Modern therapies are those that help to influence and improve the phenotype by advanced methods.
There are three main modern therapies:
- Influencing the expression of the gene
- Stem cell transplantation
- Gene therapy
Influencing the expression of the gene
- If the disease is caused by the over-production of a toxic protein eg Huntington disease, we can reduce the expression of that protein by targeting the RNA.
- siRNA's will do this by binding to the RNA(details of this are discussed in the Medical Genetics I: Epigenetics)
Stem cell transplantation
- This is when stem cells are used to produce the missing cells or tissue.
- It has two forms: nuclear transplantation and hematopoeitic stem cell transplantation.
- In nuclear transplantation, the donor nuclei(diploid nuclei) is put into an oocyte that its nucleus has been removed. This cloned embryo will go on to form a blastocyst that can be used to make several organs and