In Vitro Efficacy Testing Services for Spinal Muscular Atrophy
We provide robust and sensitive in vitro screening and characterization platforms for accelerating the discovery and screening of potential therapies for Spinal Muscular Atrophy (SMA). Our services enable detailed evaluation of candidate compounds and their effects on molecular targets relevant to SMA. Key targets include the survival motor neuron (SMN) protein, as well as associated cellular pathways involved in motor neuron survival and neuromuscular function. We assess pathological processes such as SMN protein expression, cellular viability, and neurodegeneration, supporting comprehensive therapeutic assessment.
Our testing platform offers a range of biochemical and cell-based assays tailored to evaluate potential SMA therapies. These methods are designed to quantify target engagement, enzymatic activity, and biomarker changes, providing actionable data to guide lead optimization and selection.
Arachidonic acid as substrate: Utilized in enzymatic assays to assess the activity of enzymes implicated in inflammatory or neurodegenerative processes relevant to SMA.
Chemiluminescent assay: Employs light emission for sensitive detection of specific proteins or enzymatic activities, enhancing assay sensitivity for key SMA biomarkers.
ELISA assay: Enables quantitative measurement of proteins such as SMN, facilitating the evaluation of drug effects on target protein expression.
Enzyme immunoassay (EIA): Measures the concentration of specific antigens or antibodies, supporting the analysis of molecular changes in SMA models.
Fluorescent assay: Uses fluorescence-based readouts to monitor cellular or molecular events, allowing for high-throughput screening and precise quantitation.
We focus on quantifying key pharmacological parameters that reflect drug potency and efficacy. These parameters are essential for comparing candidate compounds and guiding decisions in the drug development pipeline.
IC-50: The concentration of a compound required to inhibit a specific biological or enzymatic activity by 50%, serving as a critical indicator of compound potency and suitability for further development.
Prostaglandin-Endoperoxide Synthase 2 (PTGS2/COX-2) is implicated in neuroinflammation associated with Spinal Muscular Atrophy (SMA) progression. Testing PTGS2 activity is crucial for evaluating anti-inflammatory drug efficacy in SMA drug development. Our service utilizes enzyme immunoassay (EIA), ELISA, fluorescent, and chemiluminescent assays, employing arachidonic acid as a substrate. The main parameter measured is IC-50, enabling precise assessment of compound potency in modulating PTGS2 activity.
| Pharmacological Activity | Material | Method | Parameter |
|---|---|---|---|
| Cyclooxygenase 2 [COX 2], inhibition | Blood, human | ELISA assay | IC-50 |
| Cyclooxygenase 2 [COX 2], inhibition | Human enzyme | IC-50 | |
| Cyclooxygenase 2 [COX 2], inhibition | Recombinant enzyme | IC-50 | |
| Cyclooxygenase 2 [COX 2], inhibition | Recombinant human enzyme | Arachidonic acid as substrate | IC-50 |
| Cyclooxygenase 2 [COX 2], inhibition | Recombinant human enzyme | ELISA assay | IC-50 |
| Cyclooxygenase 2 [COX 2], inhibition | Recombinant human enzyme | Enzyme immunoassay (EIA) | IC-50 |
| Cyclooxygenase 2 [COX 2], inhibition | Recombinant human enzyme | Fluorescent assay | IC-50 |
| Cyclooxygenase 2 [COX 2], inhibition | Arachidonic acid as substrate | IC-50 | |
| Cyclooxygenase 2 [COX 2], inhibition | Chemiluminescent assay | IC-50 | |
| Cyclooxygenase 2 [COX 2], inhibition | Enzyme immunoassay (EIA) | IC-50 | |
| Cyclooxygenase 2 [COX 2], inhibition | Fluorescent assay | IC-50 | |
| Cyclooxygenase 2 [COX 2], inhibition | IC-50 |
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