I am a molecular biologist very familiar with gene therapy. There are still a lot of problems before it can be a way of cure diseases.
First of all, the delivery of DNA in to the destination in the body is simply too inefficient, the DNA needs to be coated with a chemical called cationic lipids which is toxic to cells, not well tolerated by the humans. The DNA has to be repeatedly injected since it gets degraded in the blood stream. So the toxic effects are accumulative.
Secondly, In case of zinc finger DNA binding protein which increases expression of a homone called VEGF (needed for blood vessel generation). This hormone is produced whenever an injury occurs in the concert of a lot of other factors also requied for reparing the tissue. In case of diabites, there is no injury , simply giving a patient this drug won't help much in reparing the damaged nerves and blood vessels since other tissue reparing factors are not available. Unless you gave the patient a coctail of other genes, but at the present, scientists even haven't figured out what are the other components yet, let alone design the coctail. Thirdly, this drug is no cure for diabetes, it is aimed only for one of its complications, even if you prevent amputation, the patient still have other problems and will eventually die of diabetes. Lastly, even if everything goes well for SGMO, it will take $800 mil to $1 bil to get FDA approval,therefore a lot second offerings will be necessary unless a bigger pharmacitical co buy out this co. That won't happen until promising Phase II results and more products in the pipe line. If you are a lucky share holder, by the time they get FDA approval, the pps will be $1 -2 because of dilution. Otherwise, it will be $0.0 when the co files chapter 11. Just think about the future more carefully before putting your hard-earned money on the table!
" am a molecular biologist very familiar with gene therapy. There are still a lot of problems before it can be a way of cure diseases.
First of all, the delivery of DNA in to the destination in the body is simply too inefficient, the DNA needs to be coated with a chemical called cationic lipids which is toxic to cells, not well tolerated by the humans. The DNA has to be repeatedly injected since it gets degraded in the blood stream. So the toxic effects are accumulative."
Just give us 4 or 5 examples from your exhaustive due diligence showing SGMO using a cationic lipid for delivery of genes that are toxic to cells.
Perhaps it was forgotten in all the recent excitement, but berkeley_go also failed to reply to my previous post asking what other factors were required beyond the VEGF signalling gradient for the treatment to work.
It takes a lot to be an expert in molecular biology, cell signalling and stock forecasting, and I was curious as to what the answer might be.
I don't think the activity in animal model is "profound". Animal models are always different from humans. I haven't got a chance to actually "peer review" their paper. But I know if something real good, it should be published in a high ranked journal like The Journal of Endocrinology at least, or even someting like Cell, PNAS. If it is revolutionary, it should go to Science or Nature! Good luck to your investing!
Although you rightly point out the difficulties involved in gene therapy, you should note for example, that no attempt is being made in this case to deliver the therapeutic DNA directly into the bloodstream. Instead, it is being directly administered to muscle tissue, where the expression of the DNA need only be- and, in fact, is preferably - transient. Furthermore, the gene correction technology - which might turn out to be the dominant application of this technology - will be administered ex vivo.
Neuropathy is a systemic complication, muscle injection aiming at local level will be hard to improve the systemic condition. The problem here is realy that VEGF alone won't do enough good for this condition, it should be temporary if any effet at all.