Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells

Small and cell-type restricted promoters are important tools for basic and preclinical research, and clinical delivery of gene therapies. In clinical gene therapy, ophthalmic trials have been leading the field, with over 50% of ocular clinical trials using promoters that restrict expression based on...

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Bibliographic Details
Published in:	Gene therapy Vol. 28; no. 6; pp. 351 - 372
Main Authors:	Korecki, Andrea J., Cueva-Vargas, Jorge L., Fornes, Oriol, Agostinone, Jessica, Farkas, Rachelle A., Hickmott, Jack W., Lam, Siu Ling, Mathelier, Anthony, Zhou, Michelle, Wasserman, Wyeth W., Di Polo, Adriana, Simpson, Elizabeth M.
Format:	Journal Article
Language:	English
Published:	London Nature Publishing Group UK 01-06-2021 Nature Publishing Group Stockton Press
Subjects:	14/35 14/63 42/44 631/1647/2017 631/61/201 64/60 692/699/375 Animals Biomedical and Life Sciences Biomedicine Cell Biology Clinical trials Cornea Deoxyribonucleic acid Dependoviruses DNA Endothelial Cells Gene Expression Gene Therapy Genetic aspects Genetic engineering Genetic research Human Genetics Humans Intravenous administration Methods Mice Mueller cells Nanotechnology Neonates Neuroglia Pax6 protein PAX6 Transcription Factor - genetics Promoter Regions, Genetic Promoters Promoters (Genetics) Retina Retinal Cone Photoreceptor Cells Stroma Canada
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