Retina-directed gene therapy: Achievements and remaining challenges
Josef Biber; Catharina Gandor; Elvir Bećirović; Stylianos Michalakis · 2025 · Pharmacology & Therapeutics
WASTE classifies this as Negative / Null Result Report · AI classification, approximate
The study found no significant effect — useful as a negative control or null benchmark for your own design.
Abstract
Gene therapy is an innovative medical approach that offers new treatment options for congenital and acquired diseases by transferring, correcting, inactivating or regulating genes to supplement, replace or modify a gene function. The approval of voretigene neparvovec (Luxturna), a gene therapy for RPE65-associated retinopathy, has marked a milestone for the field of retinal gene therapy, but has also helped to accelerate the development of gene therapies for genetic diseases affecting other organs. Voretigene neparvovec is a vector based on adeno-associated virus (AAV) that delivers a function
Abstract by Josef Biber; Catharina Gandor; Elvir Bećirović; Stylianos Michalakis, Pharmacology & Therapeutics (2025) — licensed CC BY 4.0.
About to run something similar?
Run an AI Precheck on your own design to catch failure modes like this one before you spend the time. Your first desk check is free.
Related failures
Power and Predictive Accuracy of Polygenic Risk Scores
Negative / Null Result ReportLocoregional Recurrence After Sentinel Lymph Node Dissection With or Without Axillary Dissection in Patients With Sentinel Lymph Node Metastases
Negative / Null Result ReportCritical aspects of using bacterial cell viability assays with the fluorophores SYTO9 and propidium iodide
Negative / Null Result ReportAlpelisib plus fulvestrant for PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor-2–negative advanced breast cancer: final overall survival results from SOLAR-1
Negative / Null Result ReportA randomized placebo-controlled trial of idebenone in Leber’s hereditary optic neuropathy
Negative / Null Result ReportThe CAFA challenge reports improved protein function prediction and new functional annotations for hundreds of genes through experimental screens
WASTE indexes this work — it does not host or republish it. Failure-type classification is automated and approximate.
Metadata source: OpenAlex · DOI 10.1016/j.pharmthera.2025.108862