These are exciting times for thalassaemia gene therapy with a number of clinical trials either ongoing or soon to start in both the USA and Europe.
All these trials will employ what is known as an “ex vivo” procedure for genetically correcting the bone marrow blood stem (“parent”) cells of the patient. The ex vivo procedure involves (i) harvesting blood stem cells from the thalassaemic person’s bone marrow, (ii) infecting them with a modified virus (known as a “lentivirus”) gene medicine that contains a normal functioning copy of the beta -globin (or alpha -globin) gene, (iii) treating the patient with chemotherapy to destroy their diseased bone marrow blood stem cells and (iv) returning the now genetically corrected bone marrow blood stem cells back to the patient.
Preimplantation Genetic Diagnosis (PGD)
PGD started nearly 25 years ago and thousands of babies have been born worldwide since then. In our centre we have been offering PGD to couples at risk of having children with serious genetic disorders since 1997 and are now in our 17th year of service. Over that time there have been many changes to what is offered and a significant increase in the number of referrals and PGD treatment cycles. Read More