First, that the introduced gene may not function, second, that when corrected cells are reintroduced, these may be outnumbered by the noncured resident cells, and third, that there are only few diseases affeciting only a single tissue.
During the last 5 -10 years, it has become routine exercise to isolate any gene. The isolated gene, may either be directly injected into the cell or be carried by a virus, to which it is linked by recombinant DNA technique.
After entering the cell, the gene may become a part of nuclear DNA or remain free in cytoplasm like extrachromosomal DNA. However, in each case RNA is synthesized only at the rate of few copies per cell in comparison to normal cells where thousands of copies are made.
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