Inherited Metabolic Disorders Stem Cell Therapy Development

Inherited metabolic disorders (IMDs) are a group of genetic disorders that affect the body's metabolic processes. These disorders are caused by mutations in genes that encode enzymes or other proteins involved in the breakdown, synthesis, or transport of molecules such as sugars, amino acids, and fats. CD BioSciences offers stem cell therapy development service for IMDs.

Introduction into Inherited Metabolic Disorders

IMDs can affect any part of the body, including the brain, liver, muscles, and bones, and can cause a wide range of symptoms. These symptoms may include developmental delays, intellectual disability, seizures, muscle weakness, and organ failure. IMDs are usually inherited in an autosomal recessive manner, meaning that a child must inherit two copies of the defective gene, one from each parent, to develop the disorder. In some cases, IMDs can also be inherited in an X-linked manner or in a dominant pattern.

Classification of IMDs (Ferreira, Carlos R et al., 2021)

Examples of Stem Cell Therapy for IMDs

Adrenoleukodystrophy (ALD)

ALD is a disorder that affects the breakdown of fatty acids, leading to damage to the brain and nervous system. Hematopoietic stem cell transplantation (HSCT) has been used successfully to treat patients with ALD. In HSCT, stem cells from a compatible donor are transplanted into the patient's bone marrow, replacing the defective cells with healthy ones.

Treatment protocol for the gene therapy in ALD (Zhu, Jia et al., 2020)

Mucopolysaccharidoses (MPS)

MPS is a group of disorders that affect the breakdown of complex sugars, leading to organ damage and developmental delays. Stem cell transplantation has been used to treat some types of MPS, such as MPS-I, MPS-II, and MPS-VI. In stem cell transplantation, stem cells are obtained from a compatible donor and transplanted into the patient's body, producing functional enzymes and replacing the defective ones.

Ex vivo vs in vivo stem cell therapy for MPS (Hurt, Sarah C et al., 2021)

Metachromatic Leukodystrophy (MLD)

MLD is a disorder that affects the breakdown of fatty acids, leading to damage to the brain and nervous system. Stem cell transplantation has been used to treat patients with MLD. In stem cell transplantation, stem cells from a compatible donor are transplanted into the patient's body, producing functional enzymes and replacing the defective ones.

Krabbe Disease

Krabbe disease is a disorder that affects the breakdown of lipids, leading to damage to the brain and nervous system. Stem cell transplantation has been used to treat some patients with Krabbe disease. In stem cell transplantation, stem cells from a compatible donor are transplanted into the patient's body, producing functional enzymes and replacing the defective ones.

Our Services

CD BioSciences offers stem cell therapy development service for IMDs, based on our advanced technology and professional team. Our stem cell therapy development services are provided in IMDs included but not limited as follows:

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.

References

1. Ferreira, Carlos R et al. "An international classification of inherited metabolic disorders (ICIMD)." Journal of inherited metabolic disease vol. 44,1 (2021): 164-177.
2. Zhu, Jia et al. "The Changing Face of Adrenoleukodystrophy." Endocrine reviews vol. 41,4 (2020): 577–593.
3. Hurt, Sarah C et al. "Mucopolysaccharidoses type I gene therapy." Journal of inherited metabolic disease vol. 44,5 (2021): 1088-1098.