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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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Despite the occurrence of leukemia in some of these patients the overall success rate of gene therapy outperforms the results obtained after allogeneic HSCT with HLA-mismatched donors.
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.
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.
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