Spinal Muscular Atrophy (SMA) Caenorhabditis elegans Model Development Service

Accelerate Your Neurological Drug Discovery! Are you currently facing long drug development cycles, limited translational models, or challenges in understanding complex disease mechanisms and identifying novel therapeutic targets for neurological disorders? Our C. elegans Spinal muscular atrophy (SMA) Models help you accelerate drug discovery, elucidate disease mechanisms, and identify high-potential therapeutic candidates through advanced genetic manipulation, high-throughput screening platforms, and comprehensive phenotypic and molecular assays.

SMA is a severe neuromuscular disorder stemming from defects in the Survival Motor Neuron 1 (SMN1) gene, leading to progressive motor neuron loss. Caenorhabditis elegans has emerged as a powerful model, possessing the smn-1 ortholog and conserved disease pathways. This platform offers a robust, cost-effective solution for dissecting SMA mechanisms and accelerating therapeutic discovery.

How Our C. elegans SMA Models Can Assist Your Project?

Our C. elegans SMA models offer a robust and versatile platform to significantly advance your drug discovery and research initiatives. We provide comprehensive solutions ranging from initial compound screening to detailed mechanistic studies, tailored to your specific project needs. You can expect precise deliverables, including validated lead compounds, clear insights into disease pathways, and identification of novel therapeutic targets and biomarkers.

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Workflow

Our streamlined workflow ensures efficient and high-quality results, guiding your project from initial concept to actionable data.

• Required Starting Materials:

To initiate a project, clients typically provide:

1. Target Genes/Pathways of Interest: Specific genetic or molecular pathways you aim to investigate or modulate.
2. Compound Libraries for Screening: Collections of small molecules or biologics for high-throughput screening.
3. Specific Research Questions: Clearly defined objectives regarding disease mechanisms, therapeutic efficacy, or target validation.

• Final Deliverables:

Upon project completion, you will receive:

1. Detailed Experimental Reports: Comprehensive documentation of all experimental procedures, results, and analyses.
2. Raw and Analyzed Data Sets: All primary and processed data in a usable format for your own analysis.
3. Identified Lead Compounds/Targets: A list of validated compounds or molecular targets with supporting efficacy and mechanistic data.

• Estimated Timeframe:

The typical timeframe for this service ranges from 8 to 16 weeks, depending on the complexity of the project, the number of compounds to be screened, and the depth of mechanistic studies required.

Why Choose Us?

Choosing Creative Biolabs for your SMA C. elegans model research offers unparalleled advantages, combining deep scientific expertise with a proven track record of accelerating drug discovery.

Key Advantages:

• Unmatched Expertise: Our team comprises scientists with over 20 years of experience in C. elegans biology, genetics, and high-throughput screening, ensuring scientific rigor and reliable data.
• Translational Relevance: We leverage the high genetic homology and conserved biological pathways between C. elegans and humans, maximizing the translatability of our findings to clinical applications.
• Comprehensive Phenotypic and Molecular Assays: Our extensive suite of assays provides robust and quantifiable readouts, from locomotion and lifespan to detailed gene expression profiling and ion channel activity.
• Custom Model Development: We specialize in creating tailored C. elegans SMA models, including patient-specific genetic mutations and advanced knockouts, to precisely address your unique research questions.
• Cost-Effective and Efficient: C. elegans models offer a significantly more economical and faster alternative to traditional vertebrate models, reducing your overall drug development costs and timelines.
• Proven Success: Our models have demonstrated the ability to predict the efficacy of neuroprotective drugs, which also shows promise in mammalian systems, validating our platform's predictive power.

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Customer Reviews:

"Invaluable Insights Using CBL's C. elegans SMA models in our research has significantly improved our understanding of SMN protein function and its impact on germline development. The detailed phenotypic assays provided invaluable insights into early developmental defects, guiding our therapeutic strategy." - 2 months ago, Dr. A***n.

"Accelerated Screening The high-throughput screening capabilities of CBL's C. elegans platform allowed us to rapidly identify novel compounds that ameliorate locomotion defects in our SMA models. Their expertise in analyzing gene expression changes, particularly in mitochondrial genes, was crucial for uncovering the mechanism of action." - 4 months ago, Dr. S***h.

"Translational Success CBL's C. elegans SMA models provided a cost-effective and highly translatable system for validating our neuroprotective drug candidates. The clear demonstration of improved neuromuscular function and the insights into SK channel modulation were pivotal for advancing our program." - 6 months ago, Dr. R***o.

SMA Caenorhabditis elegans Models

For over two decades, Caenorhabditis elegans (C. elegans), a small, free-living nematode, has emerged as an indispensable model organism in neuroscience and drug discovery. Its remarkable genetic tractability, short life cycle, transparent body, and fully mapped nervous system make it an ideal platform for studying complex human diseases. Crucially, C. elegans possesses orthologs for approximately 60-80% of human disease-associated genes, including the SMN gene. Its ease of growth and maintenance, coupled with the ability to readily obtain transgenic lines, further enhance its utility. With muscle cells and neurons making up approximately 45% of its total 959 cells, and a well-characterized nervous system wiring diagram, C. elegans offers an excellent system for developmental studies of relevant tissues.

At CBL, our expertise lies in developing and leveraging sophisticated C. elegans models that accurately recapitulate key aspects of human SMA. By manipulating the smn-1 gene through techniques such as RNA interference (RNAi) for knockdown or heat-shock inducible promoters for overexpression, we create models that display a spectrum of SMA-like phenotypes, ranging from subtle motor deficits to severe developmental abnormalities, mirroring the clinical heterogeneity observed in patients. We also utilize advanced methods like GFP reporter constructs to visualize protein expression and localization.

Fig.1 his-41p::his-41::mCherry transgene expression.¹

What We Can Offer

• Customized SMA Model Development: We design and generate bespoke C. elegans SMA models tailored to your specific research objectives, including precise genetic knockdowns (smn-1 RNAi), overexpression lines, and patient-specific mutations, ensuring the most relevant experimental system for your project.
• High-Throughput Phenotypic Screening: Leverage our advanced automated platforms for rapid and efficient screening of large compound libraries. We quantify key SMA-relevant phenotypes such as locomotion (thrashing, body bends), lifespan, brood size, and neuromuscular integrity, quickly identifying promising therapeutic candidates.
• In-Depth Mechanism of Action Studies: Uncover the precise molecular pathways and targets of your lead compounds. Our capabilities include comprehensive analysis of SMN protein levels and localization, protein-protein interactions (e.g., with fibrillarin), gene expression profiling (mitochondrial, vacuolar H+-ATPase, histone genes), and assessment of ion channel modulation (e.g., SK channels).
• Target Validation and Biomarker Discovery: Validate novel therapeutic targets identified through screening and elucidate their role in SMA pathology. We also identify and confirm new molecular biomarkers for disease progression and treatment response, enhancing clinical translatability.
• Preclinical Efficacy Assessment: Evaluate the in vivo efficacy of your compounds in a living organism. Our models provide robust data on improvements in neuromuscular function and axon outgrowth, offering critical insights before advancing to more complex and costly vertebrate models.
• Expert Consultation and Data Interpretation: Benefit from our team's over two decades of specialized expertise in C. elegans biology and SMA research. We provide comprehensive data analysis, clear interpretations, and strategic recommendations to guide your drug discovery pipeline.
• Integrated Solutions: Our services seamlessly integrate with your existing research, providing a one-stop solution from model generation to detailed mechanistic insights, all designed to accelerate your drug development timeline and reduce costs.

Related Services

To further support your drug discovery and development efforts, CBL offers a range of complementary services:

• Neuroinflammation Assays
• Neurodegenerative Disease Models
• Biomarker Discovery & Validation
• Histology & Pathology Services

Ready to advance your SMA research? Our team of experts is here to discuss your specific project needs and demonstrate how our C. elegans SMA Models can provide the solutions you require.

Contact Our Team for More Information and to Discuss Your Project.

Reference

1. Gao, Xiaoyang et al. "Defective Expression of Mitochondrial, Vacuolar H+-ATPase and Histone Genes in a C. elegans Model of SMA." Frontiers in genetics vol. 10 410. 3 May. 2019, doi:10.3389/fgene.2019.00410. Distributed under Open Access license CC BY 4.0, without modification.