As a leading provider of custom mouse and rat models, Cyagen aims to support the advancement of rare disease and related gene therapy research with our expertise. We are committed to enabling development of therapeutics for rare diseases by developing accessible animal models to study disease mechanisms, target validation, drug screening and more.
Cyagen supports the progress towards therapeutic options for rare diseases through a variety of informative and collaborative research resources. Over the month following Rare Disease Day 2021, we have published several articles covering research related to genetic rare diseases, including advancements in gene therapy, case studies, and more.
Herein, we have collected all of our resources and support materials related to rare diseases and related gene therapies.
Rare diseases are defined as any disease which affect a small percentage of people in the general population. Due to their rarity and high level of symptom complexity, rare diseases are often difficult for health professionals to diagnose. Most rare diseases appear during adulthood, although there are a portion that exhibit symptoms at birth or in childhood. Genetic changes that cause rare disease may either be inherited or occur spontaneously in the first individual diagnosed. Rare diseases are often progressive, posing additional challenges for the patients and their families.
There is still no cure for most rare diseases and few treatments are available. Given the predominant genetic basis for rare diseases, gene therapy brings hope to rare disease patients as a promising treatment – oftentimes, where no therapeutic option existed before.
To support the research of rare diseases, our recent ‘Weekly Gene’ articles have focused on specific genes linked to the pathogenesis of rare diseases, such as spinal muscular atrophy (SMA), Crohn’s disease, multiple sclerosis (MS), Huntington’s disease (HD), and more.
>> Huntington’s disease (HD) & HTT
>> Spinal muscular atrophy (SMA) & SMN1
>> Kaposi's sarcoma (Immunodeficiency 16) & CD134/TNFRSF4
Cyagen is committed to enabling the development of therapeutics for rare diseases by delivering genotype-guaranteed animal models to study disease mechanisms, target validation, drug screening and more.
>> Case Studies in Gene Therapy - Applications of Accurate Mouse Models
>> Application of AAV-CRISPR/Cas9 Gene Therapy - Point Mutation Mice in Familial Hypercholesterolemia
Rare disease researchers are invited to join Cyagen’s Rare Disease Model Collaboration Program and help inform the development of precise animal models for rare disease studies. With this program, we aim to build a community of rare disease researchers with ideas for the next generation of animal models needed to advance their field of study. If you have any plans, strategies, or recommendations on rare disease model development, please join our Program:
>> Rare Disease Model Collaboration Program
Developing a new therapy for a rare disease faces huge challenges, such as the difficulty to perform clinical trials with the small number of rare disease patients. For these clinical limitations, genetically engineered mouse or rat models plays an important role in rare disease research.
A genetically engineered disease model can display some or all the pathological processes that are observed in an actual human patient with rare disease. Developing and studying disease models can provide researchers key information on rare disease mechanisms and potential treatment approaches - supporting efficient drug development for rare diseases, from gene discovery & target validation, through drug screening, safety and efficacy studies, and more.
In under 15 years, Cyagen has become a leading provider of custom mouse and rat models – delivering over 78,400 models to researchers worldwide and receiving over 4,800 peer-reviewed citations. Cyagen prides itself on its premium customer service: including price-matching, client access to complimentary technical consultations, full confidentiality, and a 100% money-back service guarantee.
1. Complete research solutions – from custom rodent model generation to therapeutic viral packaging and injection.
2. Both in vivo (animal) and in vitro (cell) experimental models available
3. Expert scientific team provides in-depth technical support in formulating research model strategies.
From strategy design through to delivery of research-ready custom models, Cyagen offers complete outsourcing for all your animal model needs. We can create any custom mouse or rat model with guaranteed genotype validation, even offering phenotypic characterization and other downstream services required to establish a rare disease model.
Contact us with your project details to request your complimentary model generation project consultation, strategy, and quote.
We will respond to you in 1-2 business days.