Neurodegenerative Disease Therapy Development
At Protheragen, we have taken the lead in the development of novel RNA-based therapies, using piRNAs to transform the therapeutics of neurodegenerative diseases. We aim to offer the cutting-edge services in the development of piRNA-based therapies for neurodegenerative diseases, enabling researchers and biopharmaceutical companies to advance new therapies for these debilitating conditions.
piRNAs and Neurodegenerative Disease Therapy
Neurodegenerative conditions including Alzheimer's disease, amyotrophic lateral sclerosis (ALS), Huntington's disease, and Parkinson's disease are marked by a progressive loss of neuronal structure and function. New studies have revealed possible mechanisms of piRNAs in the therapeutics of neurodegenerative disorders. Using these mechanisms, piRNA-based therapies provide a feasible approach for addressing the complexity of neurodegenerative diseases.
By designing piRNAs that affect specific genes associated with disease pathology, such as those responsible for protein aggregation and neuroinflammation, scientists can alter gene expression levels to prevent the progression of the disease and the consequent neuronal loss.
Gene Silencing and Regulation
Impairment of epigenetic processes is characteristic of many neurodegenerative diseases, and brain regions that are altered can potentially recover their gene expression patterns and cellular activities through piRNAs acting as epigenetic regulators.
Epigenetic Modulation
By targeting pathways involved in neuroprotection and neuron survival, piRNAs may be improve cellular resistance to stressors causing a disease, for instance, protein misfolding or oxidative damage, and consequently enhance the survival of neurons and synaptic plasticity.
Neuroprotection
piRNA-Based Neurodegenerative Disease Therapy Development
piRNA-based therapies offer substantial advantages in the therapeutics of neurodegenerative diseases, including high specificity, long-lasting efficacy, and broad spectrum. Presently, numerous therapies for neurodegenerative diseases are in the early stages of research, with the goal of pioneering novel approaches that target disease mechanisms at the molecular level.
Table 1 piRNA-based therapeutic pipeline for neurodegenerative diseases.
| Disease | Target | Mechanism of Action | Development Stage |
| Alzheimer's Disease | Amyloid-beta, Tau | Reduce amyloid-beta plaques and tau tangles. | Preclinical |
| Parkinson's Disease | Alpha-synuclein | Target alpha-synuclein aggregation and provide neuroprotection. | Preclinical |
| Amyotrophic Lateral Sclerosis (ALS) | SOD1, TDP-43 | Silence mutant SOD1 and TDP-43 transcripts. | Early-Stage Research |
| Huntington's Disease | Mutant Huntingtin Protein | Reduce mutant huntingtin levels. | Preclinical |
| Frontotemporal Dementia (FTD) | TDP-43, Progranulin | Regulate gene expression and provide neuroprotection. | Early-Stage Research |
| Spinocerebellar Ataxia | Ataxin Proteins | Silence ataxin-1 and ataxin-3 transcripts. | Early-Stage Research |
Our Services
Utilizing the unique features of piRNAs, Protheragen is focused on creating pioneering piRNA-based therapies which would profoundly change the paradigm of neurodegenerative diseases. We ensure the utmost therapeutic safety and effectiveness through a strict workflow that includes target selection, piRNA construction, delivery systems engineering, and subsequent testing and optimization phases.
Service Workflow
Target
Identification
piRNA
Design
Delivery System
Development
In Vitro and
In Vivo Validation
Therapy
Optimization
Therapy Types and Disease Areas
Therapy Types
- Protein Misfolding Inhibitor Development
- Excitotoxicity Inhibitor Development
- Anti-inflammatory piRNA Development
- Antioxidant piRNA Development
- Mitochondrial Function Regulator Development
- More
Disease Areas
- Alzheimer's Disease
- Parkinson's Disease
- Amyotrophic Lateral Sclerosis (ALS)
- Huntington's Disease
- Frontotemporal Dementia (FTD)
- More
Protheragen seeks to advance the innovation of new therapies for neurodegenerative diseases using the unparalleled characteristics of piRNAs by providing cutting-edge piRNA research and therapeutic development services. If you are interested in our services, please don't hesitate to contact us for further information and pricing details on the services we offer.
Reference
- Sato, Kaoru, Ken-ichi Takayama, and Satoshi Inoue. "Role of piRNA biogenesis and its neuronal function in the development of neurodegenerative diseases." Frontiers in Aging Neuroscience 15 (2023): 1157818.