SMA High-Throughput Phenotypic Screening Assay Service

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by SMN1 gene mutations. Drive your SMA drug discovery forward. Our in vitro high-throughput phenotypic screening service combines cutting-edge technology with tailored solutions to expedite the development of targeted therapies. From assay design to data analysis, we provide comprehensive support for your research. For further information regarding the products and services provided, project-specific consultation, and pricing, please submit an inquiry here.

Patient-Specific iPSC-Derived Motor Neuron Models

We leverage patient-specific induced pluripotent stem cells (iPSCs) to generate motor neurons that faithfully recapitulate the pathological hallmarks of SMA. This robust in vitro model allows for comprehensive assessment of compound efficacy and safety, including quantification of SMN protein levels, evaluation of neuronal survival rates, and functional characterization through axon growth analysis and electrophysiological recordings.

Fig.1 SMA patient iPSC-derived motor neuron.¹

High-Throughput Phenotypic Screening Assays

Leveraging cutting-edge technology, Creative Biolabs delivers robust high-throughput phenotypic screening services for SMA research. Our platform encompasses fluorescence, luminescence, and colorimetric detection, supporting both array- and pool-based screening (FACS, NGS). Integrated cell immunophenotyping provides in-depth cellular analysis, enabling a multifaceted approach to discovery.

• High Content Screening (HCS) Platform

Accelerate SMA Drug Discovery with Our Advanced High Content Screening Platform. Our automated imaging and AI-powered analysis system enables simultaneous detection of multi-parametric phenotypes, including nuclear morphology, cell apoptosis, and SMN expression. Screen thousands of compounds in a single experiment and dramatically enhance your drug discovery efficiency.

• Joint Biomarker Validation

We combine candidate biomarkers, such as neurofilament protein (NF), with SMN2 splicing regulation analysis. This multidimensional approach provides robust efficacy evaluation and facilitates seamless preclinical data translation.

• Accurate Quantification of SMN Copy Number

Precise SMN Copy Number Determination for SMA Research. Utilizing qPCR and melting curve technology, we deliver highly sensitive detection of SMN1/SMN2 gene copy number variations. This accurate quantification provides a critical foundation for sample stratification and optimized program design.

Applications

Efficient Drug Discovery: Streamline your drug screening process to quickly pinpoint small molecules, siRNA, and gene therapy candidates that boost SMN protein production and neuronal health.

Comprehensive Mechanistic Studies: Delve into the intricacies of SMN2 splicing regulation, explore neuroprotective pathways, and validate potential drug targets.

Translational Preclinical Validation: Bridge the gap between in vitro findings and clinical success by predicting patient responses and refining clinical trial strategies.

We combine cutting-edge disease models, comprehensive multi-omics analysis, and automated high-throughput platforms to dramatically accelerate your research. Injecting robust scientific power into the fight against SMA, our services are designed to streamline your drug discovery process.

Ready to customize a screening plan tailored to your specific needs? Contact our expert team today and let us help you pave the way for groundbreaking SMA therapies.

Reference

1. Fuller, Heidi R., et al. "Spinal muscular atrophy patient iPSC-derived motor neurons have reduced expression of proteins important in neuronal development." Frontiers in cellular neuroscience 9 (2016): 506. Distributed under Open Access license CC BY 4.0, without modification.