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Name: Mahira
Status: other
Grade: other
Location: Outside U.S.
Country: Bosnia and Herzegovina
Date: Spring 2011


Question:
If you want to replace defective gene in fetus haw do you do it without demaging a fetus? Do you use a micro needle or something else?



Replies:
Although still in the research stage, in utero gene therapy offers much promise in the fight against heritable diseases such as cystic fibrosis and hemophilia. Fetal therapy has one major advantage over later interventions: it avoids the possibility that the immune system will view the introduced corrective DNA (and/or DNA vector such as an attenuated virus) as invasive and mount a defense. So in theory we can sneak in subtle genetic rewrites of defective genes without having to worry that the immune system will mobilize its troops!

To minimize trauma to the developing fetus, genes can be injected into the amniotic cavity (through ultrasound guidance) or into fetal circulation (umbilical vein) using needle embryofetoscopy. This procedure is scope assisted.

It should be noted that all attempts at gene therapy aim to correct somatic cell DNA, as opposed to germ cell DNA. There is a great deal of ethical resistance to germline modifications, particularly since any changes can be passed to future offspring. For couples at risk for mendelian (genetic) diseases, gene therapy does seem to be a more palatable ethical alternative to 1) abortion and 2) preimplantation selection, where “test tube” embryos are screened and selectively discarded if positive for a given disease.

Dr. Tim Durham Instructor, Office of Curriculum and Instruction University Colloquium Department of Biological Sciences Florida Gulf Coast University



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