Gene therapy improves vision in patients with congenital retinal disease


	Gene therapy improves vision in patients with congenital retinal disease

Silkweaver

5-4-2008 8:37 PM

258 views

tags:

medicine, gene therapy

Silkweaver says:

At last we see the light of gene therapy. Hope this area will soon show its immense potential.

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<div style="margin: 12px 0px; font-family: arial; color: #333333; background: #ffffff; border: solid 4px #e5e5e5; width: 100%; clear: left;"><div class="CM_CTB_Content_Wrap" style="margin: 0px; padding: 0px;background-color: #ffffff;"><div style="border-bottom: solid 1px #dcdcdc; white-space: nowrap; margin-bottom: 8px; background-color: #eeeeee ;background-image: url(http://clipmarks.com/images/source-bg.gif); background-repeat: repeat-x; height: 24px; line-height: 24px; vertical-align: middle; padding-bottom: 4px; color: #666666; font-size: 10px;" ><a href="http://clipmarks.com/clip-to-blog/" title="see clips that are hot right now"><img src="http://content.clipmarks.com/blog_embed/262e6764-43b0-4639-b6e4-c83d6759b210/15506CE2-83CF-46E5-ABD3-0118DA0BE5E4/" alt="" width="19" height="19" border="0" style="vertical-align: middle; margin: 0px 4px; display: inline; border: none; float:none;" /></a>clipped from <a title="http://www.kurzweilai.net/news/news_single.html?id=8542" href="http://www.kurzweilai.net/news/news_single.html?id=8542" style="font-size: 11px;">www.kurzweilai.net</a></div><blockquote style="text-align: left; padding: 0px 8px; margin: 4px 0px 8px 0px; background: transparent; border: none;" cite="http://www.kurzweilai.net/news/news_single.html?id=8542"><DIV>University of Pennsylvania <A class="thought" href="javascript:loadBrain('Research')">research</A>ers and colleagues have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness.
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</blockquote><div style="height: 2px; font-size: 2px; background: #dcdcdc; border-bottom: solid 1px #f5f5f5; margin: 2px 4px;"></div><blockquote style="text-align: left; padding: 0px 8px; margin: 4px 0px 8px 0px; background: transparent; border: none;" cite="http://www.kurzweilai.net/news/news_single.html?id=8542">
The patients have one form of Leber congenital amaurosis (LCA), a group of inherited blinding <A class="thought" href="javascript:loadBrain('Disease')">disease</A>s that damage <A class="thought" href="javascript:loadBrain('Light')">light</A> receptors in the <A class="thought" href="javascript:loadBrain('Retina')">retina</A>. The <A class="thought" href="javascript:loadBrain('Disease')">disease</A>s usually start in childhood and cause complete blindness by the 20's or 30's.</blockquote><div style="height: 2px; font-size: 2px; background: #dcdcdc; border-bottom: solid 1px #f5f5f5; margin: 2px 4px;"></div><blockquote style="text-align: left; padding: 0px 8px; margin: 4px 0px 8px 0px; background: transparent; border: none;" cite="http://www.kurzweilai.net/news/news_single.html?id=8542">
The <A class="thought" href="javascript:loadBrain('Research')">research</A>ers injected a genetically engineered adeno-associated <A class="thought" href="javascript:loadBrain('Virus')">virus</A> carrying a normal version of the gene RPE65 (mutated in the patients' form of LCA) into one eye of each patient.</blockquote><div style="height: 2px; font-size: 2px; background: #dcdcdc; border-bottom: solid 1px #f5f5f5; margin: 2px 4px;"></div><blockquote style="text-align: left; padding: 0px 8px; margin: 4px 0px 8px 0px; background: transparent; border: none;" cite="http://www.kurzweilai.net/news/news_single.html?id=8542"><DIV>
Although the patients didn't achieve normal eyesight, their vision improved from detecting hand movements to reading lines on an eye chart. The LCA gene therapy vector showed no signs of causing inflammation in the <A class="thought" href="javascript:loadBrain('Retina')">retina</A> or other toxic side effects. 
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