Modern therapies are those that help to influence and improve the phenotype by advanced methods.

There are three main modern therapies:

  1. Influencing the expression of the gene
  2. Stem cell transplantation
  3. 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

Subscribe now to continue reading

Join hundreds of successful students who use Meddists to ace their exams.

Gain access to all of the material and topics, custom-made just for you.

Continue