这里就楼主提到的RHO基因在网上继续找到了一点资料供大家参考。
“Genable Technologies Ltd. is a privately held, venture backed, Dublin (Ireland) based bio-pharmaceutical company developing new gene medicines to treat "dominant" genetic diseases.
Genable utilizes well-established, clinically safe & effective AAV vectors to obtain expression of RNA interference (RNAi) molecules which suppress the expression of both the faulty and normal gene copies and replaces this with a gene subtly altered to become refractory to suppression but still encoding a normal wild type protein.
The combination of suppression and replacement (S&R) overcomes the significant hurdle in dominant disease of mutation variability by eliminating the need to target specific mutations in a wide range of disorders. Genable's technology is protected by a broad suite of granted patents and patent applications in the USA, EU and worldwide.
Genable's first gene medicine - GT038 - is for treatment of patients with rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP) - a debilitating form of inherited blindness resulting from a diverse array of mutations in the RHO gene.”
Genable科技有限公司是一家私人持股，风险抵押，都柏林（爱尔兰）为基础的生物制药公司，开发新的基因药物来治疗“显性”遗传病。
Genable采用行之有效的，临床上安全和有效的AAV载体，得到的RNA干扰（RNAi）分子，其抑制二者的故障和正常基因拷贝的表达的表达，并取代本与基因巧妙地改变以变得难治性抑制，但仍编码一个正常的野生型蛋白。
抑制和替代（ S＆R ）的结合，克服了突变变异的显性疾病的显著障碍者无需针对特定的突变在多种疾病。 Genable的技术是通过广泛的在美国，欧盟和世界各地的授权专利和专利应用套件的保护。
Genable的第一个基因药物 - GT038 - 是治疗患者的视紫红质（ RHO ）联常染色体显性遗传视网膜色素变性（ ADRP ） - 遗传性失明的突变在RHO基因的多样化引起的一种使人衰弱的形式。
