據(jù)國(guó)外媒體報(bào)道，來自美國(guó)紐約州布法羅市,、俄亥俄州克利夫蘭市和俄克拉何馬州的科學(xué)家使用基因療法，改善具有視網(wǎng)膜色素變性(retinitis pigmentosa,RP)疾病的老鼠視力。這一研究結(jié)果表明,，科學(xué)家在使盲人恢復(fù)視力的道路上取得了長(zhǎng)足的進(jìn)步。
據(jù)悉,，《美國(guó)實(shí)驗(yàn)生物學(xué)學(xué)會(huì)聯(lián)合會(huì)雜志》（The FASEB Journal）2010年4月刊上發(fā)表的一篇研究報(bào)告中,，科學(xué)家詳細(xì)闡述了利用合成的納米顆粒，改善具有視網(wǎng)膜色素變性(retinitis pigmentosa,RP)疾病老鼠視力的過程,。視網(wǎng)膜色素變性是視網(wǎng)膜光感受器細(xì)胞和色素上皮細(xì)胞變性,，從而導(dǎo)致夜盲和進(jìn)行性視野缺損的一組具有臨床亞型的基因遺傳性致盲眼病。
研究小組成員,，俄克拉何馬州奧克拉荷馬大學(xué)健康科學(xué)中心細(xì)胞生物學(xué)系萊西博士和她的同事一起,，研究了一組帶有視網(wǎng)膜緩慢變性（retinal degeneration slow (Rds) ）基因的老鼠。萊西和她的同事對(duì)這些老鼠進(jìn)行了三種不同類型的治療方法：一種方法是用包含Rds基因的納米顆粒來治療,，一種方法是用正?；騺碇委煟€有一種方法是通過生理鹽水來治療,。
實(shí)施三種不同類型的治療方法后,，研究人員將實(shí)驗(yàn)老鼠和其它具有視網(wǎng)膜色素變性或視網(wǎng)膜緩慢變性疾病老鼠進(jìn)行比較，從而分析得出實(shí)驗(yàn)老鼠視網(wǎng)膜的功能和結(jié)構(gòu),。研究人員發(fā)現(xiàn),，接受納米顆粒基因療法的老鼠，其視覺功能得到改善,，具有明顯愈合的跡象,，而且這種效果到實(shí)驗(yàn)結(jié)束都還保持完好，而接受正?；蚝蜕睇}水治療的老鼠,，其視力不斷下降。上述實(shí)驗(yàn)結(jié)果表明,，納米顆粒是耐受性良好,，并且是安全無副作用的治療方法。
研究人員稱,，他們希望此研究結(jié)果可幫助治愈那些和視網(wǎng)膜色素變性,、遺傳性疾病和后天視網(wǎng)膜疾病等導(dǎo)致失明的疾病。
《美國(guó)實(shí)驗(yàn)生物學(xué)學(xué)會(huì)聯(lián)合會(huì)雜志》雜志主編,，杰拉爾· 德韋斯曼（Gerald Weissmann）說：“使盲人恢復(fù)視力曾經(jīng)被稱為奇跡,。隨著我們對(duì)進(jìn)化、遺傳學(xué)和納米技術(shù)理解的加深,，這種神奇的治療方法將變得非常普遍,。”(生物谷Bioon.com)
Science TM：用基因療法來恢復(fù)視力
Transplantation：胚胎干細(xì)胞移植助患病實(shí)驗(yàn)鼠恢復(fù)視力
Nature：用基因療法治療先天視力缺陷
生物谷推薦原文出處：
The FASEB Journal. doi: 10.1096/fj.09-139147.
Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa
Xue Cai*, Shannon M. Conley*, Zack Nash*, Steven J. Fliesler,,, Mark J. Cooper|| and Muna I. Naash*,1
The purpose of the present study was to test the therapeutic efficiency and safety of compacted-DNA nanoparticle-mediated gene delivery into the subretinal space of a juvenile mouse model of retinitis pigmentosa. Nanoparticles containing the mouse opsin promoter and wild-type mouse Rds gene were injected subretinally into mice carrying a haploinsufficiency mutation in the retinal degeneration slow (rds+/–) gene at postnatal day (P)5 and 22. Control mice were either injected with saline, injected with uncompacted naked plasmid DNA carrying the Rds gene, or remained untreated. Rds mRNA levels peaked at postinjection day 2 to 7 (PI-2 to PI-7) for P5 injections, stabilized at levels 2-fold higher than in uninjected controls for both P5 and P22 injections, and remained elevated at the latest time point examined (PI-120). Rod function (measured by electroretinography) showed modest but statistically significant improvement compared with controls after both P5 and P22 injections. Cone function in nanoparticle-injected eyes reached wild-type levels for both ages of injections, indicating full prevention of cone degeneration. Ultrastructural examination at PI-120 revealed significant improvement in outer segment structures in P5 nanoparticle-injected eyes, while P22 injection had a modest structural improvement. There was no evidence of macrophage activation or induction of IL-6 or TNF- mRNA in P5 or P22 nanoparticle-dosed eyes at either PI-2 or PI-30. Thus, compacted-DNA nanoparticles can efficiently and safely drive gene expression in both mitotic and postmitotic photoreceptors and retard degeneration in this model. These findings, using a clinically relevant treatment paradigm, illustrate the potential for application of nanoparticle-based gene replacement therapy for treatment of human retinal degenerations.—Cai, X., Conley, S. M., Nash, Z., Fliesler, S. J., Cooper, M. J., Naash, M. I. Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa.
