Experimental Gene Therapies – Tuesday 30th March 2021

Experimental Gene Therapies – Tuesday 30th March 2021

When I was working out genetics in relation to Rett Syndrome, the common belief in the field at that time was that most or many disease causing mutations were random.
In working out the cause of the mutation in Rett I discovered that at least for some, the gene error wasn’t random.

When in the laboratory and an error is engineered it is not the same as when a person is born with the error, in that the mouse who has been given the error, doesn’t have the fundamental cause of the error.

And when I finished studying genetics, I determined that for some people gene therapy would not be an option because by affecting the error in one place, it followed that this could trigger a problem elsewhere and a weakness area the likely target. And my son and I are among that particular group unfortunately. There is a strict mathematical equation in the genome and when it has utilised certain options there is no, for want of a better term, lee-way room for this type of therapy.

Gene therapy certainly has a place, but i felt that I should share my findings, for what they are worth and suggest caution for those people who may not be suitable for this type of treatment.
Sincerely,
Fiona MacLeod (C)