Fanconi Anemia Stem Cell Therapy Development

Fanconi anemia (FA) is a rare genetic disorder that affects the production of blood cells and is characterized by bone marrow failure, increased risk of cancer, and physical abnormalities. It is caused by mutations in genes responsible for DNA repair, which makes individuals with Fanconi anemia highly sensitive to the effects of certain chemicals and radiation. CD BioSciences offers stem cell therapy development services for Fanconi anemia.

Introduction into Fanconi Anemia

Fanconi anemia is a rare genetic disorder that affects the body's ability to repair damaged DNA. It is caused by mutations in genes responsible for DNA repair, which can result in bone marrow failure, increased risk of cancer, and physical abnormalities. One of the most common symptoms of Fanconi anemia is bone marrow failure, which can lead to a shortage of red blood cells, white blood cells, and platelets. This can cause anemia, infections, and bleeding disorders. Individuals with Fanconi anemia are also at an increased risk of developing certain types of cancer, including leukemia, solid tumors, and squamous cell carcinoma of the head and neck.

Diverse molecular functions of the FA pathway (Dokal, Inderjeet et al., 2022)

Proteins encoded by FA and FA-like genes are involved in DNA repair. Specifically, eight FA proteins (FANCA, FANCB, FANCC, FANCE, FANCF, FANCG, FANCL, and FANCM) interact to form a nuclear complex, the FA core complex, which is required to activate the FANCI-FANCD2 complex into a monoubiquitinated form (FANCI-FANCD2-Ub). FANCI-FANCD2-Ub then interacts with DNA repair proteins such as BRCA2, BRCA1 and RAD51, leading to DNA damage repair. FA proteins also play other functional roles, including cytokine regulation, mitochondrial autophagy, and ribosome biosynthesis.

Stem Cell Therapy for Fanconi Anemia

Stem cell therapy, specifically hematopoietic stem cell transplantation (HSCT), is currently the only curative treatment for Fanconi anemia. HSCT involves replacing a patient's defective bone marrow stem cells with healthy ones from a donor. HSCT can be a very effective treatment for Fanconi anemia, particularly in preventing or delaying the onset of leukemia and solid tumors associated with the condition. However, it also carries risks, such as graft-versus-host disease and other complications, and is not suitable for all patients.

The process of HSCT begins with a conditioning regimen, which involves the use of high-dose chemotherapy and/or radiation to destroy the patient's existing bone marrow and make room for the donor stem cells. Once the conditioning regimen is complete, the donor stem cells are infused into the patient's bloodstream. Over time, the donor stem cells migrate to the bone marrow and begin producing new blood cells.

While HSCT can be a challenging and complex treatment, it offers the potential for a cure for patients with Fanconi anemia, and has improved outcomes for many individuals with the condition.

Our Services

CD BioSciences offers stem cell therapy development service for Fanconi anemia. Based on our professional laboratory team, advanced technologies and years of experience, we guarantee to provide services of high-quality for assisting our global customers making better breakthrough in the area of Fanconi anemia therapeutic development.

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. Dokal, Inderjeet et al. "Inherited bone marrow failure in the pediatric patient." Blood vol. 140,6 (2022): 556-570.