Characterization and AAV-Mediated CRB Gene Augmentation in Human Derived CRB1KO and CRB1KOCRB2+/- Retinal Organoids

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CRB1KO and CRB1KOCRB2+/- differentiation day 210 retinal organoids showed a significant decrease in the number of photoreceptor nuclei in a row and a significant increase in the number of photoreceptor cell nuclei above the outer limiting membrane.
[Molecular Therapy-Methods & Clinical Development]
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