Patient-Derived Cell Model Development
At Protheragen, we focus on developing advanced patient-derived cell models for accelerating breakthroughs in rare neurological diseases. These models include ethically-sourced iPSC-derived neurons, glial cells, and primary cell cultures. All models undergo thorough validation for disease-specific phenotypes, drug screening, and mechanistic studies.
Overview of Patient-Derived Cell Models
Patient-derived cell models are in vitro systems generated directly from individuals suffering from rare movement and neurological disorders, and as such, they capture both the genetic and epigenetic signatures of the disease. These models chiefly employ:
Induced Pluripotent Stem Cells (iPSCs)
iPSCs are derived from reprogramming patient fibroblasts or peripheral blood mononuclear cells (PBMCs) into a pluripotent form utilizing Yamanaka factors (e.g. OCT4, SOX2, KLF4, and c-MYC), after which they are differentiated into neural subtypes associated with specific diseases.
Primary Cell Cultures
Primary cultures, resulting from patient biopsies and post-mortem tissues, provide cellular environments untouched by reprogramming artifacts. Although sample availability may be a limitation, these cultures offer crucial validation for iPSC models, especially for ultra-rare diseases where donor tissues are extremely limited.
Fig.1 Examples of neuronal models derived from patient iPSCs. (Tang B L., 2018)
Applications of Patient-Derived Cell Models
Target Discovery and Validation
The patient-derived cellular models can identify new therapeutic targets by recapitulating disease-specific molecular pathways which provides physiologically relevant systems for mechanistic research.
Preclinical Toxicity and Efficacy Assessment
Patient-derived systems offer predictive assessment of drug safety profiles and treatment responses specific to genetic backgrounds, improving clinical translation success rates for rare neurological disorders.
Drug Screening and Repurposing
Additionally, these models are potent platforms for performing high-throughput screening, enabling drug repurposing and evaluation of potential therapeutics against the backdrop of actual human diseases.
Companion Diagnostic Development
The models help devise biomarker assays and functional readouts capable of stratifying patient populations, tracking therapeutic outcomes, and thus, enabling personalized approaches to medicine.
Our Services
Focused on providing personalized platforms for diagnostic innovation and therapeutic development, Protheragen specializes in creating complex patient-derived cell models for rare motor neuron diseases and rare neurological disorders. Our models support all aspects of preclinical research which includes: identification and validation of therapeutic targets, drug screening, and pharmacodynamic, pharmacokinetic, and toxicology evaluations. We strive to expedite the creation of targeted therapies for these difficult disorders by using patient-specific iPSC-derived neurons and glial cells.
iPSC Model Development Service
Patient Sample Collection
- Skin fibroblasts
- Peripheral blood mononuclear cells (PBMCs)
Reprogramming & iPSC Generation
- Non-integrating episomal vectors
- Sendai virus
- Quality control (QC)
Neuronal/Glial Differentiation
- Motor neurons
- Dopaminergic neurons
- Oligodendrocytes
- More
Genetic Engineering (Optional)
- Gene Editing Technology
- Knock-in disease mutations
- Isogenic controls
Phenotypic & Functional Characterization
- Electrophysiology
- Disease-Relevant Assays
- Multi-Omics Profiling
Primary Cell Model Development Service
Protheragen focuses on the isolation, characterization, and culture of patient-derived primary neural cells to study rare neurological diseases. Our services ensure that native cell properties are preserved by employing tailored dissociation protocols, media formulations that improve cell viability, and functional validation.
- Central Nervous System (CNS) Primary Cell Models: Cortical neurons, hippocampal neurons, dopaminergic neurons, spinal motor neurons, etc.
- Peripheral Nervous System (PNS) Primary Cell Models: Dorsal root ganglion (DRG) sensory neurons, Schwann cells, sympathetic ganglion neurons, etc.
- Glial Cell Models: Astrocytes, microglia, oligodendrocytes, etc.
Protheragen provides rare neurological disease research with patient-derived cell models of unparalleled biological fidelity, and is ethically sourced and fully compliant. Our company takes great care to ensure each model undergoes rigorous quality control supported by GLP-grade documentation, yielding clinically relevant, publishable results. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
Reference
- Tang B L. Patient-Derived iPSCs and iNs—Shedding New Light on the Cellular Etiology of Neurodegenerative Diseases[J]. Cells, 2018, 7(5): 38.