Zongchao Han, Shannon M Conley, Rasha S Makkia, Mark J Cooper, Muna I Naash. J Clin Invest 2012
Times Cited: 100
Times Cited: 100
Times Cited
Times Co-cited
Similarity
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
37
Effect of gene therapy on visual function in Leber's congenital amaurosis.
James W B Bainbridge, Alexander J Smith, Susie S Barker, Scott Robbie, Robert Henderson, Kamaljit Balaggan, Ananth Viswanathan, Graham E Holder, Andrew Stockman, Nick Tyler,[...]. N Engl J Med 2008
James W B Bainbridge, Alexander J Smith, Susie S Barker, Scott Robbie, Robert Henderson, Kamaljit Balaggan, Ananth Viswanathan, Graham E Holder, Andrew Stockman, Nick Tyler,[...]. N Engl J Med 2008
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Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy.
J Kong, S-R Kim, K Binley, I Pata, K Doi, J Mannik, J Zernant-Rajang, O Kan, S Iqball, S Naylor,[...]. Gene Ther 2008
J Kong, S-R Kim, K Binley, I Pata, K Doi, J Mannik, J Zernant-Rajang, O Kan, S Iqball, S Naylor,[...]. Gene Ther 2008
33
Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery.
Zongchao Han, Shannon M Conley, Rasha Makkia, Junjing Guo, Mark J Cooper, Muna I Naash. PLoS One 2012
Zongchao Han, Shannon M Conley, Rasha Makkia, Junjing Guo, Mark J Cooper, Muna I Naash. PLoS One 2012
56
Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa.
Xue Cai, Shannon M Conley, Zack Nash, Steven J Fliesler, Mark J Cooper, Muna I Naash. FASEB J 2010
Xue Cai, Shannon M Conley, Zack Nash, Steven J Fliesler, Mark J Cooper, Muna I Naash. FASEB J 2010
37
Efficient non-viral ocular gene transfer with compacted DNA nanoparticles.
Rafal Farjo, Jeff Skaggs, Alexander B Quiambao, Mark J Cooper, Muna I Naash. PLoS One 2006
Rafal Farjo, Jeff Skaggs, Alexander B Quiambao, Mark J Cooper, Muna I Naash. PLoS One 2006
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S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA.
Adarsha Koirala, Rasha S Makkia, Shannon M Conley, Mark J Cooper, Muna I Naash. Hum Mol Genet 2013
Adarsha Koirala, Rasha S Makkia, Shannon M Conley, Mark J Cooper, Muna I Naash. Hum Mol Genet 2013
52
Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles.
T L Fink, P J Klepcyk, S M Oette, C R Gedeon, S L Hyatt, T H Kowalczyk, R C Moen, M J Cooper. Gene Ther 2006
T L Fink, P J Klepcyk, S M Oette, C R Gedeon, S L Hyatt, T H Kowalczyk, R C Moen, M J Cooper. Gene Ther 2006
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Effective delivery of large genes to the retina by dual AAV vectors.
Ivana Trapani, Pasqualina Colella, Andrea Sommella, Carolina Iodice, Giulia Cesi, Sonia de Simone, Elena Marrocco, Settimio Rossi, Massimo Giunti, Arpad Palfi,[...]. EMBO Mol Med 2014
Ivana Trapani, Pasqualina Colella, Andrea Sommella, Carolina Iodice, Giulia Cesi, Sonia de Simone, Elena Marrocco, Settimio Rossi, Massimo Giunti, Arpad Palfi,[...]. EMBO Mol Med 2014
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A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy.
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Gene therapy restores vision in a canine model of childhood blindness.
G M Acland, G D Aguirre, J Ray, Q Zhang, T S Aleman, A V Cideciyan, S E Pearce-Kelling, V Anand, Y Zeng, A M Maguire,[...]. Nat Genet 2001
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Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.
Mariacarmela Allocca, Monica Doria, Marco Petrillo, Pasqualina Colella, Maria Garcia-Hoyos, Daniel Gibbs, So Ra Kim, Albert Maguire, Tonia S Rex, Umberto Di Vicino,[...]. J Clin Invest 2008
Mariacarmela Allocca, Monica Doria, Marco Petrillo, Pasqualina Colella, Maria Garcia-Hoyos, Daniel Gibbs, So Ra Kim, Albert Maguire, Tonia S Rex, Umberto Di Vicino,[...]. J Clin Invest 2008
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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
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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Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease.
Katie Binley, Peter Widdowson, Julie Loader, Michelle Kelleher, Sharifah Iqball, Georgina Ferrige, Jackie de Belin, Marie Carlucci, Diana Angell-Manning, Felicity Hurst,[...]. Invest Ophthalmol Vis Sci 2013
Katie Binley, Peter Widdowson, Julie Loader, Michelle Kelleher, Sharifah Iqball, Georgina Ferrige, Jackie de Belin, Marie Carlucci, Diana Angell-Manning, Felicity Hurst,[...]. Invest Ophthalmol Vis Sci 2013
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Direct gene transfer with compacted DNA nanoparticles in retinal pigment epithelial cells: expression, repeat delivery and lack of toxicity.
Zongchao Han, Adarsha Koirala, Rasha Makkia, Mark J Cooper, Muna I Naash. Nanomedicine (Lond) 2012
Zongchao Han, Adarsha Koirala, Rasha Makkia, Mark J Cooper, Muna I Naash. Nanomedicine (Lond) 2012
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Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.
Robert E MacLaren, Markus Groppe, Alun R Barnard, Charles L Cottriall, Tanya Tolmachova, Len Seymour, K Reed Clark, Matthew J During, Frans P M Cremers, Graeme C M Black,[...]. Lancet 2014
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Effect of genome size on AAV vector packaging.
Zhijian Wu, Hongyan Yang, Peter Colosi. Mol Ther 2010
Zhijian Wu, Hongyan Yang, Peter Colosi. Mol Ther 2010
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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
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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Insights into the function of Rim protein in photoreceptors and etiology of Stargardt's disease from the phenotype in abcr knockout mice.
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A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles.
Xue Cai, Zack Nash, Shannon M Conley, Steven J Fliesler, Mark J Cooper, Muna I Naash. PLoS One 2009
Xue Cai, Zack Nash, Shannon M Conley, Steven J Fliesler, Mark J Cooper, Muna I Naash. PLoS One 2009
22
Ocular delivery of compacted DNA-nanoparticles does not elicit toxicity in the mouse retina.
Xi-Qin Ding, Alexander B Quiambao, J Browning Fitzgerald, Mark J Cooper, Shannon M Conley, Muna I Naash. PLoS One 2009
Xi-Qin Ding, Alexander B Quiambao, J Browning Fitzgerald, Mark J Cooper, Shannon M Conley, Muna I Naash. PLoS One 2009
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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
16
Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa.
Sophia Millington-Ward, Naomi Chadderton, Mary O'Reilly, Arpad Palfi, Tobias Goldmann, Claire Kilty, Marian Humphries, Uwe Wolfrum, Jean Bennett, Peter Humphries,[...]. Mol Ther 2011
Sophia Millington-Ward, Naomi Chadderton, Mary O'Reilly, Arpad Palfi, Tobias Goldmann, Claire Kilty, Marian Humphries, Uwe Wolfrum, Jean Bennett, Peter Humphries,[...]. Mol Ther 2011
16
Genomic DNA nanoparticles rescue rhodopsin-associated retinitis pigmentosa phenotype.
Zongchao Han, Marcellus J Banworth, Rasha Makkia, Shannon M Conley, Muayyad R Al-Ubaidi, Mark J Cooper, Muna I Naash. FASEB J 2015
Zongchao Han, Marcellus J Banworth, Rasha Makkia, Shannon M Conley, Muayyad R Al-Ubaidi, Mark J Cooper, Muna I Naash. FASEB J 2015
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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
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
16
AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa.
Haoyu Mao, Thomas James, Alison Schwein, Arseniy E Shabashvili, William W Hauswirth, Marina S Gorbatyuk, Alfred S Lewin. Hum Gene Ther 2011
Haoyu Mao, Thomas James, Alison Schwein, Arseniy E Shabashvili, William W Hauswirth, Marina S Gorbatyuk, Alfred S Lewin. Hum Gene Ther 2011
16
RNA interference-mediated suppression and replacement of human rhodopsin in vivo.
Mary O'Reilly, Arpad Palfi, Naomi Chadderton, Sophia Millington-Ward, Marius Ader, Thérèse Cronin, Thérèse Tuohy, Alberto Auricchio, Markus Hildinger, Amanda Tivnan,[...]. Am J Hum Genet 2007
Mary O'Reilly, Arpad Palfi, Naomi Chadderton, Sophia Millington-Ward, Marius Ader, Thérèse Cronin, Thérèse Tuohy, Alberto Auricchio, Markus Hildinger, Amanda Tivnan,[...]. Am J Hum Genet 2007
15
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
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
15
AAV2 gene therapy readministration in three adults with congenital blindness.
Jean Bennett, Manzar Ashtari, Jennifer Wellman, Kathleen A Marshall, Laura L Cyckowski, Daniel C Chung, Sarah McCague, Eric A Pierce, Yifeng Chen, Jeannette L Bennicelli,[...]. Sci Transl Med 2012
Jean Bennett, Manzar Ashtari, Jennifer Wellman, Kathleen A Marshall, Laura L Cyckowski, Daniel C Chung, Sarah McCague, Eric A Pierce, Yifeng Chen, Jeannette L Bennicelli,[...]. Sci Transl Med 2012
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Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.
Francesca Simonelli, Albert M Maguire, Francesco Testa, Eric A Pierce, Federico Mingozzi, Jeannette L Bennicelli, Settimio Rossi, Kathleen Marshall, Sandro Banfi, Enrico M Surace,[...]. Mol Ther 2010
Francesca Simonelli, Albert M Maguire, Francesco Testa, Eric A Pierce, Federico Mingozzi, Jeannette L Bennicelli, Settimio Rossi, Kathleen Marshall, Sandro Banfi, Enrico M Surace,[...]. Mol Ther 2010
15
A comprehensive review of retinal gene therapy.
Shannon E Boye, Sanford L Boye, Alfred S Lewin, William W Hauswirth. Mol Ther 2013
Shannon E Boye, Sanford L Boye, Alfred S Lewin, William W Hauswirth. Mol Ther 2013
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Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.
Frank M Dyka, Sanford L Boye, Vince A Chiodo, William W Hauswirth, Shannon E Boye. Hum Gene Ther Methods 2014
Frank M Dyka, Sanford L Boye, Vince A Chiodo, William W Hauswirth, Shannon E Boye. Hum Gene Ther Methods 2014
20
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
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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Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle.
T M Redmond, S Yu, E Lee, D Bok, D Hamasaki, N Chen, P Goletz, J X Ma, R K Crouch, K Pfeifer. Nat Genet 1998
T M Redmond, S Yu, E Lee, D Bok, D Hamasaki, N Chen, P Goletz, J X Ma, R K Crouch, K Pfeifer. Nat Genet 1998
14
Leber congenital amaurosis: genes, proteins and disease mechanisms.
Anneke I den Hollander, Ronald Roepman, Robert K Koenekoop, Frans P M Cremers. Prog Retin Eye Res 2008
Anneke I den Hollander, Ronald Roepman, Robert K Koenekoop, Frans P M Cremers. Prog Retin Eye Res 2008
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Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution.
Michael W Konstan, Pamela B Davis, Jeffrey S Wagener, Kathleen A Hilliard, Robert C Stern, Laura J H Milgram, Tomasz H Kowalczyk, Susannah L Hyatt, Tamara L Fink, Christopher R Gedeon,[...]. Hum Gene Ther 2004
Michael W Konstan, Pamela B Davis, Jeffrey S Wagener, Kathleen A Hilliard, Robert C Stern, Laura J H Milgram, Tomasz H Kowalczyk, Susannah L Hyatt, Tamara L Fink, Christopher R Gedeon,[...]. Hum Gene Ther 2004
14
Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.
William A Beltran, Artur V Cideciyan, Alfred S Lewin, Simone Iwabe, Hemant Khanna, Alexander Sumaroka, Vince A Chiodo, Diego S Fajardo, Alejandro J Román, Wen-Tao Deng,[...]. Proc Natl Acad Sci U S A 2012
William A Beltran, Artur V Cideciyan, Alfred S Lewin, Simone Iwabe, Hemant Khanna, Alexander Sumaroka, Vince A Chiodo, Diego S Fajardo, Alejandro J Román, Wen-Tao Deng,[...]. Proc Natl Acad Sci U S A 2012
14
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2.
Francesco Testa, Albert M Maguire, Settimio Rossi, Eric A Pierce, Paolo Melillo, Kathleen Marshall, Sandro Banfi, Enrico M Surace, Junwei Sun, Carmela Acerra,[...]. Ophthalmology 2013
Francesco Testa, Albert M Maguire, Settimio Rossi, Eric A Pierce, Paolo Melillo, Kathleen Marshall, Sandro Banfi, Enrico M Surace, Junwei Sun, Carmela Acerra,[...]. Ophthalmology 2013
14
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
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
14
Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors.
P Colella, I Trapani, G Cesi, A Sommella, A Manfredi, A Puppo, C Iodice, S Rossi, F Simonelli, M Giunti,[...]. Gene Ther 2014
P Colella, I Trapani, G Cesi, A Sommella, A Manfredi, A Puppo, C Iodice, S Rossi, F Simonelli, M Giunti,[...]. Gene Ther 2014
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High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors.
Hilda Petrs-Silva, Astra Dinculescu, Qiuhong Li, Seok-Hong Min, Vince Chiodo, Ji-Jing Pang, Li Zhong, Sergei Zolotukhin, Arun Srivastava, Alfred S Lewin,[...]. Mol Ther 2009
Hilda Petrs-Silva, Astra Dinculescu, Qiuhong Li, Seok-Hong Min, Vince Chiodo, Ji-Jing Pang, Li Zhong, Sergei Zolotukhin, Arun Srivastava, Alfred S Lewin,[...]. Mol Ther 2009
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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
13
18
Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies.
Steven D Schwartz, Carl D Regillo, Byron L Lam, Dean Eliott, Philip J Rosenfeld, Ninel Z Gregori, Jean-Pierre Hubschman, Janet L Davis, Gad Heilwell, Marc Spirn,[...]. Lancet 2015
Steven D Schwartz, Carl D Regillo, Byron L Lam, Dean Eliott, Philip J Rosenfeld, Ninel Z Gregori, Jean-Pierre Hubschman, Janet L Davis, Gad Heilwell, Marc Spirn,[...]. Lancet 2015
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Vector platforms for gene therapy of inherited retinopathies.
Ivana Trapani, Agostina Puppo, Alberto Auricchio. Prog Retin Eye Res 2014
Ivana Trapani, Agostina Puppo, Alberto Auricchio. Prog Retin Eye Res 2014
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Nanoparticle-based technologies for retinal gene therapy.
Jeffrey Adijanto, Muna I Naash. Eur J Pharm Biopharm 2015
Jeffrey Adijanto, Muna I Naash. Eur J Pharm Biopharm 2015
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AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia.
Ji-jing Pang, Wen-Tao Deng, Xufeng Dai, Bo Lei, Drew Everhart, Yumiko Umino, Jie Li, Keqing Zhang, Song Mao, Sanford L Boye,[...]. PLoS One 2012
Ji-jing Pang, Wen-Tao Deng, Xufeng Dai, Bo Lei, Drew Everhart, Yumiko Umino, Jie Li, Keqing Zhang, Song Mao, Sanford L Boye,[...]. PLoS One 2012
13
Gene therapy rescues cone function in congenital achromatopsia.
András M Komáromy, John J Alexander, Jessica S Rowlan, Monique M Garcia, Vince A Chiodo, Asli Kaya, Jacqueline C Tanaka, Gregory M Acland, William W Hauswirth, Gustavo D Aguirre. Hum Mol Genet 2010
András M Komáromy, John J Alexander, Jessica S Rowlan, Monique M Garcia, Vince A Chiodo, Asli Kaya, Jacqueline C Tanaka, Gregory M Acland, William W Hauswirth, Gustavo D Aguirre. Hum Mol Genet 2010
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Nanoparticle-mediated gene transfer specific to retinal pigment epithelial cells.
Adarsha Koirala, Rasha S Makkia, Mark J Cooper, Muna I Naash. Biomaterials 2011
Adarsha Koirala, Rasha S Makkia, Mark J Cooper, Muna I Naash. Biomaterials 2011
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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.