Department of Ophthalmology, Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida.
Retina. 2023 Sep 1;43(9):1608-1611. doi: 10.1097/IAE.0000000000003085.
To report surgical observations formulated during the first 120 cases of subretinal gene therapy in patients with inherited retinal degenerations (IRDs).
A two-surgeon team compiled surgical observations and formulated surgical pearls based on the consecutive cases of subretinal viral vector injection in patients enrolled in clinical trials focusing on choroideremia, achromatopsia, and RP GTPase regulator associated retinitis pigmentosa, as well as patients with retinal pigment epithelium-specific-65-kDa (RPE65) associated Leber congenital amaurosis receiving Food and Drug Administration-approved voretigene neparvovec-rzyl therapy.
One hundred twenty subretinal surgeries were performed by a two-surgeon team. Key anatomical features pertinent to surgical management were noted and are described in this article. Surgical decision making for successful subretinal administration of viral vectors and management of potential surgical challenges were formulated.
Lessons learned during subretinal gene therapy cases may be helpful to other surgeons entering clinical trials or performing postapproval gene therapy administration. Surgical pearls outlined in this article may also be helpful for other targeted subretinal therapies, such as cellular transplantation or retinal prosthesis implantation.
报告在遗传性视网膜疾病（IRDs）患者的视网膜下基因治疗的前 120 例中所制定的手术观察结果。
由两位外科医生团队根据在临床试验中接受脉络膜视网膜色素变性、色盲和 RP GTPase 调节剂相关视网膜色素变性以及接受食品和药物管理局批准的 voretigene neparvovec-rzyl 治疗的视网膜色素上皮特异性 65-kDa（RPE65）相关莱伯先天性黑矇患者的视网膜下病毒载体注射连续病例，编译手术观察结果并制定手术要点。
由两位外科医生团队进行了 120 例视网膜下手术。本文描述了与手术管理相关的关键解剖特征。制定了成功进行视网膜下病毒载体给药的手术决策以及管理潜在手术挑战的方案。
在视网膜下基因治疗病例中获得的经验教训可能有助于其他进入临床试验或进行批准后基因治疗管理的外科医生。本文中概述的手术要点也可能对其他靶向性视网膜下治疗，如细胞移植或视网膜假体植入有帮助。
