Krabbe Disease Stem Cell Therapy Development
Krabbe disease, also known as globoid cell leukodystrophy, is a rare and progressive genetic disorder that affects the nervous system. Krabbe disease is classified as a leukodystrophy, a group of disorders characterized by the degeneration of myelin, the protective covering of nerve cells. CD BioSciences has launched a development service for Krabbe disease stem cell therapies, dedicated to advancing the treatment of this disease.
Introduction into Krabbe Disease
Krabbe disease, also known as globoid cell leukodystrophy, is a rare and often fatal genetic disorder that affects the nervous system. The primary cause of Krabbe disease is a mutation in the GALC gene, which leads to a deficiency of galactosylceramidase. This enzyme is responsible for breaking down a fatty substance called galactosylceramide, which is found in myelin. Without sufficient levels of galactosylceramidase, galactosylceramide accumulates in the nervous system, resulting in the destruction of myelin.
The severity and progression of Krabbe disease can vary widely among affected individuals. The disease is inherited in an autosomal recessive manner, which means that both parents must carry a mutated GALC gene to pass on the condition to their child. If both parents are carriers, each of their children has a 25% chance of inheriting two copies of the mutated gene and developing Krabbe disease.
| Disease Classification | Symptoms | Disease Progression | Median Survival |
|---|---|---|---|
|
Crying/Irritability, feeding difficulties, poor head control, fisted hands, developmental delay | Progressive neurologic deterioration, seizures, psychomotor regression, loss of vision, hearing and voluntary movement | 1.5-2 years |
|
Similar to early infantile | Similar to early infantile but slower progression | 9.5 years |
|
Vision problems, muscle weakness, gait changes, loss of developmental milestones | Variable progression with older patients generally experiencing slower progression | >16 years |
|
Gait disturbance, weakness, lower limb hypoesthesia, cognitive regression, spastic paraparesis | Variable progression with older patients generally experiencing slower progression | >33 years |
Early Infantile
Late Infantile
Juvenile Onset
Adult OnsetStem Cell Therapy for Krabbe Disease
Stem cell therapy, particularly hematopoietic stem cell transplantation (HSCT), has shown promise as a treatment option for Krabbe disease. HSCT involves transplanting healthy stem cells from a donor into the patient's body. These stem cells can then develop into healthy cells that produce the missing enzyme galactocerebrosidase, potentially slowing or halting the progression of the disease. HSCT has been most successful in patients who are diagnosed and treated early in the course of the disease, before irreversible neurological damage has occurred. In these cases, HSCT can potentially restore or preserve neurological function and improve quality of life.
However, HSCT is not without risks, and the procedure can have serious complications, including graft failure, infections, and graft-versus-host disease (GVHD), in which the transplanted cells attack the patient's own tissues. Therefore, more studies need to be performed for exploring the correct and safe stem cell therapy for Krabbe disease.
Our Services
CD BioSciences offers stem cell therapy development service for Krabbe disease based on our years of experience and edge-cutting technologies.
Workflow of our services
Isolating HSCs from iPSCs or primary tissue.
Engineering the HSCs to analyze gene expression.
Differentiation of HSCs into the blood cell types.
Validating pluripotency, differentiation status, and health of HSCs.
As a pioneer in biotechnology, CD BioSciences has grown into one of the largest independent biotechnology companies in the world. CD BioSciences is committed to providing professional and efficient service to our customers around the world. If you are interested in our service, please contact us.
Reference
- Feltri, M Laura et al. "Mechanisms of demyelination and neurodegeneration in globoid cell leukodystrophy." Glia vol. 69,10 (2021): 2309-2331.
For research use only, not for clinical use.
