A review written by The Cochrane Collaboration entitled, “Gene
Therapy for Hemophilia” has some telling information in the pros and cons of
what gene therapy has to offer the medical community as well as how a clinical
trial should be set up. This review discusses how promising gene therapy is for
people who are greatly affected by genetic diseases and how this could be used
for things other than just hemophilia in the future. Hemophilia is a X-linked
recessive gene where clotting factors are unable to function and excessive
bleeding occurs in patients.
Something I found interesting was that the clinical trial
that would be done for haemophilia would have a lot of cons. Unfortunately,
because the gene therapy treatment is not being mass produced it is extremely
expensive. In the case of those affected by haemophilia, the therapy would have
to be administered every two to three days because of the short half-live of
clotting factors in the blood. The price for treatment was not listed, but it
can be assumed that at that rate, it would be extremely hard to keep purchasing
injections for therapy. There were some interesting points brought up about the
side effects of this therapy such as the fact that some patients’ might not
adapt well because the treatment introduces new genes into a cell’s DNA which can
cause some insert mutations and could leave the patient worse off perhaps
giving them another genetic defect.
In contrast, there are an immense amount of opportunities
that gene therapy could offer for those affected by genetic disorders. It is
believed that if this therapy is successful many patients would have a lifelong
cure and mortality rates would greatly decrease. Not only the patients would
benefit but the researchers and medical physicians would as well. By gaining
knowledge about how gene therapy is successful could help broaden the types of
diseases that are treated.
References:
Sharma A, Easow
Mathew M, Sriganesh V, Neely JA, Kalipatnapu S. Gene therapy for haemophilia. Cochrane
Database of Systematic Reviews 2014, Issue 11. Art. No.: CD010822.
DOI: 10.1002/14651858.CD010822.pub2.
DOI: 10.1002/14651858.CD010822.pub2.
surprisingly the therapy has a side effects and that would decrease the amount of patient that would take the treatment, beside extremely expensive cost of the treatment.
ReplyDeleteI can imagine how expensive the price would be for patients with Hemophilia because they have to inject themselves every two or three days. Gene therapy is a very promising science idea which could improve health and economy.
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