Human Pluripotent Stem Cell-Derived Retinal Organoids: A Viable Platform for Investigating the Efficacy of Adeno-Associated Virus Gene Therapy
Author:
Affiliation:
1. Stem Cell Convergence Research Center, Korea Research Institute of Bioscience and Biotechnology (KRIBB), Daejeon, Korea
2. KRIBB School of Bioscience, Korea University of Science & Technology (UST), Daejeon, Korea
Funder
Korea Research Institute of Bioscience and Biotechnology
National Research Foundation of Korea
Publisher
Korean Society for Stem Cell Research
Link
https://pdf.medrang.co.kr/Ijsc2/2024/017/Ijsc017-02-11.pdf
Reference23 articles.
1. HumanRPE65Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year
2. Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery
3. Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa
4. Adeno-associated virus vector as a platform for gene therapy delivery
5. Self-Formation of Optic Cups and Storable Stratified Neural Retina from Human ESCs
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