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Times Cited
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Safety and efficacy of gene transfer for Leber's congenital amaurosis.
Albert M Maguire, Francesca Simonelli, Eric A Pierce, Edward N Pugh, Federico Mingozzi, Jeannette Bennicelli, Sandro Banfi, Kathleen A Marshall, Francesco Testa, Enrico M Surace,[...]. N Engl J Med 2008
Albert M Maguire, Francesca Simonelli, Eric A Pierce, Edward N Pugh, Federico Mingozzi, Jeannette Bennicelli, Sandro Banfi, Kathleen A Marshall, Francesco Testa, Enrico M Surace,[...]. N Engl J Med 2008
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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
William W Hauswirth, Tomas S Aleman, Shalesh Kaushal, Artur V Cideciyan, Sharon B Schwartz, Lili Wang, Thomas J Conlon, Sanford L Boye, Terence R Flotte, Barry J Byrne,[...]. Hum Gene Ther 2008
William W Hauswirth, Tomas S Aleman, Shalesh Kaushal, Artur V Cideciyan, Sharon B Schwartz, Lili Wang, Thomas J Conlon, Sanford L Boye, Terence R Flotte, Barry J Byrne,[...]. Hum Gene Ther 2008
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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Stephen Russell, Jean Bennett, Jennifer A Wellman, Daniel C Chung, Zi-Fan Yu, Amy Tillman, Janet Wittes, Julie Pappas, Okan Elci, Sarah McCague,[...]. Lancet 2017
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Long-term effect of gene therapy on Leber's congenital amaurosis.
James W B Bainbridge, Manjit S Mehat, Venki Sundaram, Scott J Robbie, Susie E Barker, Caterina Ripamonti, Anastasios Georgiadis, Freya M Mowat, Stuart G Beattie, Peter J Gardner,[...]. N Engl J Med 2015
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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.
Artur V Cideciyan, Tomas S Aleman, Sanford L Boye, Sharon B Schwartz, Shalesh Kaushal, Alejandro J Roman, Ji-Jing Pang, Alexander Sumaroka, Elizabeth A M Windsor, James M Wilson,[...]. Proc Natl Acad Sci U S A 2008
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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.
Albert M Maguire, Katherine A High, Alberto Auricchio, J Fraser Wright, Eric A Pierce, Francesco Testa, Federico Mingozzi, Jeannette L Bennicelli, Gui-shuang Ying, Settimio Rossi,[...]. Lancet 2009
Albert M Maguire, Katherine A High, Alberto Auricchio, J Fraser Wright, Eric A Pierce, Francesco Testa, Federico Mingozzi, Jeannette L Bennicelli, Gui-shuang Ying, Settimio Rossi,[...]. Lancet 2009
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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Albert M Maguire, Stephen Russell, Jennifer A Wellman, Daniel C Chung, Zi-Fan Yu, Amy Tillman, Janet Wittes, Julie Pappas, Okan Elci, Kathleen A Marshall,[...]. Ophthalmology 2019
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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
Samuel G Jacobson, Artur V Cideciyan, Ramakrishna Ratnakaram, Elise Heon, Sharon B Schwartz, Alejandro J Roman, Marc C Peden, Tomas S Aleman, Sanford L Boye, Alexander Sumaroka,[...]. Arch Ophthalmol 2012
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Gene therapy restores vision in a canine model of childhood blindness.
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Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.
Jean Bennett, Jennifer Wellman, Kathleen A Marshall, Sarah McCague, Manzar Ashtari, Julie DiStefano-Pappas, Okan U Elci, Daniel C Chung, Junwei Sun, J Fraser Wright,[...]. Lancet 2016
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Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.
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In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous.
Deniz Dalkara, Leah C Byrne, Ryan R Klimczak, Meike Visel, Lu Yin, William H Merigan, John G Flannery, David V Schaffer. Sci Transl Med 2013
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Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.
Artur V Cideciyan, Samuel G Jacobson, William A Beltran, Alexander Sumaroka, Malgorzata Swider, Simone Iwabe, Alejandro J Roman, Melani B Olivares, Sharon B Schwartz, András M Komáromy,[...]. Proc Natl Acad Sci U S A 2013
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Adeno-associated virus vector as a platform for gene therapy delivery.
Dan Wang, Phillip W L Tai, Guangping Gao. Nat Rev Drug Discov 2019
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Improvement and decline in vision with gene therapy in childhood blindness.
Samuel G Jacobson, Artur V Cideciyan, Alejandro J Roman, Alexander Sumaroka, Sharon B Schwartz, Elise Heon, William W Hauswirth. N Engl J Med 2015
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Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect.
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Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR.
Jasmina Cehajic-Kapetanovic, Kanmin Xue, Cristina Martinez-Fernandez de la Camara, Anika Nanda, Alexandra Davies, Laura J Wood, Anna Paola Salvetti, M Dominik Fischer, James W Aylward, Alun R Barnard,[...]. Nat Med 2020
Jasmina Cehajic-Kapetanovic, Kanmin Xue, Cristina Martinez-Fernandez de la Camara, Anika Nanda, Alexandra Davies, Laura J Wood, Anna Paola Salvetti, M Dominik Fischer, James W Aylward, Alun R Barnard,[...]. Nat Med 2020
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Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.
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Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle.
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Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.
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Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates.
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Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.
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Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.
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Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
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Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.
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Search-and-replace genome editing without double-strand breaks or donor DNA.
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In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa.
Benjamin Bakondi, Wenjian Lv, Bin Lu, Melissa K Jones, Yuchun Tsai, Kevin J Kim, Rachelle Levy, Aslam Abbasi Akhtar, Joshua J Breunig, Clive N Svendsen,[...]. Mol Ther 2016
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Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.
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AAV2 gene therapy readministration in three adults with congenital blindness.
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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
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Choroideremia Gene Therapy Phase 2 Clinical Trial: 24-Month Results.
Byron L Lam, Janet L Davis, Ninel Z Gregori, Robert E MacLaren, Aniz Girach, Jennifer D Verriotto, Belen Rodriguez, Potyra R Rosa, Xiaojun Zhang, William J Feuer. Am J Ophthalmol 2019
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Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience.
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Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year.
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Leber congenital amaurosis: genes, proteins and disease mechanisms.
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Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia.
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Effective delivery of large genes to the retina by dual AAV vectors.
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Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial.
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Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results.
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Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration.
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Novel mobility test to assess functional vision in patients with inherited retinal dystrophies.
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Mutations in RPE65 cause autosomal recessive childhood-onset severe retinal dystrophy.
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Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey.
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Co-cited is the co-citation frequency, indicating how many articles cite the article together with the query article. Similarity is the co-citation as percentage of the times cited of the query article or the article in the search results, whichever is the lowest. These numbers are calculated for the last 100 citations when articles are cited more than 100 times.