Matches in SemOpenAlex for { <https://semopenalex.org/work/W4313473368> ?p ?o ?g. }
- W4313473368 endingPage "103" @default.
- W4313473368 startingPage "103" @default.
- W4313473368 abstract "Leber congenital amaurosis caused by mutations in the RPE65 gene belongs to the most severe early-onset hereditary childhood retinopathies naturally progressing to legal blindness. The novel gene therapy voretigene neparvovec is the first approved causative treatment option for this devastating eye disease and is specifically designed to treat RPE65-mediated retinal dystrophies. Herein, we present a follow-up of the youngest treated patients in Germany so far, including four pre-school children who received treatment with voretigene neparvovec at a single treatment center between January 2020 and May 2022. All patients underwent pars plana vitrectomy with circumferential peeling of the internal limiting membrane at the injection site and subretinal injection of voretigene neparvovec. Pre- and postoperative diagnostics included imaging (spectral domain optical coherence tomography, fundus autofluorescence, fundus wide-angle imaging), electrophysiologic examination (ERG), retinal light sensitivity measurements (FST) and visual acuity testing. Behavioral changes were assessed using a questionnaire and by observing the children's vision-guided behavior in different levels of illumination. All children showed marked increase in vision-guided behavior shortly after therapy, as well as marked increase in visual acuity in the postoperative course up to full visual acuity in one child. Two eyes showed partial electrophysiological recovery of an ERG that was undetectable before treatment-a finding that has not been described in humans before." @default.
- W4313473368 created "2023-01-06" @default.
- W4313473368 creator A5008689260 @default.
- W4313473368 creator A5009038224 @default.
- W4313473368 creator A5013075360 @default.
- W4313473368 creator A5013230086 @default.
- W4313473368 creator A5021803362 @default.
- W4313473368 creator A5037238357 @default.
- W4313473368 creator A5041342462 @default.
- W4313473368 creator A5057543594 @default.
- W4313473368 creator A5070419882 @default.
- W4313473368 creator A5083191480 @default.
- W4313473368 date "2022-12-30" @default.
- W4313473368 modified "2023-10-13" @default.
- W4313473368 title "Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis" @default.
- W4313473368 cites W1543729365 @default.
- W4313473368 cites W1963846033 @default.
- W4313473368 cites W1981432109 @default.
- W4313473368 cites W1995949600 @default.
- W4313473368 cites W1996435602 @default.
- W4313473368 cites W1996875707 @default.
- W4313473368 cites W2002818751 @default.
- W4313473368 cites W2002914110 @default.
- W4313473368 cites W2005510817 @default.
- W4313473368 cites W2023962213 @default.
- W4313473368 cites W2031824541 @default.
- W4313473368 cites W2036996443 @default.
- W4313473368 cites W2047747556 @default.
- W4313473368 cites W2057737605 @default.
- W4313473368 cites W2068039220 @default.
- W4313473368 cites W2069708962 @default.
- W4313473368 cites W2080258263 @default.
- W4313473368 cites W2082733587 @default.
- W4313473368 cites W2083993535 @default.
- W4313473368 cites W2087518452 @default.
- W4313473368 cites W2096060458 @default.
- W4313473368 cites W2100522480 @default.
- W4313473368 cites W2106221931 @default.
- W4313473368 cites W2109599255 @default.
- W4313473368 cites W2110283302 @default.
- W4313473368 cites W2132919883 @default.
- W4313473368 cites W2152853777 @default.
- W4313473368 cites W2162290849 @default.
- W4313473368 cites W2339001755 @default.
- W4313473368 cites W2368623011 @default.
- W4313473368 cites W265142245 @default.
- W4313473368 cites W2729411160 @default.
- W4313473368 cites W2736065260 @default.
- W4313473368 cites W2756078072 @default.
- W4313473368 cites W2894534989 @default.
- W4313473368 cites W2902094233 @default.
- W4313473368 cites W3005260758 @default.
- W4313473368 cites W3009940848 @default.
- W4313473368 cites W3014227573 @default.
- W4313473368 cites W3016488212 @default.
- W4313473368 cites W3148863837 @default.
- W4313473368 cites W3149032887 @default.
- W4313473368 cites W3177509185 @default.
- W4313473368 cites W4200390871 @default.
- W4313473368 cites W4206925733 @default.
- W4313473368 cites W4281382958 @default.
- W4313473368 cites W4303579288 @default.
- W4313473368 cites W4306983479 @default.
- W4313473368 cites W57491276 @default.
- W4313473368 doi "https://doi.org/10.3390/biomedicines11010103" @default.
- W4313473368 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/36672611" @default.
- W4313473368 hasPublicationYear "2022" @default.
- W4313473368 type Work @default.
- W4313473368 citedByCount "4" @default.
- W4313473368 countsByYear W43134733682023 @default.
- W4313473368 crossrefType "journal-article" @default.
- W4313473368 hasAuthorship W4313473368A5008689260 @default.
- W4313473368 hasAuthorship W4313473368A5009038224 @default.
- W4313473368 hasAuthorship W4313473368A5013075360 @default.
- W4313473368 hasAuthorship W4313473368A5013230086 @default.
- W4313473368 hasAuthorship W4313473368A5021803362 @default.
- W4313473368 hasAuthorship W4313473368A5037238357 @default.
- W4313473368 hasAuthorship W4313473368A5041342462 @default.
- W4313473368 hasAuthorship W4313473368A5057543594 @default.
- W4313473368 hasAuthorship W4313473368A5070419882 @default.
- W4313473368 hasAuthorship W4313473368A5083191480 @default.
- W4313473368 hasBestOaLocation W43134733681 @default.
- W4313473368 hasConcept C103796816 @default.
- W4313473368 hasConcept C118487528 @default.
- W4313473368 hasConcept C15744967 @default.
- W4313473368 hasConcept C169760540 @default.
- W4313473368 hasConcept C2776391266 @default.
- W4313473368 hasConcept C2777093970 @default.
- W4313473368 hasConcept C2778024200 @default.
- W4313473368 hasConcept C2778257484 @default.
- W4313473368 hasConcept C2779113765 @default.
- W4313473368 hasConcept C2780724529 @default.
- W4313473368 hasConcept C2780827179 @default.
- W4313473368 hasConcept C2781040256 @default.
- W4313473368 hasConcept C71924100 @default.
- W4313473368 hasConceptScore W4313473368C103796816 @default.
- W4313473368 hasConceptScore W4313473368C118487528 @default.
- W4313473368 hasConceptScore W4313473368C15744967 @default.