Zebrafish Disease Research

Zebrafish are playing an increasingly pivotal role in human disease modeling. These models enable scientists to replicate, observe, and manipulate human disease mechanisms in a cost-effective and genetically accessible system. The examples below illustrate how zebrafish are used to understand complex diseases and support drug discovery, positioning them as powerful tools in translational medicine.

Case Studies: Zebrafish in Disease Modeling and Therapeutic Research

The following zebrafish disease research articles demonstrate how zebrafish disease models contribute to our understanding of neurological, cardiovascular, oncological, and ophthalmological disorders. These models provide critical insights into disease progression, genetic pathways, and therapeutic screening, helping bridge the gap between bench and bedside.

A review of MPTP-induced parkinsonism in adult zebrafish to explore pharmacological interventions for human Parkinson’s disease

Therapeutic Area: Neurology

Disease/Application: Parkinson’s Disease

Impact of Zebrafish on Understanding & Modeling Diseases:
-Degeneration of dopaminergic neurons
-Mechanisms of neuroprotection

Zebrafish pivotal role in drug discovery: Identifying pathological mechanisms, and evaluating new treatments efficiently (neuroprotectors).

Studying zebrafish nervous system structure and function in health and disease with electron microscopy

Therapeutic Area: Neurology

Disease/Application: Alzheimer’s Disease

Impact of Zebrafish on Understanding & Modeling Diseases:
-Buildup of amyloid-beta plaques
-Mechanisms of neurodegeneration.

Zebrafish pivotal role in drug discovery: Revealing the impact of certain compounds on the reduction of amyloid-beta plaque formation.

ZebraReg-a novel platform for discovering regulators of cardiac regeneration using zebrafish

Therapeutic Area: Cardiology

Disease/Application: Myocardial Infarction

Impact of Zebrafish on Understanding & Modeling Diseases: Key pathways involved in heart regeneration.

Zebrafish pivotal role in drug discovery:
-Identifying potential drug targets for treating myocardial infarction in humans (target ID)
-Screening of potential therapeutic interventions.

Zebrafish Congenital Heart Disease Models: Opportunities and Challenges

Therapeutic Area: Cardiology

Disease/Application: Congenital Heart Disease

Impact of Zebrafish on Understanding & Modeling Diseases: Mechanisms (mutations) of heart malformations.

Zebrafish pivotal role in drug discovery:
-Identifying several genetic mutations associated with genetic heart disease (target ID)
-Screening of potential therapeutic interventions

Mutations of TTN, encoding the giant muscle filament titin, cause familial dilated cardiomyopathy

Therapeutic Area: Cardiology

Disease/Application: Dilated Cardiomyopathy

Impact of Zebrafish on Understanding & Modeling Diseases: Mechanisms (mutations) of cardiomyopathies.

Zebrafish pivotal role in drug discovery:
-Identifying potential drug targets for treating dilated cardiomyopathy in humans (target ID)
-Screening of potential therapeutic interventions.

Zebrafish MITF-Low Melanoma Subtype Models Reveal Transcriptional Subclusters and MITF-Independent Residual Disease

Therapeutic Area: Oncology

Disease/Application: Melanoma

Impact of Zebrafish on Understanding & Modeling Diseases:
-Tumor growth studies
-Metastasis studies
-Track cancer progression in real time
-Study cancers with poor mammalian model relevance, such as melanoma & pancreatic cancer

Zebrafish pivotal role in drug discovery: Testing the efficacy of targeted therapies for melanoma & other drug screenings.

Zebrafish: Speeding Up the Cancer Drug Discovery Process

Therapeutic Area: Oncology

Disease/Application: Personalized Medicine/ Precision Medicine (Zebrafish ‘avatars’ are gaining traction as a tool to guide treatment plans for a particular patient with cancers)

Impact of Zebrafish on Understanding & Modeling Diseases: Zebrafish serve as exceptional tools for creating human cancer models, enabling the engineering of patient-specific mutations through the development of mutant and transgenic lines, as well as the transplantation of tumor cells.

Zebrafish pivotal role in drug discovery: Zebrafish avatars can be especially useful for:
-Determining dose and treatment length interventions
-Validating potential disease-causing alleles
-Looking for therapies that might reverse the disease once the phenotype is established.

Zebrafish Model in Ophthalmology to Study Disease Mechanism and Drug Discovery

Therapeutic Area: Ophthalmology

Disease/Application:
-Corneal Dystrophy
-Cataract
-Glaucoma
-Vascular disease
-Diabetic Retinopathy
-Age-related Macular Degeneration
-Photoreceptor Degeneration (Retinitis Pigmentosa, Leber Congenital Amaurosis)

Impact of Zebrafish on Understanding & Modeling Diseases: With genetic accessibility, similar characteristics of human ocular development, and easy controllability of living environment, zebrafish have been used as a popular model to study eye disorders.

Zebrafish pivotal role in drug discovery:
Screening of potential therapeutic interventions:
-Anti-Angiogenic Compounds
-Neuroprotective Drugs
-Drug Oculotoxicity