Casey Eye Institute, Department of Ophthalmology, Oregon Health & Science University, Portland, OR, USA.
Department of Ophthalmology, Mayo Clinic, Rochester, MN, USA.
Gene Ther. 2022 Nov;29(10-11):601-607. doi: 10.1038/s41434-021-00294-2. Epub 2021 Sep 28.
Retinal damage has been associated with increased injection pressure during subretinal gene therapy delivery in various animal models, yet there are no human clinical data regarding the pressures required to initiate and propagate subretinal blebs. This study characterized the intraoperative pressure levels for subretinal gene therapy delivery across eight retinal conditions. A total of 116 patients with retinal degenerative diseases have been treated with subretinal gene therapy at OHSU-Casey Eye Institute as of June 2020; seventy patients (60.3%) were treated using a pneumatic-assisted subretinal delivery system. All retinal blebs were performed using a 41-gauge injection cannula, and use of a balanced salt solution (BSS) "pre-bleb" prior to gene therapy delivery was performed at the discretion of the surgeon. Patient age and intraoperative data for BSS and vector injections were analyzed in a masked fashion for all patients who received pneumatic-assisted subretinal gene therapy. The median age of the patients was 35 years (range 4-70). No significant differences in injection pressures were found across the eight retinal conditions. In this study, patient age was shown to affect maximum injection pressures required for bleb propagation, and the relationship between age and pressure varied based on retinal condition. These data have important implications in optimizing surgical protocols for subretinal injections.
视网膜损伤与各种动物模型中视网膜下基因治疗传递时的注射压力增加有关,但尚无关于引发和传播视网膜下气泡所需压力的人类临床数据。本研究通过八项视网膜疾病特征描述了视网膜下基因治疗传递的术中压力水平。截至 2020 年 6 月,OHSU-Casey Eye Institute 已经对 116 名患有视网膜退行性疾病的患者进行了视网膜下基因治疗,其中 70 名患者(60.3%)接受了气动辅助视网膜下传递系统的治疗。所有视网膜下气泡均采用 41 号注射套管进行,基因治疗传递前是否使用平衡盐溶液(BSS)“预气泡”由外科医生自行决定。对所有接受气动辅助视网膜下基因治疗的患者进行了 BSS 和载体注射的术中数据进行了盲法分析。患者的中位年龄为 35 岁(范围 4-70 岁)。在八项视网膜疾病中,注射压力没有显著差异。在这项研究中,患者年龄被证明会影响气泡传播所需的最大注射压力,并且年龄与压力之间的关系因视网膜疾病而异。这些数据对优化视网膜下注射的手术方案具有重要意义。