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During gene editing by CRISPR/Cas9, the genes encoding the Cas9 protein and sgRNA are introduced into a cell where a targeted genomic change is planned. The sgRNA has a region complementary to the chosen genomic target sequence (purple); this region can be engineered to include any desired sequence. A complex consisting of the CRISPR sgRNA and the Cas9 protein forms within the cell & binds to the chosen target site in the DNA.
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In April 2016 Chinese scientists were reported to have made a second unsuccessful attempt to alter the DNA of non-viable human embryos using CRISPR - this time to alter the CCR9 gene to make the embryo HIV resistant.
Heritable modifications could have unintended and far-reaching consequences for human evolution, genetically (e.g. gene/environment interactions) and culturally (e.g. social Darwinism). Altering of gametocytes and embryos to generate inheritable changes in humans was defined to be responsible.
Viral vectors carry the risks of toxicity, inflammatory responses, and gene control and targeting issues.
Some commonly occurring disorders, such as heart disease, high blood pressure, Alzheimer's disease, arthritis, and diabetes, are affected by variations in multiple genes, which complicate gene therapy.
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