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Since the CD199 antigen is also present on normal B-cells, and Kymriah will also destroy those normal B cells that produce antibodies, there may be an increased risk of infections for a prolonged period of time.
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Gene therapy involves a whole lot of complicated sets of activities involving tissue targeting, cellular trafficking, delivery of genes to organs, safety of the vector, activity of therapeutic protein, etc.
Gene therapy seems elegantly complicated in its concept: supply the human body with a gene that can correct a biological malfunction that causes a disease. However, there are many obstacles and some distinct questions concerning the viability of gene therapy.
Some viral vectors, like the adenoviruses, often are recognized and destroyed by the immune system so their therapeutic effects are long-lived.
In gene therapy, maintaining gene expression so it performs its role properly after vector delivery is difficult. As a result, some therapies need to be repeated often to provide short-lasting benefits.
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