Applications

Start Your Project Now!

Please note: Our products and services are not intended to be used directly in diagnostic or therapeutic procedures.

Here's how you can reach us...

Tel:
Email:

Gene Therapy for Neurodegenerative Diseases

Inquiry

Neurodegenerative diseases are characterized by progressive neuronal degeneration and dysfunction, and advances in the field of gene therapy have opened new pathways for the treatment of these diseases by targeting the root cause of the disease. CD Formulation specializes in the development of gene therapy formulations, and we provide innovative solutions for the development of gene therapy formulations designed to facilitate our clients' progress in gene therapy research for neurodegenerative diseases.

Advantages of Gene Therapy for Neurodegenerative Diseases

Precision targeting. Gene therapy can be designed to target specific neurons or pathologic mechanisms (e.g., beta-amyloid aggregation), significantly outperforming the broad-spectrum effects of conventional drugs.
Long-term efficacy and individualization. Long-term gene expression can be achieved with a single therapy, negating the requirement for regular dosage. tailored treatment plans according to the patient's genotype (for example, ALS patients with particular mutations) to improve safety and effectiveness.
Breaking through the limitations of traditional treatments. Gene therapy is utilized to restore endogenous dopamine synthesis, which provides longer-lasting symptom control for Parkinson's disease. Additionally, gene therapy can bridge the gap in rare disorders where no medication is currently available.

Main Applications of Gene Therapy for Neurodegenerative Diseases

Parkinson's disease

The relationship between mitochondrial dysfunction and neuroinflammatory processes is highly relevant, and these two factors are often considered to be common features of several neurodegenerative diseases. The use of gene modification, gene knockout or insertion, epigenetic alteration, and transcriptional activation/repression in CRISPR/Cas9 technology opens up the possibility of treating PD and makes it possible to study the underlying genetic causes of the disease.

Alzheimer's disease

Gene therapy slows the progression of the disease by lowering levels of beta-amyloid (Aβ) and Tau proteins in the brain. For example, expression of miRNA using AAV vectors can reduce Aβ levels. In addition, carrying specific genes, such as the leptin gene, through lentiviral vectors can reduce Aβ and Tau accumulation and improve synaptic density. In addition gene therapy works to regulate the expression of genes associated with neuroinflammation to reduce neuronal damage from inflammatory responses.

Huntington's disease

The buildup of mutant Huntington's protein (mHTT), which results in neuronal death, is the primary pathogenic characteristic of Huntington's disease, a neurodegenerative condition brought on by mutations in the Huntington gene (HTT). By precisely destroying the mHTT, RNAi technology efficiently lowers the level of mHTT and improves neuropathology and motor function. By modifying the HTT gene to permanently silence or repair mHTT, CRISPR/Cas9 gene editing technology shows promise for treating Huntington's disease. Additionally, using AAV vectors containing artificial microRNAs (miRNAs) to specifically silence the HTT gene lowers the amount of mHTT in patients' cerebrospinal fluid.

Our Capabilities in Neurodegenerative Diseases Gene Therapy

Innovative vector development. We continue to develop novel AAV coatings to improve vector invasion efficiency into the nervous system so that the blood-brain barrier is no longer a barrier to drug delivery.
Individualized solutions. We develop treatment plans based on the characteristics of our client's projects and continuously optimize our technical systems to ensure that our client's research needs are met.
Diverse gene therapy strategies. We combine multiple strategies such as gene replacement, gene silencing, and gene editing to personalize treatment solutions for different types of neurodegenerative disease research.

Explore Our Gene Therapy Formulation Development for Cardiovascular diseases

Adeno-Associated Viral Vector Development

Gene therapy using AAV as a vector is a very promising therapeutic modality for neurological diseases, which not only helps to transform some molecules that could not be drug targets before into drug targets, but also provides better targeting. The novel AAV capsid screening and development we provide play an important role in neurodegenerative disease research.

Gene Editing Technology

We offer gene editing technologies that can control gene expression or correct pathogenic mutations. In gene editing, we recognize DNA-binding structural domains of specific genomic sequences and nuclease enzymes that produce double-stranded breaks (DSBs), which, together with other cellular components, allow for the knockout, insertion, or modification of specific genes.

CD Formulation specializes in gene therapy technologies and drug development for neurological disorders, and we already have deep basic research in the field of neurodegenerative disease research. If you are interested in us, please feel free to contact us.