Gene Therapy Improves Vision In Nearly Blind Patients

[TZ]

ScienceDaily (Apr. 28, 2008) — In a clinical trial at The Children's Hospital of Philadelphia, researchers from The University of Pennsylvania have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness. Although the patients have not achieved normal eyesight, the preliminary results set the stage for further studies of an innovative treatment for this and possibly other retinal diseases.

An international team led by The University of Pennsylvania, The Children's Hospital of Philadelphia, the Second University of Naples and the Telethon Institute of Genetics and Medicine (both in Italy), and several other American institutions reported their findings today in an online article in the New England Journal of Medicine.

"This is the first gene therapy trial for a nonlethal pediatric condition," said Albert M. Maguire, M.D., Associate Professor, Department of Ophthalmology, University of Pennsylvania School of Medicine and a physician at The Children's Hospital of Philadelphia. Maguire, together with his wife, Jean Bennett, M.D., Ph.D., Professor of Ophthalmology at Penn and Senior Investigator at the F.M. Kirby Center for Molecular Ophthalmology at Penn's Scheie Eye Institute, have been researching inherited retinal degenerations such as Leber congenital amaurosis (LCA), for 18 years. LCA is a group of inherited blinding diseases that damages light receptors in the retina. It usually begins stealing sight in early childhood and causes total blindness during a patient's twenties or thirties. Currently, there is no treatment for LCA.

http://www.sciencedaily.com/releases/2008/04/080427194726.htm

Cross posted from GD..This is such an exciting medical breakthrough. I really do think gene therapy has the potential to be a very effective treatment/cure for many diseases/conditions

[FirstLight]

1. I am totally excited about this...

My daughter has a birth defect and is totally blind in her left eye...I just sent the leading DR an email asking if they have used this therapy in her condition... cross your fingers!!

edit- typos from excited typing!!!!

[TZ]

2. Good luck!

I hope your daughter qualifies for treatment...:bounce:

[Orrex]

3. Meh. This whole "blindness" fad was created to boost profits for Big Pharma

And did you know that, thanks to the FDA, it's illegal to treat imperfect vision? Google told me so.

[TZ]

4. *snerk*

Yeah you know, I went to Mexico to have my illegal eye laser surgery the other year...What else did you say..something about the FDA I can't quite make out the words.....*squint*
:rofl:

[WildClarySage]

5. You know, blindness is caused by thimrosol

and the MMR vax...

[dropkickpa]

6. Very cool

Hopefully all of the preliminary testing is positive enough to warrant testing in younger LCA patients, when it may do even more good than in the older participants.

[LeftishBrit]

7. Very exciting

There are some really interesting new developments in medicine lately. I hope this helps lots of people!

I have a friend who is almost completely blind from retinitis pigmentosa (also a genetic disorder)- I wonder if eventually they'll find a way of treating that.