In Vitro Efficacy Testing Services for Leber Hereditary Optic Neuropathy

We provide robust and sensitive in vitro screening and characterization platforms for accelerating the discovery and screening of potential therapies for Leber hereditary optic neuropathy (LHON). Our service offers disease-relevant cellular and biochemical assays tailored to assess mitochondrial function, oxidative stress, and neuroprotective effects of candidate compounds for LHON. Key targets include mitochondrial complex I, reactive oxygen species, and apoptosis pathways, which are central to LHON pathogenesis. We are equipped to evaluate pathological processes such as mitochondrial dysfunction, oxidative damage, and cell viability associated with LHON.

Our testing portfolio encompasses a diverse range of in vitro methods designed to evaluate the efficacy and mechanism of potential LHON therapies. These assays provide quantitative and qualitative insights into bioactivity, mitochondrial health, and relevant signaling pathways. The overall purpose is to enable rapid, reliable assessment of therapeutic candidates in models relevant to LHON.

Arachidonic acid as substrate: This assay uses arachidonic acid to model oxidative stress and mitochondrial dysfunction, enabling evaluation of compound effects on these LHON-relevant processes.

Chemiluminescent assay: This sensitive method measures reactive oxygen species and other luminescent biomarkers, providing insight into oxidative damage and mitochondrial activity.

ELISA assay: Enzyme-linked immunosorbent assays quantify specific proteins or signaling molecules involved in LHON, such as markers of apoptosis, inflammation, or mitochondrial health.

Fluorescent assay: Fluorescent probes and dyes are used to detect changes in mitochondrial function, cell viability, and oxidative status with high sensitivity and throughput.

RNA assay: Quantitative RNA assays assess gene expression changes in key pathways implicated in LHON, supporting mechanistic studies and target validation.

We measure multiple pharmacological parameters that are critical for evaluating the potency, efficacy, and safety of potential LHON therapies. These parameters help define the therapeutic window, establish dose-response relationships, and inform lead optimization. Accurate parameter measurement is essential for effective preclinical decision-making.

IC-50: The half maximal inhibitory concentration indicates the potency of a compound in inhibiting a specific biological process, providing a benchmark for comparing candidate molecules.

MEC: The minimum effective concentration is the lowest dose at which a compound demonstrates the desired biological activity, guiding dose selection for further development.

MED: The minimum effective dose identifies the smallest amount of a compound that produces a measurable therapeutic effect, supporting clinical translation.

MIC: The minimum inhibitory concentration measures the lowest concentration required to inhibit target activity or cellular processes, important for efficacy and safety profiling.

Recommended In Vitro Efficacy Tests

Cyclin D1

Cyclin D1 regulates cell cycle progression and is implicated in retinal ganglion cell survival in Leber hereditary optic neuropathy (LHON). Cyclin D1 testing is vital for assessing drug effects on cellular proliferation and neuroprotection in LHON models. Key methods include immunohistochemistry, Western blot, and qPCR. Main parameters measured are Cyclin D1 expression levels, localization, and correlation with cellular health outcomes, providing critical data for LHON drug development.

Dna Methyltransferase 1

DNA Methyltransferase 1 (DNMT1) regulates epigenetic modifications implicated in Leber hereditary optic neuropathy (LHON) pathogenesis. DNMT1 testing is vital for identifying drug candidates that modulate its activity, potentially improving LHON outcomes. Our service uses chemiluminescent, fluorescent, and RNA assays to assess DNMT1 function. Key parameters measured include MIC and IC-50, enabling precise evaluation of drug efficacy and potency during LHON drug development.

Epidermal Growth Factor Receptor

Our Epidermal Growth Factor Receptor (EGFR) testing service supports Leber hereditary optic neuropathy (LHON) drug development by assessing EGFR's role in disease pathogenesis and therapeutic response. Accurate EGFR quantification, using chemiluminescent and RNA assays, enables targeted therapy optimization. Key parameters, such as Minimum Inhibitory Concentration (MIC), ensure drug efficacy and safety profiling, facilitating precise and efficient LHON drug candidate evaluation.

Intercellular Adhesion Molecule 1

Intercellular Adhesion Molecule 1 (ICAM-1) is implicated in the inflammatory response and retinal ganglion cell damage in Leber hereditary optic neuropathy (LHON). Testing ICAM-1 expression is crucial for evaluating drug efficacy and mechanism in LHON therapies. Our service offers quantitative analysis using fluorescent and RNA assays, with Minimum Inhibitory Concentration (MIC) as a key parameter, providing essential insights for targeted drug development and candidate screening.

Prostaglandin-Endoperoxide Synthase 1

Prostaglandin-Endoperoxide Synthase 1 (PTGS1/COX-1) may modulate inflammation in Leber hereditary optic neuropathy (LHON). Assessing PTGS1 activity is vital for evaluating anti-inflammatory drug candidates for LHON. Our testing service employs ELISA assays to quantify PTGS1, determining drug efficacy by measuring IC-50 values. This enables precise screening of compounds for their potential to modulate PTGS1 and impact LHON pathogenesis.

Prostaglandin-Endoperoxide Synthase 2

Prostaglandin-Endoperoxide Synthase 2 (PTGS2/COX-2) is implicated in the inflammatory pathways contributing to Leber hereditary optic neuropathy (LHON) progression. Testing PTGS2 activity is vital for evaluating potential therapeutics. Using arachidonic acid as substrate, key methods include ELISA, RNA, and chemiluminescent assays. Main parameters measured are MIC, MEC, MED, and IC-50, providing critical data on drug efficacy and potency in modulating PTGS2 relevant to LHON drug development.