题目内容
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.
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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.
In gene therapy, vector–chromatin interaction and its consequences have become more unpredictable, but vector-induced leukemogenesis remains an predictable factor in gene therapy due to its multifactorial nature.
Indeed, the success and feasibility of gene therapy is undeniable considering that the majority of the more than 60 patients treated for ADA-SCID and SCID-X1 within the last two decades experienced a clear clinical benefit.
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