Ku Cristy A, Pennesi Mark E
Center for Neuroscience, Robert C. Byrd Health Sciences Center, West Virginia University, Morgantown, West Virginia, 26505, USA.
Casey Eye Institute, Oregon Health & Science University, Portland, OR, 97239, USA.
Expert Rev Ophthalmol. 2015 Jun;10(3):281-299. doi: 10.1586/17469899.2015.1035711. Epub 2015 Apr 10.
Clinical trials treating inherited retinal dystrophy caused by mutations had put retinal gene therapy at the forefront of gene therapy. Both successes and limitations in these clinical trials have fueled developments in gene vectors, which continue to further advance the field. These novel gene vectors aim to more safely and efficiently transduce retinal cells, expand the gene packaging capacity of AAV, and utilize new strategies to correct the varying mechanisms of dysfunction found with inherited retinal dystrophies. With recent clinical trials and numerous pre-clinical studies utilizing these novel vectors, the future of ocular gene therapy continues to hold vast potential.
治疗由突变引起的遗传性视网膜营养不良的临床试验已使视网膜基因治疗处于基因治疗的前沿。这些临床试验中的成功与局限推动了基因载体的发展,基因载体的发展又继续推动该领域的进一步进步。这些新型基因载体旨在更安全、高效地转导视网膜细胞,扩大腺相关病毒(AAV)的基因包装能力,并采用新策略来纠正遗传性视网膜营养不良中发现的各种功能障碍机制。随着近期利用这些新型载体开展的临床试验和大量临床前研究,眼部基因治疗的未来仍具有巨大潜力。