Finding a safe way to
get therapeutic genes into the cells has been a major challenge, however.
Not exact matches
By comparing all of the
genes that are active in disease and normal tissue, one can
get valuable information on new pathways and potential
therapeutic targets.
«Many
therapeutic genes are only expressed transiently before they
get shut off by the host cell or they are not expressed as expected because they don't occupy the same position in the genome as the natural
genes,» explains postdoctoral researcher and co-first author Keiichiro Suzuki.
These alternate strategies include using small - molecule drugs that affect RNA metabolism or protein stability, as well as administering modified viruses for
therapeutic gene delivery (see «
Getting a fix on SMA»).
Having been excited by the potential of retroviral
gene transfer as a
therapeutic, it has been a fascinating opportunity to
get to grips with the various processes in the lab; particularly more recently when I have spent time working independently to optimise the process in various cell subsets.