Research graph
References from <i>USH2A</i> High-Capacity Adenoviral Vector Restores usherin, whirlin, and ADGRV1 in Photoreceptor Cells of Retinal Organoids. Local targets link to admitted publications; unresolved targets remain external evidence.
10.3389/fnins.2020.00838
10.3389/fnins.2020.00838 · External reference
10.3389/fnagi.2022.948279
10.3389/fnagi.2022.948279 · External reference
10.1016/j.exer.2020.108330
10.1016/j.exer.2020.108330 · External reference
10.3390/genes10121015
10.3390/genes10121015 · External reference
PO-0675 Prevalence Of Mutation C.11864g &gt;a (p. Trp3955x) In The Ush2a Gene In Patients With Usher Ii Syndrome From Volgaural Region Of Russia
2014 · External reference
The usherin mutation c.2299delG leads to its mislocalization and disrupts interactions with whirlin and VLGR1
10.1038/s41467-023-36431-1 · 2023 · External reference
Expression of the human usherin c.2299delG mutation leads to early-onset auditory loss and stereocilia disorganization
10.1038/s42003-023-05296-x · 2023 · External reference
10.1101/2020.02.04.934240
10.1101/2020.02.04.934240 · External reference
USH2A-Mutated Human Retinal Organoids Model Rod-Cone Dystrophy
10.1167/iovs.66.14.2 · 2025 · External reference
USH2A variants causing retinitis pigmentosa or Usher syndrome provoke differential retinal phenotypes in disease-specific organoids
2023 · External reference
10.3390/ijms27041636
10.3390/ijms27041636 · External reference
10.3390/ijms231911669
10.3390/ijms231911669 · External reference
Systematic empirical evaluation of individual base editing targets: Validating therapeutic targets in USH2A and comparison of methods
10.1016/j.ymthe.2025.01.042 · 2025 · External reference
10.1128/jvi.78.1.9-22.2004
10.1128/jvi.78.1.9-22.2004 · External reference
Long-term transgene expression in the RPE after gene transfer with a high-capacity adenoviral vector
2002 · External reference
10.3390/cells9040953
10.3390/cells9040953 · External reference
10.3390/ijms21103643
10.3390/ijms21103643 · External reference
10.1073/pnas.202300099
10.1073/pnas.202300099 · External reference
10.1038/mt.2008.23
10.1038/mt.2008.23 · External reference
Subretinal gene delivery using helper-dependent adenoviral vectors
10.1186/2045-3701-1-15 · 2011 · External reference
Retinal transduction profiles by high-capacity viral vectors
10.1038/gt.2014.57 · 2014 · External reference
10.3390/ijms26010055
10.3390/ijms26010055 · External reference
Human iPSC-Derived Retinas Recapitulate the Fetal CRB1 CRB2 Complex Formation and Demonstrate that Photoreceptors and Muller Glia Are Targets of AAV5
10.1016/j.stemcr.2019.03.002 · 2019 · External reference
10.1089/hum.2019.159
10.1089/hum.2019.159 · External reference
10.1016/j.ymthe.2025.04.045
10.1016/j.ymthe.2025.04.045 · External reference
Adeno-associated virus as a delivery vector for gene therapy of human diseases
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10.1089/hum.2019.220
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10.1038/s41467-023-42386-0
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AAVLINK: A potent DNA-recombination method for large cargo delivery in gene therapy
10.1016/j.cell.2025.12.039 · 2026 · External reference
10.1038/sj.gt.3302612
10.1038/sj.gt.3302612 · External reference
Highly efficient retinal gene delivery with helper-dependent adenoviral vectors
10.1016/j.gendis.2014.09.002 · 2014 · External reference
10.1172/jci.insight.174179
10.1172/jci.insight.174179 · External reference
Successful large gene augmentation of USH2A with non-viral episomal vectors
10.1016/j.ymthe.2023.06.012 · 2023 · External reference
10.3390/ijms22052318
10.3390/ijms22052318 · External reference
10.1038/s12276-018-0196-9
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10.1167/iovs.10-6141
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10.1167/iovs.07-0257
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10.1016/j.ymthe.2017.05.004
10.1016/j.ymthe.2017.05.004 · External reference
10.1056/nejmoa2309915
10.1056/nejmoa2309915 · External reference
10.1111/jcmm.13504
10.1111/jcmm.13504 · External reference
Challenges and Prospects for Helper-Dependent Adenoviral Vector-Mediated Gene Therapy
10.3390/biomedicines2020132 · 2014 · External reference
10.3390/v15091801
10.3390/v15091801 · External reference
Retargeting adenoviruses for therapeutic applications and vaccines
10.1002/1873-3468.13731 · 2020 · External reference
10.1083/jcb.201201140
10.1083/jcb.201201140 · External reference
Retargeting adenoviruses for therapeutic applications and vaccines
10.1002/1873-3468.13731 · ExternalCitation · doi-reference
AAVLINK: A potent DNA-recombination method for large cargo delivery in gene therapy
10.1016/j.cell.2025.12.039 · ExternalCitation · doi-reference
10.1016/j.exer.2020.108330
10.1016/j.exer.2020.108330 · ExternalCitation · doi-reference
Highly efficient retinal gene delivery with helper-dependent adenoviral vectors
10.1016/j.gendis.2014.09.002 · ExternalCitation · doi-reference
Human iPSC-Derived Retinas Recapitulate the Fetal CRB1 CRB2 Complex Formation and Demonstrate that Photoreceptors and Muller Glia Are Targets of AAV5
10.1016/j.stemcr.2019.03.002 · ExternalCitation · doi-reference
10.1016/j.ymthe.2017.05.004
10.1016/j.ymthe.2017.05.004 · ExternalCitation · doi-reference
Successful large gene augmentation of USH2A with non-viral episomal vectors
10.1016/j.ymthe.2023.06.012 · ExternalCitation · doi-reference
Systematic empirical evaluation of individual base editing targets: Validating therapeutic targets in USH2A and comparison of methods
10.1016/j.ymthe.2025.01.042 · ExternalCitation · doi-reference
10.1016/j.ymthe.2025.04.045
10.1016/j.ymthe.2025.04.045 · ExternalCitation · doi-reference
Retinal transduction profiles by high-capacity viral vectors
10.1038/gt.2014.57 · ExternalCitation · doi-reference
10.1038/mt.2008.23
10.1038/mt.2008.23 · ExternalCitation · doi-reference
10.1038/s12276-018-0196-9
10.1038/s12276-018-0196-9 · ExternalCitation · doi-reference
Adeno-associated virus as a delivery vector for gene therapy of human diseases
10.1038/s41392-024-01780-w · ExternalCitation · doi-reference
The usherin mutation c.2299delG leads to its mislocalization and disrupts interactions with whirlin and VLGR1
10.1038/s41467-023-36431-1 · ExternalCitation · doi-reference
10.1038/s41467-023-42386-0
10.1038/s41467-023-42386-0 · ExternalCitation · doi-reference
Expression of the human usherin c.2299delG mutation leads to early-onset auditory loss and stereocilia disorganization
10.1038/s42003-023-05296-x · ExternalCitation · doi-reference
10.1038/sj.gt.3302612
10.1038/sj.gt.3302612 · ExternalCitation · doi-reference
10.1056/nejmoa2309915
10.1056/nejmoa2309915 · ExternalCitation · doi-reference
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10.1073/pnas.202300099 · ExternalCitation · doi-reference
10.1083/jcb.201201140
10.1083/jcb.201201140 · ExternalCitation · doi-reference
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10.1089/hum.2012.125 · ExternalCitation · doi-reference
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10.1089/hum.2019.220 · ExternalCitation · doi-reference
10.1101/2020.02.04.934240
10.1101/2020.02.04.934240 · ExternalCitation · doi-reference
10.1111/jcmm.13504
10.1111/jcmm.13504 · ExternalCitation · doi-reference
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10.1126/scitranslmed.aav4523 · ExternalCitation · doi-reference
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10.1128/jvi.78.1.9-22.2004 · ExternalCitation · doi-reference
10.1167/iovs.07-0257
10.1167/iovs.07-0257 · ExternalCitation · doi-reference
10.1167/iovs.10-6141
10.1167/iovs.10-6141 · ExternalCitation · doi-reference
USH2A-Mutated Human Retinal Organoids Model Rod-Cone Dystrophy
10.1167/iovs.66.14.2 · ExternalCitation · doi-reference
10.1172/jci.insight.174179
10.1172/jci.insight.174179 · ExternalCitation · doi-reference
Subretinal gene delivery using helper-dependent adenoviral vectors
10.1186/2045-3701-1-15 · ExternalCitation · doi-reference
10.3389/fnagi.2022.948279
10.3389/fnagi.2022.948279 · ExternalCitation · doi-reference
10.3389/fnins.2020.00838
10.3389/fnins.2020.00838 · ExternalCitation · doi-reference
Challenges and Prospects for Helper-Dependent Adenoviral Vector-Mediated Gene Therapy
10.3390/biomedicines2020132 · ExternalCitation · doi-reference
10.3390/cells9040953
10.3390/cells9040953 · ExternalCitation · doi-reference
10.3390/genes10121015
10.3390/genes10121015 · ExternalCitation · doi-reference
10.3390/ijms21103643
10.3390/ijms21103643 · ExternalCitation · doi-reference
10.3390/ijms22052318
10.3390/ijms22052318 · ExternalCitation · doi-reference
10.3390/ijms231911669
10.3390/ijms231911669 · ExternalCitation · doi-reference
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10.3390/ijms26010055 · ExternalCitation · doi-reference
10.3390/ijms27041636
10.3390/ijms27041636 · ExternalCitation · doi-reference
10.3390/v15091801
10.3390/v15091801 · ExternalCitation · doi-reference
