Fatal familial insomnia, or FFI, is a genetic condition marked by neurodegeneration that is genetically inherited and ultimately fatal to the individual. It is categorized as a type of prion disease which is a group of diseases that affect the brain and the nerves in a gradually degenerative manner. Our company, which is the leader in research on infectious diseases, provides a full range of multi-work integrated vaccine and therapeutic development solutions to assist researchers and scientists in this area of FFI.
Overview of Fatal Familial Insomnia (FFI)
FFI, which is classified as a genetic prion disease, appears to be a chromosome disorder that affects a limited number of individuals and has serious consequences for those afflicted. The attack of FFI tends to manifest as a slowly progressive disease with prominent features of insomnia that worsen over time and can eventually lead to the total absence of sleep. In addition to the typical presentation, such individuals may have hallucinations, loss of some cognitive function, and even weight loss without any apparent cause.
Fig.1 The cranial magnetic resonance imaging (MRI) results. (Yukang, T., et al., 2021)
Pathogenesis of Fatal Familial Insomnia (FFI)
The FFI disease is caused by a genetic anomaly, its association with a mutation at codon 178 of the prion protein gene, leading to a D178N substitution in the protein. This gene malfunction results in the accumulation of a misfolded protein within the brain and eventually results in neuron damage and death. Within FFI, features of presentation also include atrophy of mediodorsal and anteroventral thalamic nuclei which leads to severe debilitating symptoms.
Fig.2 Physiological functions of disease-onset-associated genes and PRNP. (Thüne, K., et al., 2023)
Vaccine and Therapeutic Development for Fatal Familial Insomnia (FFI)
Vaccine Development
Efforts are being made in some research that regard the problems of vaccine development in great detail.
- DNA vaccines that deliver certain sequences of prion proteins are also been worked on.
- Use of several modified forms of the PrP; truncated, dimeric, heterologous, and crosslinked PrP peptides.
- Uses either bacterial or viral vectors to circumvent immune tolerance and elicit an immune response that protects against FFI.
Therapeutic Development
Therapeutic approaches that include antiprion drugs, immunotherapy, and gene therapy appear promising.
- Antiprion compounds such as amphotericin B for instance disrupt the abnormal form of prion protein.
- Immunotherapy comprises the administration of antibodies to target and clear the abnormal prion proteins.
- Gene therapy involves the deletion of the abnormal gene and introducing normal copies of the PRNP gene.
Our Services
The developed platform for vaccine and therapeutic creation, as well as the model of infectious disease provided by our company, facilitates the researchers by providing the necessary resources. Such an environment promotes collaboration and the exchange of ideas among specialists and is aimed at new interpretations of the mechanisms of action of diseases like FFI.
Vaccine Development Platforms
Therapeutic Development Platforms
Infectious Disease Models
Preclinical model systems guide us in comprehending the mechanisms associated with different diseases coupled with testing potential therapeutic agents. We provide you with a variety of animal models that could help to comprehend the pathology of FFI with the intention of developing effective therapeutic and intervention approaches.
Transgenic animals that express mutated forms of the human prion protein gene associated with FFI have been utilized to study prion diseases, they can develop symptoms of FFI.
Optional Models: ki-3F4-FFI; Tg15972; FFI-K5, etc.
Why Choose Us
Utilizing the latest and burgeoning technologies, along with extraordinary support, our firm motivates scientists to progress the edges of their expertise as stated in their research revolving around infectious diseases, while seeking to further enhance therapeutic effects.
If you are interested in our services, we invite you to contact us for further information and to obtain detailed quotations.
References
- Yukang, Tan et al. "A fatal familial insomnia patient newly diagnosed as having depression: A case report." Medicine 100.41 (2021): e27544.
- Thüne, Katrin et al. "Genetic Variants Associated with the Age of Onset Identified by Whole-Exome Sequencing in Fatal Familial Insomnia." Cells 12.16 (2023): 2053.
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