Gene Therapy

[ed34]

The first Gene Therapy treatment in Europe has finally been given the go ahead. The Drug Glybera
I believe uses a viral technique to replace faulty genes. I have to say I am disappointed in that they have
picked a condition which only affects one in a million people. There are far more pressing diseases, but
I suppose it's a good start anyway.
The disease being treated is the inability to break down fats.

[Paderla Anitha, DNB]

Hello and hope you are doing well.

Agree with you. But here its not clear if they are actually correcting the defective gene. Its more like introducing a healthy gene. Glybera is capable of encasing the correct LPL gene in an adeno-associated virus (AAV) which hones in on muscle cells. The virus infects muscle cells with a copy of a healthy gene and a one-off treatment is effective. Lets hope for the best.

[ed34]

Thank you for your reply. It would appear that the human genome project didn't lead to the promising hopes they led us to believe. I did read an interesting article a few years ago by a geneticist, who said that just switching genes is not the key. He believed we would make situations much worse. He stated that the key is switching genes in the right sequence and at the right time during development. He said that many genes reference each other, so switching a few genes could have implications across many hundreds of them.
I suppose it makes sense because genes are the codes for maintaining and building cells. If we just change instructions for building a car, say the wheel diameter, then it would cause all kinds of design flaws unless lots of modifications are done. Sounds simple enough to just change the wheel size, but the suspension, braking system, axle, wheel arches, etc are all going to possibly be affected. If the wheel size is changed during manufacture, then those adjustments could all be done at the time of assembly giving perfect results.

[Paderla Anitha, DNB]

Hello and hope you are doing well.

Gene therapy has been tried to treat Parkinsons disease and Diabetes mellitus. Its still under trial basis and long term studies are needed to know its effectiveness. For the Drug Glybera used to correct  lipoprotein lipase deficiency, trials have resulted in some patients developing leukemia.

Hope this helped and do keep us posted.