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Article

The Road towards Gene Therapy for X-Linked Juvenile Retinoschisis: A Systematic Review of Preclinical Gene Therapy in Cell-Based and Rodent Models of XLRS.

International journal of molecular sciences - 19 Jan 2024

van der Veen Isa, Heredero Berzal Andrea, Koster Céline, Ten Asbroek Anneloor L M A, Bergen Arthur A, Boon Camiel J F

Abstract excerpt

X-linked juvenile retinoschisis (XLRS) is an early-onset progressive inherited retinopathy affecting males. It is characterized by abnormalities in the macula, with formation of cystoid retinal cavities, frequently accompanied by splitting of the retinal layers, impaired synaptic transmission of visual signals, and associated loss of visual acuity. XLRS is caused by loss-of-function mutations in the retinoschisin...

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Identifiers

PMID: 38279267
DOI: 10.3390/ijms25021267
PMCID: PMC10816913

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