See the Light
New advances in regenerative medicine bring hope to patients suffering from blinding disorders of the outer retina.
Steve Charles | | Quick Read
Landmark literature: YH Jung et al., “3D Microstructured Scaffolds to Support Photoreceptor Polarization and Maturation”, Advanced Materials, 30 (2018). DOI: 201803550
Regenerative medicine is a promising and relatively new area of research inclusive of both gene therapy and cell-based approaches. There are many misconceptions regarding regenerative medicine approaches for retinal disorders; gene therapy should be thought of principally as a prevention scheme dependent on early intervention before significant cell loss. Gene therapy cannot produce visual improvement with the exception of the modest visual improvement observed with Luxturna (voretigene neparvovec-rzyl) for RPE 65 type of Leber Congenital Amarosis and RPE 65 subtype retinitis pigmentosa. On the other hand, cell-based therapies do have the potential to restore function. But intravitreal, subretinal stem cell or retinal progenitor cell injection cannot produce highly organized retinal architecture. Stem cells must be converted to RPE cells for geographic atrophy associated with AMD or to photoreceptors for inherited retinal disorders or possibly chronic retinal detachment treatment. The conversion process is complex, takes over 100 days, and has many potential pitfalls.
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