Doctors make breakthrough in repairing genetic defects

Doctors make breakthrough in repairing genetic defects

Doctors have treated a life-threatening blood disease by repairing flaws in the genetic code of a living animal, the first time such an ambitious feat has been achieved.

The work raises the prospect of powerful new therapies that can target and repair the genetic defects behind a wide range of human diseases that cannot be tackled with modern medicines.

The new technique, called genome editing, holds particular promise for a group of illnesses that run in families and are caused by faults in genes that underpin the healthy working of the immune system, bone marrow and liver.

To demonstrate the therapy, researchers treated mice that were bred to develop haemophilia B, an inherited bleeding disorder that destroys the body's ability to form blood clots.

Normally, when the body suffers a cut or graze, proteins called clotting factors combine with platelet cells in the blood to make it sticky and form a clot that stops any bleeding.

But people born with haemophilia B carry a defect in a gene that makes clotting proteins, leaving them vulnerable to excessive bleeding, even when they have not sustained an injury.

About one in 30,000 boys are born with haemophilia B which, at its most severe, requires patients to have frequent infusions of blood clotting factors to prevent spontaneous haemorrhages.

In a report in the journal Nature, a team led by Katherine High at the Children's Hospital of Philadelphia describes how genome editing reversed haemophilia B in mice, restoring their blood clotting times to near-normal without causing any apparent side-effects.

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Science has once again advanced the realm of regenrative medicine. I think that major cures and irrdadication of dieases is just around the corner for us. Hopefully we can keep the dark side of this medical breakthrough in check in hopes these advancemens are not used to genetically enhance people.

Stem cell test done on burn victim repaired the affected areas ina few days instead of months.
A HIV ingected male in Germany is cured of that disease.
Ability to implant stem cells into pig in order to grow human body parts.
Advances in the realm of blood where only one source is needed since its compatible with all blood types.
The French locate the enzyme as well as gene location that directly affects aging and death, and now have a theory on how to trun the enzyme back on, theoretically extending life indefinitely.

Qite the exciting times we live in.. Now, if we can just move beyond politics, nationalism war and greed and come together as a species..

I was going to end with the phrase the sky is the limit, until I found another quote that says -

Don't tell me thee skys the limit when their are footprints on the moon.

That last one seemed more appropriate and hopeful.

Limits are endless
Possibilities are limitless
S&F

reply to post by Xcathdra


I seem to remember that years back-- they actually tried this on humans. I remember there was a small test group of people who were genetically born with hemophilia and a pharmaceutical company altered a virus. The virus was supposed to change the genetic code of hemophiliacs.

The altered virus was injected into all the hemophiliacs in the study. And all died, but one. The only survivor of the experiment is the only human being cured of hemophilia. So it was more of a failure than a cure since every other participant died.

Not sure when the experiment was done, but I did read it somewhere on the net in the past. So it's probably still on the net somewhere.

reply to post by Xcathdra


Eventually, fixing human beings will be as simple as fixing cars.

Originally posted by SteveR
reply to post by Xcathdra

 

Eventually, fixing human beings will be as simple as fixing cars.

Hopefully it will be that easy, without the cost of labor of course

I came across a few more articles talking about other imoprivements in regenerative medicine. Hopefully there wont be to many hiccups with human application. We need all the help we can get right now.