RETINA SPECIFIC GENE THERAPY WITH MODIFIED LIPOSOMES

目前还没有有效的治疗方法来阻止癌症的进展。 视网膜色素变性及相关遗传性视网膜疾病。 部分致病基因的发现和克隆 这些疾病带来了基因替换的可能性，或者 体细胞基因疗法。在获奖期间，我打算学习 细胞和分子生物学的基本技术 专注于体细胞基因的独立医生-科学家研究员 治疗技术。为此，埃默里大学 眼科，已经同意为我提供一个理想的环境 通过结合说教、苏格拉底和 实践经验。由于我的临床训练，我有一家公司 对本病的病理生理学、内外科治疗有一定的了解 很多视网膜疾病。有约翰·尼克森博士做我的导师，我会 通过参加研究生级别的课程开始第一阶段的培训 旨在学习基本的生物学原理，这是 从细胞和分子的角度理解视网膜。此外, 在第一阶段，我将开始实验室工作，开发一种新的基因 一种利用生物活性脂质体的递送系统 在非眼组织中显示，显著提高了转染率 体外和体内与其他转导系统的比较。这些 脂质体将被进一步修改，以特异性地针对 光感受器细胞(特效目标1)。在第二阶段的培训中 在此期间，我将更加独立地工作，继续在Dr。 Nickerson说，为了在小鼠动物身上进一步开发这种DNA载体系统 模式(具体目标2)。最后，我将把这个基因载体系统应用于 一种治疗遗传性视网膜变性(特异性)小鼠的尝试 目标3)。

Currently there are no effective treatments that arrest the progression of retinitis pigmentosa and related disabling inherited retinal diseases. The discovery and cloning of the defective genes responsible for some of these diseases has given rise to the possibility of gene replacement, or somatic gene therapy. During the period of this award I intend to learn the fundamental cell and molecular biological techniques to become an independent physician-scientist investigator concentrating in somatic gene therapy techniques. To this end, The Emory University, Department of ophthalmology, has agreed to provide an ideal environment for me to achieve this goal through a combination of didactic, socratic, and practical experiences. As a result of my clinical training, I have a firm understanding of the pathophysiology, medical and surgical treatments of many retinal diseases. With John Nickerson, Ph.D., as my mentor, I will begin Phase I of the training period by attending graduate level classes directed at learning fundamental biological principles necessary to understand the retina on a cellular and molecular basis. In addition, during Phase I, I will begin laboratory work developing a novel gene delivery system that utilizes biologically active liposomes that have been shown in non-ocular tissue to dramatically increase transfection rates in vitro and in vivo as compared to other transfection systems. These liposomes will be modified further to target them specifically to the photoreceptor cell (Specific Aim #1). During Phase II of the training period, I will work more independently, remaining under the auspices Dr. Nickerson, to further develop this DNA vector system in a mouse animal model (Specific Aim #2). Finally, I will apply this gene vector system in an attempt to treat mice with inherited retinal degenerations (Specific Aim #3).