Gene Therapy Development for Genetic Disorders

Genetic disorder is a genetic problem caused by one or more abnormalities formed in the genome with complex and multifactorial pathogenesis. The disease with single or multiple genetic mutations is likely associated with lifestyles and environmental factors. With the identification of numerous genes associated with inherited diseases, gene therapy can be viewed as a promising option in some instances. Creative Biolabs possesses the world first-class technology platforms and skillful scientific teams in the field of gene therapy development. We are able to provide customer services and advanced technologies for your research, such as the addition of a normal copy of the mutated gene, modification of messenger RNA to avoid the consequences of mutation, inhibition of expression of a mutated gene, and repair of the mutated gene by genetic editing. We are glad to cooperate with our client and accelerate gene therapy products development for a better tomorrow.

• Gene Therapy for Monogenic Disorder

Figure 1. Basic strategies for clinical gene therapy. (Emery 2004)

Monogenic disorder results from a single mutated gene and the mutation may be present on one or both chromosomes inherited from each parent. Currently, there is a growing list of single-gene diseases considered as targets for gene therapy. Monogenic disorders include cystic fibrosis (CF), polycystic kidney and Tay-Sachs disease, hematopoietic disorders (β-thalassemia and sickle cell disease, etc.). The use of modified versions of the defective β-globin gene or recombinant retrovirus vectors contains either the human γ-globin or β-globin genes that can prevent red cell sickling. Hemophilia B is another monogenic disorder caused by the factor IX deficiency that is a critical component in the blood-clotting pathway. Some groups have injected the recombinant AAV designed to express factor IX into muscle or hepatic artery for the treatment of hemophilia B.

• Gene Therapy for Multifactorial Disorders

Figure 2. AAV-mediated transfer of the transgenic insulin gene to liver cells and expression of the gene. (Yoon 2002)

Multifactorial disorders are caused by the abnormalities on two or more disease-causing genes. Compared with monogenic disorder, it not only determined is by genetic factors, but also plays a role together with genetic factors and environmental factors. Common medical problems such as Alzheimer's disease, diabetes, hearing disorders, vision disorders, congenital heart disease, muscular dystrophy, asthma, and obesity belong to multifactorial disorders. Gene therapy approaches have been introduced to treat multiple-gene disease, such as the management of diabetes. The use of AAV vector containing insulin and glucokinase genes keeps normoglycemic via long-term efficacy of diabetes gene therapy without the supply of exogenous insulin. Another successful example is injecting lentivirus vector that carries insulin gene into the diabetic rats, which allows liver cells to sense glucose, in turn, synthesize, release and store human insulin as a response.

• Features of Our Services

1. Skillful technicians and extensive experience
2. Custom service and timely feedback
3. Advanced gene therapy development platform and a GLP-compliant & IACUC-regulated facility
4. A series of stringent criteria are applied to implement quality control of experiment in order to guarantee reliability

With extensive experience on preclinical gene therapy development and project management, Creative Biolabs is dedicated to establishing the most exquisite service platform for our clients all over the world. Well-trained and experienced technicians perform all studies and our scientists can work with you to design the program that best fits your requirements. For more information in our services, please feel free to contact us to discuss your specific requirements.

References

1. Yoon, J. (2002). Recent advances in insulin gene therapy for type 1 diabetes. Trends in Molecular Medicine, 8(2), pp.62-68.
2. Emery, D. (2004). Gene therapy for genetic diseases: On the horizon. Clinical and Applied Immunology Reviews, 4(6), pp.411-422.
3. Rasko AO, J. (2016). Progress in gene therapy for genetic diseases. Pathology, 48, p. S39.