Bhattacharya Sujoy, Gangaraju Rajashekhar, Chaum Edward
Departments of Ophthalmology, and Anatomy and Neurobiology, University of Tennessee Health Science Center, Memphis, TN 38163, USA.
Curr Mol Biol Rep. 2017 Sep;3(3):172-182. doi: 10.1007/s40610-017-0069-3. Epub 2017 Jul 10.
Progress in stem cell research for blinding diseases over the past decade is now being applied to patients with retinal degenerative diseases and soon perhaps, glaucoma. However, the field still has much to learn about the conversion of stem cells into various retinal cell types, and the potential delivery methods that will be required to optimize the clinical efficacy of stem cells delivered into the eye.
Recent groundbreaking human clinical trials have demonstrated both the opportunities and current limitations of stem cell transplantation for retinal diseases. New progress in developing retinal organoids, coupled with the maturation of bio-printing technology, and non-invasive high-resolution imaging have created new possibilities for repairing and regenerating the diseased retina and rigorously validating its clinical impact .
While promising progress is being made, meticulous clinical trials with cells derived using good manufacturing practice, novel surgical methods, and improved methods to derive all of the neuronal cell types present in the retina will be indispensable for developing stem cell transplantation as a paradigm shift for the treatment of blinding diseases.
过去十年间,干细胞研究在致盲性疾病方面取得的进展如今正应用于视网膜退行性疾病患者,或许不久后还会应用于青光眼患者。然而,在将干细胞转化为各种视网膜细胞类型以及为优化眼部干细胞临床疗效所需的潜在递送方法方面,该领域仍有许多需要了解的地方。
近期具有开创性的人体临床试验已证明了干细胞移植治疗视网膜疾病的机遇和当前局限性。视网膜类器官开发方面的新进展,加上生物打印技术的成熟以及非侵入性高分辨率成像技术,为修复和再生患病视网膜以及严格验证其临床影响创造了新的可能性。
虽然正在取得有前景的进展,但对于将干细胞移植发展成为治疗致盲性疾病的范式转变而言,使用良好生产规范衍生的细胞进行细致的临床试验、新颖的手术方法以及改进的方法来衍生视网膜中所有神经元细胞类型将是不可或缺的。