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Potential adverse effects of gene therapy have to be balanced against the clinical benefit expected for the individual patient, taking into consideration the clinical complications associated with alternative treatment options.
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Gene therapy may cause off-target effects and there is a risk that the new gene will insert itself into the path of another gene, disrupting its activity or causing insertional mutagenesis.
The team led by Kathy Niakan at the Francis Crick Institute in London used CRISPR-Cas9 to switch off a particular gene involved in embryo development, known as OCT6. Blocking the functioning of this gene means the resulting protein, also called OCT6, cannot be produced, eventually halting an embryo's development.
The team led byJunjiu Huang at Sun Yat-Sen Universityattempted to modify the gene HBB responsible for β-thalassemia, using CRISPR/Cas9. HBB encodes the β-globin protein. Mutations in the gene are responsible for β-thalassemia.
Researchers in China have reported editing the genes of human embryos to try to make them resistant to HIV infection, published in J Assist Reprod Genet (2018) used CRISPR-editing tools in non-viable embryos that were destroyed after 3 days.
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