SMA High-Throughput Phenotypic Screening Assay

Spinal Muscular Atrophy (SMA) is a genetic disorder that affects the motor neurons in the spinal cord and brainstem, leading to muscle weakness and atrophy. There is no known cure for SMA, but early diagnosis and treatment can improve the quality of life for affected individuals. Creative Biolabs has developed a High-Throughput Phenotypic Screening Assay to aid in the discovery of new therapies for SMA.

SMA Patient iPSC-Derived Motor Neurons

To develop an accurate and reliable assay for SMA, it is important to use a relevant cellular model. Patient-derived induced pluripotent stem cells (iPSCs) offer a promising approach, as they can be differentiated into a variety of cell types, including motor neurons. These motor neurons can then be used to screen potential therapies for SMA.

Fig. 1 General Protocol for Neuron Production.

Characterization of Motor Neurons

Creative Biolabs utilizes a range of highly specialized techniques to accurately identify and characterize motor neurons. These techniques include immunofluorescence staining, electrophysiology, calcium imaging, MEA, and molecular profiling. These advanced technologies allow us to evaluate various aspects of motor neuron function and physiology, including morphology, neurotransmitter release, and gene expression.

Assay Development and Optimization

Our team of experts utilizes state-of-the-art technologies and platforms to develop and optimize high-throughput phenotypic screening assays for SMA. We specialize in the development of assays that enable screening of a large number of compounds in a relatively short time frame, facilitating the identification of novel compounds with therapeutic potential.

We also offer assay optimization services to enhance the sensitivity, specificity, and robustness of our clients' SMA assays. Our team utilizes advanced statistical approaches and assay optimization strategies to identify and address potential limitations or issues, such as variability, false positives, or low signal-to-noise ratios.

Quantitative HTS (qHTS) Screen

To increase the efficiency and accuracy of the SMA screening assay, Creative Biolabs uses a Quantitative High-Throughput Screening (qHTS) approach. This approach involves testing a large number of compounds simultaneously, using a range of concentrations, to identify those that are most effective at improving the motor neuron phenotype. The qHTS approach enables the identification of compounds that may not have been identified through traditional screening methods.

Fig.2 qHTS analysis workflow.

Customized Assay Development

Creative Biolabs offers customized assay development to meet the specific needs of individual research projects. This ensures that the most appropriate assays are used to screen for compounds that are most relevant to the specific research question. With our expertise, customization, efficiency, and quality control, we are committed to providing high-quality service to our clients and advancing the field of SMA drug discovery. Get in touch with us today to find out more about our SMA assay services.