Molecular aspects of medicineHumansGenetic TherapyDependovirusGenetic VectorsRetinal Dystrophies
Precision gene therapy: Tailoring rAAV-mediated gene therapies for inherited retinal dystrophies (IRDs).
Fábio Duarte, Yvan Arsenijevic
Published: 202510.1016/j.mam.2025.101424
Abstract
Inherited retinal dystrophies (IRDs) are a heterogeneous group of rare genetic disorders that affect multiple retinal cell types, including photoreceptors, Müller glia, bipolar cells, retinal ganglion cells (RGCs), and the retinal pigment epithelium…
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