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Review
. 2022 May 9;12(5):708.
doi: 10.3390/life12050708.

Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review

Giacomo Maria Bacci  1 , Valentina Becherucci  2 , Elisa Marziali  1 , Andrea Sodi  3 , Franco Bambi  2 , Roberto Caputo  1
Affiliations
Review

Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review

Giacomo Maria Bacci et al. Life (Basel). .

Abstract

Inherited retinal dystrophies and retinal degenerations related to more common diseases (i.e., age-related macular dystrophy) are a major issue and one of the main causes of low vision in pediatric and elderly age groups. Advancement and understanding in molecular biology and the possibilities raised by gene-editing techniques opened a new era for clinicians and patients due to feasible possibilities of treating disabling diseases and the reduction in their complications burden. The scope of this review is to focus on the state-of-the-art in somatic cell therapy medicinal products as the basis of new insights and possibilities to use this approach to treat rare eye diseases.

Keywords: Stargardt disease; advanced therapy medicinal products; embryonic stem cells; good manufacturing practices; induced pluripotent stem cells; inherited retinal dystrophies; mesenchymal stromal cells; retinal organoids; retinal progenitor cells; retinitis pigmentosa; somatic cell therapy medicinal products.

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Conflict of interest statement

The authors declare no conflict of interest.

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