In Vivo Model Development for Retinitis Pigmentosa

Protheragen offers a comprehensive in vivo animal model development service dedicated to advancing retinitis pigmentosa (RP) research. Our portfolio encompasses a diverse array of established and custom-developed animal models, enabling the rigorous evaluation of therapeutic interventions targeting this complex group of inherited retinal degenerative disorders. With a commitment to scientific excellence and translational relevance, Protheragen supports clients throughout the entire preclinical pipeline, from model selection and validation to endpoint analysis and data interpretation.

Retinitis pigmentosa is a genetically and phenotypically heterogeneous group of retinal diseases leading to progressive vision loss. Animal models are indispensable tools for unraveling disease mechanisms and assessing the efficacy and safety of novel therapies. At Protheragen, we utilize a broad spectrum of species—including mice (Mus musculus, with strains such as C57BL/6, Balb/c, Rd1, Rd10, and others), rats (Rattus norvegicus), dogs (Canis familiaris), zebrafish (Danio rerio), rabbits (Oryctolagus cuniculus), and pigs (Sus scrofa)—to recapitulate the genetic, biochemical, and pathological hallmarks of human RP. Our models feature diverse etiologies, from genetic mutations (e.g., RHO, PDE6A/B, CNGB1, PRPF31) and transgenic lines to chemically- and light-induced degeneration, ensuring high translational value and relevance for both mechanistic studies and therapeutic development.

Genetic Models

Genetic models are established by introducing or mimicking mutations found in human RP patients, such as knockouts, knockins, or transgenic expression of disease-associated genes (e.g., RHO, PDE6A, PDE6B, CNGB1, PRPF31, Mertk, Gnat2, Fam161a). These models are developed in various species, including mice, rats, dogs, rabbits, and pigs. Methodologically, genetic alterations are achieved via targeted gene editing (CRISPR/Cas9, homologous recombination), transgenic technologies, or selective breeding. Advantages include faithful recapitulation of human RP genotypes and phenotypes, stable inheritance for longitudinal studies, and suitability for studying gene-specific pathogenesis and gene therapy. These models are primarily used for mechanistic investigations, gene therapy evaluation, pharmacological testing, and biomarker discovery.

Chemically-Induced Models

Chemically-induced models employ retinal toxic agents, such as sodium iodate (NaIO3), to selectively damage photoreceptor or retinal pigment epithelial cells, thereby inducing retinal degeneration that mimics aspects of RP. Typically performed in mice (e.g., C57BL/6) or rats, the methodology involves systemic or local administration of the chemical agent, followed by monitoring of retinal structure and function. Key advantages are the rapid induction of retinal degeneration, reproducibility, and controllability of the extent of damage. These models are ideal for rapid screening of neuroprotective compounds, evaluating anti-apoptotic strategies, and studying acute retinal injury responses.

Light-Induced Models

Light-induced models leverage controlled exposure to intense light to trigger photoreceptor degeneration, simulating environmental or acquired retinal damage. This methodology is applied in species such as mice (Balb/c, Rd10) and dogs, with protocols involving defined light intensity and duration. Advantages include non-invasive induction, temporal control, and the ability to study both acute and chronic degeneration. These models are valuable for investigating photoreceptor cell death pathways, testing neuroprotective agents, and modeling environmental risk factors in RP.

Transgenic Models

Transgenic models are generated by introducing exogenous genes or reporter constructs (e.g., RHO, CX3CR1, EGFP) under specific promoters to study gene function, disease progression, or therapeutic efficacy in vivo. These models are available in mice, zebrafish, rabbits, and pigs. The methodology involves microinjection or viral transduction of genetic constructs into embryos or germ cells. Advantages include targeted gene expression, real-time monitoring of disease progression, and suitability for cell-type-specific studies. Applications include gene therapy validation, cell tracking, and functional genomics.

Protheragen delivers a full-spectrum solution for in vivo retinitis pigmentosa model development and application. Our services include model selection and customization, animal husbandry, induction protocols, therapeutic administration, and comprehensive endpoint analysis. Key efficacy endpoints encompass retinal morphology (histology, OCT), photoreceptor survival, visual function (ERG, optomotor response), molecular and cellular biomarkers (immunohistochemistry, qPCR, Western blot), and behavioral assessments. Advanced imaging, genotyping, and bioanalytical platforms support in-depth data generation. Rigorous quality control measures—such as standardized protocols, validated assays, and routine health monitoring—ensure reproducibility and scientific integrity at every stage.

Partnering with Protheragen provides access to a robust, scientifically validated suite of in vivo retinitis pigmentosa models and expert-driven preclinical services. Our collaborative approach, technical expertise, and commitment to quality empower clients to accelerate therapeutic discovery and development with confidence. Contact us today to discuss your research needs and discover how Protheragen can help bring your RP therapies closer to clinical success.