Bill Maurits sat in a waiting room in Philadelphia, ready to receive intravenous injections containing trillions of viral particles. He thought in his head: "Get started! I don't want to wait anymore."
Moritz suffers from hemophilia B, causing his body to fail to produce a blood coagulation protein: Factor IX, which can cause a lot of blood loss even in small wounds. Since the age of 10, he has relied on injections of "extraordinary" exaggerated alternative proteins to sustain life. His joints have been severely damaged by repeated blood stasis. Recently, his left ankle hurts.
Moritz is an engineering designer. He joined a clinical trial to receive an injection of a viral vector containing a normal gene that produces the coagulation protein factor IX. During the two months after the end of treatment, he did not receive a replacement protein for factor IX. After 1 year, the effect of the treatment is still maintained. He will not bleed when he sprains his ankle or bruise. He believes: "Gene therapy works, this is the only explanation."
â–²Bill Moritz visited Niagara Falls with his family after 2 months of gene therapy (Source: technologyreview)
These results are undoubtedly satisfactory for patients like Moritz and for scientists who have been working on genetic therapy for 30 years. If gene therapy is successful, catastrophic diseases like hemophilia can be cured in one dose, although its price may be high. “But it may be worth it,†said Mark Skinner, a former chairman of the World Federation of Hemophilia. Hemophilia patients face enormous challenges every day. Due to the lack of coagulation factor protein in the body, any small wounds and bruises can be dangerous, and undetected bleeding in the body can be life-threatening. Gene therapy seems to be a good choice because it may be a one-time "cure."
Gene therapy for 30 years
The gene therapy accepted by Moritz comes from a company called Spark. The company's president and chief scientific officer, co-founder Dr. Katherine High is a hematologist with a long-standing interest in gene therapy for genetic diseases.
â–² Dr. Katherine High, President of Spark (Source: Spark official website)
In 1989, Dr. Catherine, a professor at the University of Pennsylvania, studied the isolation of the Factor IX protein from dogs. In the following decade, Dr. Catherine, the director of the Cell and Molecular Therapy Center at the Children's Hospital of Philadelphia (CHOP), formed a multidisciplinary team of scientists and researchers dedicated to discovering new genes for genetic diseases and Cell therapy and promote rapid transformation of preclinical findings into clinical applications. Gene therapy continues to effectively cure hundreds of hemophilia dogs. But attempts in the human body have encountered bottlenecks.
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