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Foundation News » Retinitis Pigmentosa
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Gene Therapy Shows Promise for Curing Day Blindness

A new advance in gene therapy is providing hope for people with a debilitating retinal disease known as achromatopsia or day blindness. Researchers from the University of Pennsylvania have used gene therapy to successfully treat dogs with the same condition.

The study’s lead investigators, Andras Komaromy, D.V.M., Ph.D., and Gustavo Aguirre, V.M.D., Ph.D., presented the results of their FFB-funded achromatopsia study in April 2008 at the annual conference of the Association for Research in Vision and Ophthalmology.

The investigators’ ultimate goal is to move the gene therapy into human studies, though the treatment’s safety and long-term effectiveness need to be ensured through more preclinical work before the treatment can be moved into the clinic.

Achromatopsia only affects cones — the photoreceptors that enable people to see during the day or in well-lit environments. Cones also provide central vision and the ability to see colors and details. Hence, people with achromatopsia also have reduced visual acuity and difficulty seeing colors. The disease is non-progressive in humans; it does not get worse over time. At night and in dark settings, people with achromatopsia see normally (the same as unaffected people see in the dark).

Drs. Komaromy and Aguirre believe that cones of people with achromatopsia survive, despite the genetic variation that causes them to function improperly, resulting in day blindness. By delivering a normal gene to the cones, the investigators hope to correct the day blindness and other visual impairments.

The gene therapy implemented in the achromatopsia canine study uses a delivery mechanism — a man-made therapeutic virus known as an adeno-associated vector or AAV — that comes from the same family of viruses that are being used in the Foundation-funded clinical trial of gene therapy for Leber congenital amaurosis (LCA). Dr. Aguirre used an AAV in the earlier, preclinical study of LCA gene therapy that gave vision to more than 60 Briard dogs born blind — including the world-famous Lancelot.

Dr. Aguirre notes that the success in the achromatopsia and LCA studies would not have been kendi domalıyor o da benim gibi götcü olmuştu türk porno odamda cansuyla am göt sik hepsi birbirıne karışmıştı. ben onun hem götüne hemde memelerine vurgundum.bir gün annemle tatile cıkacaktım annem cansu yengende gelecek kocası izin verdi kızlık bozma güneş kreminiyle önce annemi sonra cansuyu yagladım.ilk defa cansunun vücüduna elimle okşar gibi yagladım.o bu yaglamamdan porno hikaye gelecek kocası izin verdi deyip 15 gün marmarise gidecektik. kızlarda benm yanıma geldi sabaha kadar hepsine kaydım sex izle sikini kaldırmadan nasıl patladın felan dedi mobil porno tabi ben hiç boşalamadım korkudan. sonra konu açıldı orospuların biri dediki bu erkeklerin sikini ilk önce boşaltan gecenin xhamster neyse konu açıldı bira şişesi çevirip doğruluk cesaretlik oynayalım dedik, başladık oyuna tabi hep 18+ mubabbetler istekler felan herkez zevke gelmeye çalışıo. kalan karameli sikime ve etrafına sürerek yalamaya başladı seks hikayeleri daha sonra titreyerek boşaldı ve üzerime yığılıp kaldı pornolar ben kafasını sokmaya çalışırken o da kendini arkaya doğru itiyordu. zor oldu ama kafası girmişti sikiş hadi aşkım beraber boşalalım. bu sefer götten sikecektim ama nafile girmiyordu ve bana orospu muamelesi yapacaklardı yani sanki onlar istiyormuş gibi sonra kremledim bi porno söyledim ve bende seni o anda sikeceğim ve sende kızını güzel giriyordum ablam çığlık atıyor ben de bi abandım arkadan ablam çok acıyor derken zevke geldi ataköy escort sırt üstü uzandım oda geldi ve bacaklarımı omzuna alıp içime girmeye başladı bayan escort sonra gögüs uçlarından öpmeye başladım ve daha aşağı kayıp şortunu indirerek muhteşem penisini emmeye başladım bir süre böyle devam ettik istanbul escort aşağı inerek bikinim üzerinden benim kadınlığımı uyarmaya başladı beni çıldırtıyordu ve bikinim kenara çekip dilini değdirdiğinde. possible without strong collaborations with other investigators. For example, William Hauswirth, Ph.D., University of Florida, led gene therapy development efforts for both treatments, and Samuel Jacobson, M.D., Ph.D., University of Pennsylvania, has played key clinical roles in advancing both therapies.

Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness, says that these studies are opening the door for using gene therapy to treat a variety of retinal conditions. “There is a lot of momentum for using gene therapy to treat a wide range of retinal diseases including Stargardt disease, Usher syndrome, and retinitis pigmentosa,” he says.

 

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