Adrenoleukodystrophy Stem Cell Therapy Development

Adrenoleukodystrophy (ALD) is a genetic disorder that affects the nervous system and the adrenal gland. Stem cell therapy is a treatment option for individuals with Adrenoleukodystrophy (ALD) that involves the transplantation of healthy stem cells to replace damaged cells in the body. CD BioSciences offers stem cell therapy development service for ALD, with the guaranteed quality and effectiveness.

Introduction into Adrenoleukodystrophy

Adrenoleukodystrophy (ALD) is caused by a mutation in the ABCD1 gene, which provides instructions for making a protein called adrenoleukodystrophy protein (ALDP). ALDP plays a critical role in the breakdown of very long-chain fatty acids (VLCFAs), which are essential components of cell membranes and are found throughout the body. ALD is an X-linked disorder, which means that it primarily affects males. Females can also carry the mutated gene and pass it on to their children, but they typically do not develop the severe symptoms associated with the disease. The severity and age of onset of ALD can vary depending on the type of mutation in the ABCD1 gene, as well as other factors such as age and gender.

The molecular mechanisms associated with the pathogenesis of ALD (Jia, Ming-Rui et al., 2019)

ALD can manifest in different ways and can affect people at different ages. The most severe form of the disease is known as cerebral ALD, which affects young boys and leads to progressive neurological deterioration, ultimately resulting in severe disability or death. Other forms of ALD include adrenomyeloneuropathy (AMN), which affects older males and can result in progressive weakness and numbness in the legs and difficulty with bladder and bowel control.

Stem Cell Therapy for Adrenoleukodystrophy

Stem cell therapy is a treatment option for individuals with Adrenoleukodystrophy (ALD) that involves the transplantation of healthy stem cells to replace damaged cells in the body. The goal of this therapy is to slow or halt the progression of the disease and improve the quality of life for individuals with ALD.

Stem cells used in the therapy may come from the patient's own bone marrow or from a donor, typically a sibling who is a close match. The transplant is performed through a process called hematopoietic stem cell transplantation (HSCT), in which the patient's bone marrow is first destroyed with chemotherapy and radiation therapy, and then replaced with healthy stem cells.

Advantages of Stem Cell Therapy for ALD

• Replacement of Defective Cells: By replacing the faulty cells with healthy cells that can produce the necessary enzyme to break down VLCFAs, thereby reducing their accumulation and preventing further damage.
• Potential Disease Modification: Stem cell therapy can restore normal enzyme activity and reduce VLCFA levels, preventing further deterioration of the nervous system.
• Long-term Treatment: Successful stem cell transplantation can provide a long-term treatment solution for ALD.
• Expanded Treatment Options: Stem cell therapy can potentially benefit a broader range of ALD patients, including those who may not be suitable candidates for other treatment approaches.

Our Services

CD BioSciences offers stem cell therapy development service for adrenoleukodystrophy, based on our advanced technology and professional team.

Workflow of our services

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

1. Jia, Ming-Rui et al. "Clinical characteristics and phenotype distribution in 10 Chinese patients with X-linked adrenoleukodystrophy." Experimental and therapeutic medicine vol. 18,3 (2019): 1945-1952.