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Stem Cell Gene Therapy to Revolutionize Medicine

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Stem cells and regenerative medicine are revolutionizing and improving the medical arena on many fronts. With the use of stem cell therapy victims of incurable and lethal diseases, such as AIDS and Leukemia, are experiencing a decrease in symptoms, in some cases complete remission, and as a result restoring the quality of their lives. The latest research in stem cell therapy is gene therapy, a process in which stem cells are being engineered and delivered to help the body properly function.


Neurodegenerative disorders such as Parkinson's and amyotrophic lateral sclerosis (ALS) have been considered life threatening diseases that have many different underlying causes which diminish the quality of life. However, according to Jason Williams, M.D., founder and CEO of Neuralgene, a start up biotech company for AAV gene therapy, the process of gene therapy may have found a way to reduce and prevent the re-occurrence of Neurodegenerative disorder symptoms. Dr. Williams said "This is a completely new therapy for ALS, and the groundwork for this technology will lead to the treatment of many other diseases."

Throughout his extensive stem cell based scientific research, Dr. Williams has discovered a new way to treat victims of neural-degenerative diseases that my have long-lasting effects. It all started when he found that the production of Factor H by fat-derived mesenchymal stem cells may be a key to activity and the production of proteins that inhibit the attack of ALS. As Dr. Williams began to develop gene therapy he targeted neural growth factors, and a protein, TDP-43, that inhibits ALS. As a result, what is known as the AAV9 viral vector delivers and distributes neural growth factors, TDP-43 protein and multiple genes including the Factor H, to the malfunctioning neural cells. Animal studies have since demonstrated the safety of this gene therapy platform.


His development today focuses on gene delivery to the brain and spinal cord with genetically engineered stem cells. Dr. Williams was quoted saying "Our gene therapy will target several of the main underlying mechanisms related to ALS with the hopes of getting a good response in a larger group of patients. However, our platform is versatile, allowing us to change and add different target genes. We expect that soon we will be able to perform a detailed genetic analysis of the patient, identifying their exact underlying cause of ALS. Then we will be able to tailor the therapy to each individual patient."

To read the original press release of this story please click here.

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