题目内容

Random House Kernerman Webster's College Dictionarydefines'genetherapy'as:"The treatment of a disease by replacing aberrant genes with normal ones, esp. through the use of viruses to transport the ________________ into the nuclei of blood cells."

查看答案

搜索结果不匹配？点我反馈

更多问题

The American Heritage® Student Science Dictionarydefines'genetherapy'as:"The treatment of disease, esp. one caused by the inheritage of a defective gene, by replacing __________________ with healthy ones through genetic engineering."

In April 2015, Chinese scientists reported results of an attempt to alter the DNA of non-viable human embryos using CRISPR/Cas9 to correct a mutation that causes β-thalassemia. The study had previously been rejected by both Nature and Science in part because of ___________________.

Before gene therapy can become a permanent cure for a condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be stable. Problems with integrating therapeutic DNA into the genome and the rapidly dividing nature of many cells prevent it from achieving _______________. So patients require multiple treatments.

If the DNA is integrated in a sensitive spot in the genome, for example in a tumor suppressor gene, the therapy could induce a tumor. This has occurred in clinical trials for X-linked severe combined immunodeficiency (X-SCID) patients, in which hematopoietic stem cells were transduced with a corrective transgene using a retrovirus, and this led to the development of _______________ in 3 of 20 patients.