cell models encompass a variety of modifications, including:

RNU4-2 Mutated Cell Lines: Incorporate precise RNU4-2 mutations, such as n.64_65insT, n.77_78insT, and n.65A>G, enabling researchers to study the impact of specific disease-associated variants. These models are crucial tools for dissecting how specific mutations alter the RNA splicing process, leading to the production of aberrant mRNA transcripts and ultimately contributing to neurological dysfunction.
RNU4-2 Knockout Cell Lines: These are designed with the RNU4-2 gene completely knocked out, allowing us to investigate the phenotypic consequences of its complete absence. By doing this, we can mimic the effects of a total gene deletion or loss-of-function variants, and that can really throw a wrench in the spliceosome machinery. These models are incredibly useful for figuring out exactly what RNU4-2 normally does in our cells and how its absence kicks off disease. Researchers can then use these tools to dig into how losing RNU4-2 changes the bigger picture of gene activity, messes with splicing, and affects other important cell processes.
RNU4-2 Knock-in Cell Lines: By integrating patient-derived RNU4-2 mutations, these models facilitate the dissection of disease mechanisms. Distinct from knockout lines lacking RNU4-2, knock-in models introduce refined, clinically relevant sequence alterations. This enables the investigation of how particular mutations modulate RNA splicing, gene expression, and cellular function. These models are very useful. They help us look closely at what happens when RNA splicing goes wrong.
RNU4-2 Overexpression Cell Lines: These models, engineered to express increased RNU4-2 levels, enable the investigation of potential gain-of-function phenotypes. Although RNU4-2 loss-of-function commonly underlies neurological disorders, examining overexpression effects yields valuable insights. For instance, elevated RNU4-2 may perturb spliceosome stoichiometry, causing splicing defects. These models help us learn how RNU4-2 is controlled and how it works.

Features of Our RNU4-2 Gene-Engineered Cell Models

Feature	Description
Precise Gene Editing	Utilizing cutting-edge technology, we ensure accurate and targeted modification of the RNU4-2 gene. This technology enables the precise introduction of specific mutations or alterations. This minimizes unintended off-target effects and ensures the reliability of the generated cell models.
Disease Relevance	Cell models are designed to recapitulate key aspects of neurological diseases associated with RNU4-2 dysfunction. These models accurately mimic the genetic alterations seen in patients. Therefore, they provide a valuable tool for studying the underlying disease mechanisms and for developing targeted therapeutic interventions.
Rigorous Validation	All cell models undergo thorough validation to confirm the intended genetic modification and ensure optimal performance. This includes techniques such as Sanger sequencing, PCR analysis, and functional assays to verify the correct gene editing and the resulting phenotypic changes.

Advantages of Our RNU4-2 Gene-Engineered Cell Models

Accelerated Research: These models provide a powerful tool to investigate disease mechanisms, screen potential drug candidates, and advance your research more efficiently. Compared to traditional animal models, they offer higher throughput and shorter generation times, thereby accelerating the research pipeline.
Improved Accuracy: Our refined gene-editing methodologies guarantee the creation of highly precise and dependable models. In contrast to conventional approaches employing stochastic mutagenesis or selective breeding, our technology achieves exceptional accuracy. This precision yields more reproducible and pertinent data for downstream analyses.
Reduced Costs: For many research applications, cell-based models offer a cost-effective alternative to animal models. They require fewer resources, including reduced labor, reagents, and maintenance, significantly lowering overall research expenses.
Ethical Considerations: Utilizing cell models can help reduce the reliance on animal experimentation, aligning with the growing emphasis on ethical research practices and the principles of the 3Rs (Replacement, Reduction, and Refinement).

A picture presents Schematic of U4 binding to U6 snRNA. (OA Literature)

Fig.1 Schematic of U4 (teal) binding to U6 snRNA (grey).¹

RNU4-2 Gene: Mechanisms and Principles

The RNU4-2 gene's role is to direct the production of U4 snRNA. U4 snRNA is a very important part of the spliceosome. The spliceosome is a complex machine. This machine's job is to remove introns from pre-mRNA. After removing introns, the exons can join together. This joining forms mature mRNA. This whole process is very important. It helps make many different kinds of proteins. These proteins are needed for cells to work correctly. This is especially true when the brain is growing.

Also, changes in RNU4-2 can affect certain areas. These areas include the T-loop and stem III. When these areas are affected, RNU4-2 cannot connect with U6 snRNA as well as it should. Because of this, the spliceosome cannot find and process the correct places on the RNA. This causes wrong splicing. It also changes how genes are expressed. In the end, this can lead to the start of brain development problems.

FAQs

What types of RNU4-2 variants do your cell models cover?
For RNU4-2 variants in our cell models, we can offer the common mutations, such as n.64_65insT. Also, we can make models for other special variants. Please ask us about what you need.
What cell types are available for the RNU4-2 gene-engineered cell models?
For our RNU4-2 gene-engineered cell models, we primarily employ neuronal progenitor cells and induced pluripotent stem cells (iPSCs). These cell types exhibit high relevance to the study of neurodevelopmental disorders. While these are our standard offerings, additional cell types may be available. Furthermore, we possess the capability to generate specific cell types based on your research requirements.
Are these cell models suitable for drug screening?
Indeed, our RNU4-2 gene-engineered cell models are highly suitable for drug screening assays. They enable the evaluation of both the efficacy and the specificity profiles of prospective therapeutic compounds.
Why is RNU4-2 important for neurological function?
RNU4-2 is important for correct RNA splicing. This process is needed to make proteins. These proteins help the brain grow and work well.
Are RNU4-2 mutations common in neurological disorders?
RNU4-2 changes might cause brain development problems more often than we thought before.

Creative Biolabs is a partner you can trust. We help develop RNU4-2 cell models. These models have high quality. We know a lot about changing genes. Also, we are dedicated to making progress in research on brain diseases. Because of these things, we are the best choice for your next project. Contact us today to discuss your specific requirements and learn how our RNU4-2 cell models can accelerate your research.