Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis
Article
Gerhardt, Maximilian J., Priglinger, Claudia S.
, Rudolph, Günther, Hufendiek, Karsten , Framme, Carsten, Jägle, Herbert, Salchow, Daniel J., Anschütz, Andreas, Michalakis, Stylianos and Priglinger, Siegfried G.
(2022)
Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis.
Biomedicines 11 (1), p. 103.
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| Item type | Article | ||||
| Journal or Publication Title | Biomedicines | ||||
| Publisher | MDPI | ||||
| Place of Publication | BASEL | ||||
| Volume | 11 | ||||
| Number of Issue or Book Chapter | 1 | ||||
| Page Range | p. 103 | ||||
| Date | 2022 | ||||
| Date of publication | 18 Mar 2025 10:09 | ||||
| Institutions | Medicine > Lehrstuhl für Augenheilkunde | ||||
| Identification Number |
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| Keywords | RECESSIVE RETINITIS-PIGMENTOSA; RETINAL DYSTROPHY; VISUAL FUNCTION; RPE65; MUTATIONS; CHILDHOOD; MODEL; CONE; EYE; ISOMERASE; gene therapy; voretigene neparvovec; RPE65; leber congenital amaurosis; early-onset retinal dystrophy; electrophysiology; vision improvement; gene therapy in children | ||||
| Dewey Decimal Classification | 600 Technology > 610 Medical sciences Medicine | ||||
| Status | Published | ||||
| Refereed | Yes, this version has been refereed | ||||
| Created at the University of Regensburg | Yes | ||||
| Item ID | 76077 |
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