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, Andrew J Lotery, Susan M Downes, Andrew R Webster, Miguel C Seabra. Lancet 2014
Times Cited: 520
Times Cited: 520
Times Cited
Times Co-cited
Similarity
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
37
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
32
Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience.
Ioannis S Dimopoulos, Stephanie C Hoang, Alina Radziwon, Natalia M Binczyk, Miguel C Seabra, Robert E MacLaren, Rizwan Somani, Matthew T S Tennant, Ian M MacDonald. Am J Ophthalmol 2018
Ioannis S Dimopoulos, Stephanie C Hoang, Alina Radziwon, Natalia M Binczyk, Miguel C Seabra, Robert E MacLaren, Rizwan Somani, Matthew T S Tennant, Ian M MacDonald. Am J Ophthalmol 2018
38
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
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
39
Visual Acuity after Retinal Gene Therapy for Choroideremia.
Thomas L Edwards, Jasleen K Jolly, Markus Groppe, Alun R Barnard, Charles L Cottriall, Tanya Tolmachova, Graeme C Black, Andrew R Webster, Andrew J Lotery, Graham E Holder,[...]. N Engl J Med 2016
Thomas L Edwards, Jasleen K Jolly, Markus Groppe, Alun R Barnard, Charles L Cottriall, Tanya Tolmachova, Graeme C Black, Andrew R Webster, Andrew J Lotery, Graham E Holder,[...]. N Engl J Med 2016
26
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
26
Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients With Choroideremia: A Randomized Clinical Trial.
M Dominik Fischer, G Alex Ochakovski, Benjamin Beier, Immanuel P Seitz, Yousof Vaheb, Constanze Kortuem, Felix F L Reichel, Laura Kuehlewein, Nadine A Kahle, Tobias Peters,[...]. JAMA Ophthalmol 2019
M Dominik Fischer, G Alex Ochakovski, Benjamin Beier, Immanuel P Seitz, Yousof Vaheb, Constanze Kortuem, Felix F L Reichel, Laura Kuehlewein, Nadine A Kahle, Tobias Peters,[...]. JAMA Ophthalmol 2019
62
Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia.
Kanmin Xue, Jasleen K Jolly, Alun R Barnard, Anna Rudenko, Anna P Salvetti, Maria I Patrício, Thomas L Edwards, Markus Groppe, Harry O Orlans, Tanya Tolmachova,[...]. Nat Med 2018
Kanmin Xue, Jasleen K Jolly, Alun R Barnard, Anna Rudenko, Anna P Salvetti, Maria I Patrício, Thomas L Edwards, Markus Groppe, Harry O Orlans, Tanya Tolmachova,[...]. Nat Med 2018
26
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
24
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
21
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
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
20
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
18
Characterizing the Natural History of Visual Function in Choroideremia Using Microperimetry and Multimodal Retinal Imaging.
Jasleen K Jolly, Kanmin Xue, Thomas L Edwards, Markus Groppe, Robert E MacLaren. Invest Ophthalmol Vis Sci 2017
Jasleen K Jolly, Kanmin Xue, Thomas L Edwards, Markus Groppe, Robert E MacLaren. Invest Ophthalmol Vis Sci 2017
29
A Qualitative and Quantitative Assessment of Fundus Autofluorescence Patterns in Patients With Choroideremia.
Jasleen K Jolly, Thomas L Edwards, Jonathan Moules, Markus Groppe, Susan M Downes, Robert E MacLaren. Invest Ophthalmol Vis Sci 2016
Jasleen K Jolly, Thomas L Edwards, Jonathan Moules, Markus Groppe, Susan M Downes, Robert E MacLaren. Invest Ophthalmol Vis Sci 2016
30
Technique of retinal gene therapy: delivery of viral vector into the subretinal space.
K Xue, M Groppe, A P Salvetti, R E MacLaren. Eye (Lond) 2017
K Xue, M Groppe, A P Salvetti, R E MacLaren. Eye (Lond) 2017
21
Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.
Catherine Cukras, Henry E Wiley, Brett G Jeffrey, H Nida Sen, Amy Turriff, Yong Zeng, Camasamudram Vijayasarathy, Dario Marangoni, Lucia Ziccardi, Sten Kjellstrom,[...]. Mol Ther 2018
Catherine Cukras, Henry E Wiley, Brett G Jeffrey, H Nida Sen, Amy Turriff, Yong Zeng, Camasamudram Vijayasarathy, Dario Marangoni, Lucia Ziccardi, Sten Kjellstrom,[...]. Mol Ther 2018
15
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
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
14
CHANGES IN RETINAL SENSITIVITY AFTER GENE THERAPY IN CHOROIDEREMIA.
M Dominik Fischer, G Alex Ochakovski, Benjamin Beier, Immanuel P Seitz, Yousof Vaheb, Constanze Kortuem, Felix F L Reichel, Laura Kuehlewein, Nadine A Kahle, Tobias Peters,[...]. Retina 2020
M Dominik Fischer, G Alex Ochakovski, Benjamin Beier, Immanuel P Seitz, Yousof Vaheb, Constanze Kortuem, Felix F L Reichel, Laura Kuehlewein, Nadine A Kahle, Tobias Peters,[...]. Retina 2020
42
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
14
The Spectrum of CHM Gene Mutations in Choroideremia and Their Relationship to Clinical Phenotype.
Matthew P Simunovic, Jasleen K Jolly, Kanmin Xue, Thomas L Edwards, Markus Groppe, Susan M Downes, Robert E MacLaren. Invest Ophthalmol Vis Sci 2016
Matthew P Simunovic, Jasleen K Jolly, Kanmin Xue, Thomas L Edwards, Markus Groppe, Susan M Downes, Robert E MacLaren. Invest Ophthalmol Vis Sci 2016
24
Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.
Richard G Weleber, Mark E Pennesi, David J Wilson, Shalesh Kaushal, Laura R Erker, Lauren Jensen, Maureen T McBride, Terence R Flotte, Margaret Humphries, Roberto Calcedo,[...]. Ophthalmology 2016
Richard G Weleber, Mark E Pennesi, David J Wilson, Shalesh Kaushal, Laura R Erker, Lauren Jensen, Maureen T McBride, Terence R Flotte, Margaret Humphries, Roberto Calcedo,[...]. Ophthalmology 2016
13
Visual Function and Central Retinal Structure in Choroideremia.
Elise Heon, Talal Alabduljalil, David B McGuigan III, Artur V Cideciyan, Shuning Li, Shiyi Chen, Samuel G Jacobson. Invest Ophthalmol Vis Sci 2016
Elise Heon, Talal Alabduljalil, David B McGuigan III, Artur V Cideciyan, Shuning Li, Shiyi Chen, Samuel G Jacobson. Invest Ophthalmol Vis Sci 2016
26
Retrospective, longitudinal, and cross sectional study of visual acuity impairment in choroideraemia.
M Flynn Roberts, G A Fishman, D K Roberts, J R Heckenlively, R G Weleber, R J Anderson, S Grover. Br J Ophthalmol 2002
M Flynn Roberts, G A Fishman, D K Roberts, J R Heckenlively, R G Weleber, R J Anderson, S Grover. Br J Ophthalmol 2002
13
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
13
Clinically Focused Molecular Investigation of 1000 Consecutive Families with Inherited Retinal Disease.
Edwin M Stone, Jeaneen L Andorf, S Scott Whitmore, Adam P DeLuca, Joseph C Giacalone, Luan M Streb, Terry A Braun, Robert F Mullins, Todd E Scheetz, Val C Sheffield,[...]. Ophthalmology 2017
Edwin M Stone, Jeaneen L Andorf, S Scott Whitmore, Adam P DeLuca, Joseph C Giacalone, Luan M Streb, Terry A Braun, Robert F Mullins, Todd E Scheetz, Val C Sheffield,[...]. Ophthalmology 2017
13
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
13
Remodeling of the human retina in choroideremia: rab escort protein 1 (REP-1) mutations.
Samuel G Jacobson, Artur V Cideciyan, Alexander Sumaroka, Tomas S Aleman, Sharon B Schwartz, Elizabeth A M Windsor, Alejandro J Roman, Edwin M Stone, Ian M MacDonald. Invest Ophthalmol Vis Sci 2006
Samuel G Jacobson, Artur V Cideciyan, Alexander Sumaroka, Tomas S Aleman, Sharon B Schwartz, Elizabeth A M Windsor, Alejandro J Roman, Edwin M Stone, Ian M MacDonald. Invest Ophthalmol Vis Sci 2006
12
Correlation of Optical Coherence Tomography and Autofluorescence in the Outer Retina and Choroid of Patients With Choroideremia.
Kanmin Xue, Marta Oldani, Jasleen K Jolly, Thomas L Edwards, Markus Groppe, Susan M Downes, Robert E MacLaren. Invest Ophthalmol Vis Sci 2016
Kanmin Xue, Marta Oldani, Jasleen K Jolly, Thomas L Edwards, Markus Groppe, Susan M Downes, Robert E MacLaren. Invest Ophthalmol Vis Sci 2016
21
High-resolution adaptive optics retinal imaging of cellular structure in choroideremia.
Jessica I W Morgan, Grace Han, Eva Klinman, William M Maguire, Daniel C Chung, Albert M Maguire, Jean Bennett. Invest Ophthalmol Vis Sci 2014
Jessica I W Morgan, Grace Han, Eva Klinman, William M Maguire, Daniel C Chung, Albert M Maguire, Jean Bennett. Invest Ophthalmol Vis Sci 2014
15
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect.
Artur V Cideciyan, Samuel G Jacobson, Arlene V Drack, Allen C Ho, Jason Charng, Alexandra V Garafalo, Alejandro J Roman, Alexander Sumaroka, Ian C Han, Maria D Hochstedler,[...]. Nat Med 2019
Artur V Cideciyan, Samuel G Jacobson, Arlene V Drack, Allen C Ho, Jason Charng, Alexandra V Garafalo, Alejandro J Roman, Alexander Sumaroka, Ian C Han, Maria D Hochstedler,[...]. Nat Med 2019
12
Natural History of the Central Structural Abnormalities in Choroideremia: A Prospective Cross-Sectional Study.
Tomas S Aleman, Grace Han, Leona W Serrano, Nicole M Fuerst, Emily S Charlson, Denise J Pearson, Daniel C Chung, Anastasia Traband, Wei Pan, Gui-Shuang Ying,[...]. Ophthalmology 2017
Tomas S Aleman, Grace Han, Leona W Serrano, Nicole M Fuerst, Emily S Charlson, Denise J Pearson, Daniel C Chung, Anastasia Traband, Wei Pan, Gui-Shuang Ying,[...]. Ophthalmology 2017
16
Cloning of a gene that is rearranged in patients with choroideraemia.
F P Cremers, D J van de Pol, L P van Kerkhoff, B Wieringa, H H Ropers. Nature 1990
F P Cremers, D J van de Pol, L P van Kerkhoff, B Wieringa, H H Ropers. Nature 1990
11
Retinal degeneration in choroideremia: deficiency of rab geranylgeranyl transferase.
M C Seabra, M S Brown, J L Goldstein. Science 1993
M C Seabra, M S Brown, J L Goldstein. Science 1993
11
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.
Nicola G Ghazi, Emad B Abboud, Sawsan R Nowilaty, Hisham Alkuraya, Abdulrahman Alhommadi, Huimin Cai, Rui Hou, Wen-Tao Deng, Sanford L Boye, Abdulrahman Almaghamsi,[...]. Hum Genet 2016
Nicola G Ghazi, Emad B Abboud, Sawsan R Nowilaty, Hisham Alkuraya, Abdulrahman Alhommadi, Huimin Cai, Rui Hou, Wen-Tao Deng, Sanford L Boye, Abdulrahman Almaghamsi,[...]. Hum Genet 2016
11
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
11
Clinical and Genetic Features of Choroideremia in Childhood.
Kamron N Khan, Farrah Islam, Anthony T Moore, Michel Michaelides. Ophthalmology 2016
Kamron N Khan, Farrah Islam, Anthony T Moore, Michel Michaelides. Ophthalmology 2016
26
Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial.
M Dominik Fischer, Stylianos Michalakis, Barbara Wilhelm, Ditta Zobor, Regine Muehlfriedel, Susanne Kohl, Nicole Weisschuh, G Alex Ochakovski, Reinhild Klein, Christian Schoen,[...]. JAMA Ophthalmol 2020
M Dominik Fischer, Stylianos Michalakis, Barbara Wilhelm, Ditta Zobor, Regine Muehlfriedel, Susanne Kohl, Nicole Weisschuh, G Alex Ochakovski, Reinhild Klein, Christian Schoen,[...]. JAMA Ophthalmol 2020
23
Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo.
Tanya Tolmachova, Oleg E Tolmachov, Alun R Barnard, Samantha R de Silva, Daniel M Lipinski, Nathan J Walker, Robert E Maclaren, Miguel C Seabra. J Mol Med (Berl) 2013
Tanya Tolmachova, Oleg E Tolmachov, Alun R Barnard, Samantha R de Silva, Daniel M Lipinski, Nathan J Walker, Robert E Maclaren, Miguel C Seabra. J Mol Med (Berl) 2013
15
Retinal Degeneration in Choroideremia follows an Exponential Decay Function.
James W Aylward, Kanmin Xue, Maria I Patrício, Jasleen K Jolly, Jonathan C Wood, Jonathan Brett, Kirti M Jasani, Robert E MacLaren. Ophthalmology 2018
James W Aylward, Kanmin Xue, Maria I Patrício, Jasleen K Jolly, Jonathan C Wood, Jonathan Brett, Kirti M Jasani, Robert E MacLaren. Ophthalmology 2018
45
Measurement and Reproducibility of Preserved Ellipsoid Zone Area and Preserved Retinal Pigment Epithelium Area in Eyes With Choroideremia.
Amir H Hariri, Swetha B Velaga, Aniz Girach, Michael S Ip, Phuc V Le, Byron L Lam, M Dominik Fischer, Eeva-Marja Sankila, Mark E Pennesi, Frank G Holz,[...]. Am J Ophthalmol 2017
Amir H Hariri, Swetha B Velaga, Aniz Girach, Michael S Ip, Phuc V Le, Byron L Lam, M Dominik Fischer, Eeva-Marja Sankila, Mark E Pennesi, Frank G Holz,[...]. Am J Ophthalmol 2017
23
Correlation of retinal structure and function in choroideremia carriers.
Thomas L Edwards, Markus Groppe, Jasleen K Jolly, Susan M Downes, Robert E MacLaren. Ophthalmology 2015
Thomas L Edwards, Markus Groppe, Jasleen K Jolly, Susan M Downes, Robert E MacLaren. Ophthalmology 2015
28
Choroideremia: from genetic and clinical phenotyping to gene therapy and future treatments.
Andreas Mitsios, Adam M Dubis, Mariya Moosajee. Ther Adv Ophthalmol 2018
Andreas Mitsios, Adam M Dubis, Mariya Moosajee. Ther Adv Ophthalmol 2018
34
Surgical Technique for Subretinal Gene Therapy in Humans with Inherited Retinal Degeneration.
Janet L Davis, Ninel Z Gregori, Robert E MacLaren, Byron L Lam. Retina 2019
Janet L Davis, Ninel Z Gregori, Robert E MacLaren, Byron L Lam. Retina 2019
38
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
10
Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results.
John Guy, William J Feuer, Janet L Davis, Vittorio Porciatti, Phillip J Gonzalez, Rajeshwari D Koilkonda, Huijun Yuan, William W Hauswirth, Byron L Lam. Ophthalmology 2017
John Guy, William J Feuer, Janet L Davis, Vittorio Porciatti, Phillip J Gonzalez, Rajeshwari D Koilkonda, Huijun Yuan, William W Hauswirth, Byron L Lam. Ophthalmology 2017
10
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
10
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Amit C Nathwani, Edward G D Tuddenham, Savita Rangarajan, Cecilia Rosales, Jenny McIntosh, David C Linch, Pratima Chowdary, Anne Riddell, Arnulfo Jaquilmac Pie, Chris Harrington,[...]. N Engl J Med 2011
Amit C Nathwani, Edward G D Tuddenham, Savita Rangarajan, Cecilia Rosales, Jenny McIntosh, David C Linch, Pratima Chowdary, Anne Riddell, Arnulfo Jaquilmac Pie, Chris Harrington,[...]. N Engl J Med 2011
9
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential.
Qiuhong Li, Rehae Miller, Ping-Yang Han, Jijing Pang, Astra Dinculescu, Vince Chiodo, William W Hauswirth. Mol Vis 2008
Qiuhong Li, Rehae Miller, Ping-Yang Han, Jijing Pang, Astra Dinculescu, Vince Chiodo, William W Hauswirth. Mol Vis 2008
9
Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.
Guylène Le Meur, Pierre Lebranchu, Fanny Billaud, Oumeya Adjali, Sébastien Schmitt, Stéphane Bézieau, Yann Péréon, Romain Valabregue, Catherine Ivan, Christophe Darmon,[...]. Mol Ther 2018
Guylène Le Meur, Pierre Lebranchu, Fanny Billaud, Oumeya Adjali, Sébastien Schmitt, Stéphane Bézieau, Yann Péréon, Romain Valabregue, Catherine Ivan, Christophe Darmon,[...]. Mol Ther 2018
14
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.