Spinal Muscular Atrophy (SMA) Zebrafish Model Development Service

Are you currently facing long drug development cycles and challenges in identifying effective therapeutic compounds for neurodegenerative disorders? Our Spinal muscular atrophy (SMA) zebrafish models help you accelerate drug discovery and obtain high-quality preclinical data through advanced genetic engineering and high-throughput screening platforms.

SMA is a severe genetic neuromuscular disorder caused by insufficient Survival Motor Neuron (SMN) protein. Zebrafish (Danio rerio) have emerged as a powerful and highly translational model for SMA research. Their genetic homology to humans, rapid external development, and optical transparency enable efficient high-throughput screening and detailed mechanistic studies. Cited literature confirms their utility in recapitulating human SMA pathologies and accelerating the identification of therapeutic targets, making them indispensable for preclinical drug discovery.

How Our Spinal Muscular Atrophy (SMA) Zebrafish Models Can Assist Your Project?

At Creative Biolabs, we provide comprehensive solutions leveraging our cutting-edge SMA zebrafish models to streamline your drug discovery and validation efforts. Clients can expect precise phenotypic characterization, efficient compound screening, and robust data for lead candidate selection, significantly reducing time and cost in preclinical development.

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Workflow: From Concept to Breakthrough

Our streamlined workflow ensures a clear, professional, and efficient path from project initiation to final deliverables, designed for optimal visualization as a flowchart.

Required Starting Materials:

To initiate your project, we typically require:

Target Genes or Pathways of Interest: Specific genetic information or molecular pathways you aim to modulate.
Compound Libraries: Your proprietary small molecule libraries or a defined set of compounds for screening.
Specific Research Questions/Hypotheses: Clear objectives for the study, including desired therapeutic outcomes.

Fig.1 Workflow of our Spinal Muscular Atrophy (SMA) Zebrafish Models Development Services Can Assist Your Project. (Creative Biolabs Original)

Final Deliverables:

Upon project completion, you will receive:

Detailed Study Reports: Comprehensive documentation of methodologies, results, and conclusions.
Raw and Analyzed Data: All raw data files, along with statistically analyzed datasets and graphical representations.
Validated Lead Compound Profiles: Profiles of identified lead compounds, including efficacy, preliminary mechanism of action, and recommendations for further development.

Estimated Timeframe:

The typical timeframe for this service ranges from 8 to 16 weeks, depending on the complexity of the model generation, the size of the compound library, and the scope of mechanistic studies. Factors such as the novelty of the target, the number of compounds, and specific assay requirements can influence the overall duration.

Why Choose Us?

CBL stands as a leader in leveraging SMA zebrafish models for drug discovery, offering unparalleled expertise and a proven track record. Our commitment to scientific rigor, efficiency, and client success sets us apart.

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Customer Reviews: Real Impact, Real Solutions

"Enhanced Screening Efficiency Using CBL's SMA zebrafish models in our research has significantly improved our high-throughput screening efficiency, allowing us to test more compounds in less time. The rapid phenotype development truly speeds up the process." [March 2025], Dr. L***a S.

"Precise Phenotypic Data The quantifiable motor function data provided by CBL's SMA zebrafish models is exceptionally precise, facilitating our understanding of compound efficacy and mechanism of action. This level of detail is crucial for our lead optimization." [January 2025], Prof. M***n J.

"Cost-Effective and Ethical We chose CBL's zebrafish platform for its cost-effectiveness and ethical advantages over traditional mammalian models. The ability to visualize motor neuron changes in real-time without invasive procedures has been a game-changer for our SMA research." [November 2024], Dr. R***d K.

Spinal Muscular Atrophy (SMA) Zebrafish Models

The zebrafish (Danio rerio) has rapidly become a cornerstone in biomedical research, particularly for neurological and genetic disorders. For SMA research, its unique biological attributes offer distinct advantages over traditional mammalian models.

Key Phenotypes and Pathologies Mimicked

Our SMA zebrafish models meticulously replicate the core pathological hallmarks observed in human patients, providing a robust system for studying disease mechanisms and evaluating therapeutic interventions:

Phenotypes and Pathologies	Description
Motor Neuron Degeneration	A hallmark of SMA, the progressive loss and dysfunction of motor neurons are clearly observable in our zebrafish models, often leading to reduced axonal outgrowth and impaired neuromuscular junction formation. Studies have shown that even models with intermediate Smn expression can develop significant neuromuscular junction deficiencies, aligning with the pathology of the disease in humans where SMN protein deficiency leads to the death of lower motor neurons.
Locomotion Deficits	SMA zebrafish larvae exhibit quantifiable reductions in swimming activity, tail coiling, and overall motor function, providing easily measurable phenotypic readouts for drug efficacy. These deficits are a consistent feature in models mimicking intermediate SMA, reflecting the muscle weakness and limited mobility seen in patients.
Muscle Atrophy	Similar to human patients, our models demonstrate muscle fiber abnormalities and atrophy, reflecting the downstream effects of motor neuron loss. This muscular atrophy is a key recapitulated aspect, even in models with intermediate disease severity, and contributes to the overall pathology including spinal curvature.
SMN Protein Deficiency and Splicing Defects	Zebrafish models can be engineered to mimic the reduced SMN protein levels and aberrant SMN2 splicing characteristic of human SMA, making them ideal for testing SMN-enhancing therapies.
Systemic Manifestations	Beyond the neuromuscular system, SMA can impact other organs and functions, such as feeding, breathing, and eye movement. Zebrafish models allow for the investigation of broader systemic effects and the potential for multi-target therapies.

Applications in Drug Discovery and Therapeutic Development

The versatility of SMA zebrafish models makes them indispensable across the entire drug discovery pipeline:

Applications	Description
High-Throughput Small Molecule Screening	Rapidly identify novel compounds that restore SMN protein levels, improve motor neuron survival, or enhance motor function. The development of models faithfully recapitulating intermediate SMA phenotypes provides a valuable resource for testing new drugs specifically targeted towards these forms, including large-scale screening for drug repositioning. Pharmacological models are particularly useful for this application.
Validation of Gene Therapies	Evaluate the efficacy and safety of gene replacement therapies (e.g., AAV-mediated delivery of SMN1) and gene editing approaches in a living organism. Transgenic SMA zebrafish models are especially valuable for assessing later-stage SMA phenotypes in the context of gene therapy.
Antisense Oligonucleotide (ASO) Testing	Assess the ability of ASOs to modulate SMN2 splicing and increase functional SMN protein, mimicking the mechanism of action of approved SMA drugs. Transient morphant models can be particularly useful for rapid evaluation of ASO efficacy.
Mechanistic Studies	Delve deeper into the molecular and cellular pathways underlying SMA pathogenesis, identifying new therapeutic targets (such as U snRNPs and PLS3) and understanding disease progression.
Biomarker Identification	Discover and validate novel biomarkers that can be used to monitor disease progression and therapeutic response in preclinical and clinical settings.

What We Can Offer

At CBL, our SMA zebrafish models service is designed to provide comprehensive, high-quality, and customizable solutions for your drug discovery needs. We offer:

Customized SMA Zebrafish Model Development: Tailored generation of stable transgenic and transient morphant SMA zebrafish models, incorporating specific human-relevant mutations (e.g., smn A6T) and varying disease severities to perfectly match your research objectives.
High-Throughput Screening (HTS) Capabilities: Access to advanced automated platforms for large-scale screening of small molecules, gene therapies, and antisense oligonucleotides (ASOs), enabling rapid identification of therapeutic candidates with results often available within 48 hours for initial screens.
Quantifiable Phenotypic Assays: Robust and precise assays for evaluating motor neuron integrity, axonal outgrowth, neuromuscular junction function, and quantifiable locomotion deficits, providing clear and reliable readouts of therapeutic efficacy.
In-Depth Mechanistic Elucidation: Comprehensive studies to uncover the precise mechanisms of action of lead compounds and identify novel therapeutic targets (e.g., U snRNPs, PLS3), guiding rational drug design and optimization.
Biomarker Identification and Validation: Expertise in discovering and validating novel biomarkers for monitoring disease progression and therapeutic response, crucial for preclinical and clinical translation.
Rigorous Quality Control and Data Reporting: A well-established quality system ensuring meticulous screening, validation, and comprehensive documentation, delivering detailed study reports, raw and analyzed data, and validated lead compound profiles.
End-to-End Project Support: A one-stop service from initial consultation and experimental design to final data analysis and strategic recommendations, ensuring a seamless and efficient drug discovery process.

Related Services

To further support your research and development goals, CBL offers a suite of complementary services designed to integrate seamlessly with our SMA zebrafish models platform:

Ready to advance your SMA drug discovery project? Our expert team is eager to discuss your specific requirements and demonstrate how our SMA zebrafish models can empower your next breakthrough.

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

For Research Use Only. Not For Clinical Use.

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