Mary O'Reilly, Arpad Palfi, Naomi Chadderton, Sophia Millington-Ward, Marius Ader, Thérèse Cronin, Thérèse Tuohy, Alberto Auricchio, Markus Hildinger, Amanda Tivnan, Niamh McNally, Marian M Humphries, Anna-Sophia Kiang, Pete Humphries, Paul F Kenna, G Jane Farrar. Am J Hum Genet 2007
Times Cited: 133
Times Cited: 133
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
43
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
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43
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
41
Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy.
Naomi Chadderton, Sophia Millington-Ward, Arpad Palfi, Mary O'Reilly, Gearóid Tuohy, Marian M Humphries, Tiansen Li, Peter Humphries, Paul F Kenna, G Jane Farrar. Mol Ther 2009
Naomi Chadderton, Sophia Millington-Ward, Arpad Palfi, Mary O'Reilly, Gearóid Tuohy, Marian M Humphries, Tiansen Li, Peter Humphries, Paul F Kenna, G Jane Farrar. Mol Ther 2009
40
Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa.
A S Lewin, K A Drenser, W W Hauswirth, S Nishikawa, D Yasumura, J G Flannery, M M LaVail. Nat Med 1998
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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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Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy.
M M LaVail, D Yasumura, M T Matthes, K A Drenser, J G Flannery, A S Lewin, W W Hauswirth. Proc Natl Acad Sci U S A 2000
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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
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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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Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme.
M Gorbatyuk, V Justilien, J Liu, W W Hauswirth, A S Lewin. Exp Eye Res 2007
M Gorbatyuk, V Justilien, J Liu, W W Hauswirth, A S Lewin. Exp Eye Res 2007
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Toward a gene therapy for dominant disease: validation of an RNA interference-based mutation-independent approach.
Anna-Sophia Kiang, Arpad Palfi, Marius Ader, Paul F Kenna, Sophia Millington-Ward, Gerry Clark, Avril Kennan, Mary O'reilly, Lawrence C T Tam, Aileen Aherne,[...]. Mol Ther 2005
Anna-Sophia Kiang, Arpad Palfi, Marius Ader, Paul F Kenna, Sophia Millington-Ward, Gerry Clark, Avril Kennan, Mary O'reilly, Lawrence C T Tam, Aileen Aherne,[...]. Mol Ther 2005
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Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.
Haoyu Mao, Marina S Gorbatyuk, Brian Rossmiller, William W Hauswirth, Alfred S Lewin. Hum Gene Ther 2012
Haoyu Mao, Marina S Gorbatyuk, Brian Rossmiller, William W Hauswirth, Alfred S Lewin. Hum Gene Ther 2012
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A point mutation of the rhodopsin gene in one form of retinitis pigmentosa.
T P Dryja, T L McGee, E Reichel, L B Hahn, G S Cowley, D W Yandell, M A Sandberg, E L Berson. Nature 1990
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Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery.
M Gorbatyuk, V Justilien, J Liu, W W Hauswirth, A S Lewin. Vision Res 2007
M Gorbatyuk, V Justilien, J Liu, W W Hauswirth, A S Lewin. Vision Res 2007
40
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
20
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
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Mechanisms of cell death in rhodopsin retinitis pigmentosa: implications for therapy.
Hugo F Mendes, Jacqueline van der Spuy, J Paul Chapple, Michael E Cheetham. Trends Mol Med 2005
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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
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Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa.
Claudio Mussolino, Daniela Sanges, Elena Marrocco, Ciro Bonetti, Umberto Di Vicino, Valeria Marigo, Alberto Auricchio, Germana Meroni, Enrico Maria Surace. EMBO Mol Med 2011
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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
18
RNAi-based suppression and replacement of rds-peripherin in retinal organotypic culture.
Arpad Palfi, Marius Ader, Anna-Sophia Kiang, Sophia Millington-Ward, Gerry Clark, Mary O'Reilly, Helena P McMahon, Paul F Kenna, Peter Humphries, G Jane Farrar. Hum Mutat 2006
Arpad Palfi, Marius Ader, Anna-Sophia Kiang, Sophia Millington-Ward, Gerry Clark, Mary O'Reilly, Helena P McMahon, Paul F Kenna, Peter Humphries, G Jane Farrar. Hum Mutat 2006
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Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10).
Lawrence C S Tam, Anna-Sophia Kiang, Avril Kennan, Paul F Kenna, Naomi Chadderton, Marius Ader, Arpad Palfi, Aileen Aherne, Carmen Ayuso, Matthew Campbell,[...]. Hum Mol Genet 2008
Lawrence C S Tam, Anna-Sophia Kiang, Avril Kennan, Paul F Kenna, Naomi Chadderton, Marius Ader, Arpad Palfi, Aileen Aherne, Carmen Ayuso, Matthew Campbell,[...]. Hum Mol Genet 2008
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Knockdown of wild-type mouse rhodopsin using an AAV vectored ribozyme as part of an RNA replacement approach.
M S Gorbatyuk, J J Pang, J Thomas, William W Hauswirth, Alfred S Lewin. Mol Vis 2005
M S Gorbatyuk, J J Pang, J Thomas, William W Hauswirth, Alfred S Lewin. Mol Vis 2005
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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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Transgenic mice with a rhodopsin mutation (Pro23His): a mouse model of autosomal dominant retinitis pigmentosa.
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Retinopathy induced in mice by targeted disruption of the rhodopsin gene.
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Adeno-associated virus-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene.
Arpad Palfi, Sophia Millington-Ward, Naomi Chadderton, Mary O'Reilly, Tobias Goldmann, Marian M Humphries, Tiansen Li, Uwe Wolfrum, Peter Humphries, Paul F Kenna,[...]. Hum Gene Ther 2010
Arpad Palfi, Sophia Millington-Ward, Naomi Chadderton, Mary O'Reilly, Tobias Goldmann, Marian M Humphries, Tiansen Li, Uwe Wolfrum, Peter Humphries, Paul F Kenna,[...]. Hum Gene Ther 2010
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Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78.
Marina S Gorbatyuk, Tessa Knox, Matthew M LaVail, Oleg S Gorbatyuk, Syed M Noorwez, William W Hauswirth, Jonathan H Lin, Nicholas Muzyczka, Alfred S Lewin. Proc Natl Acad Sci U S A 2010
Marina S Gorbatyuk, Tessa Knox, Matthew M LaVail, Oleg S Gorbatyuk, Syed M Noorwez, William W Hauswirth, Jonathan H Lin, Nicholas Muzyczka, Alfred S Lewin. Proc Natl Acad Sci U S A 2010
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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
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The molecular basis of human retinal and vitreoretinal diseases.
Wolfgang Berger, Barbara Kloeckener-Gruissem, John Neidhardt. Prog Retin Eye Res 2010
Wolfgang Berger, Barbara Kloeckener-Gruissem, John Neidhardt. Prog Retin Eye Res 2010
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Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.
Jeannette Bennicelli, John Fraser Wright, Andras Komaromy, Jonathan B Jacobs, Bernd Hauck, Olga Zelenaia, Federico Mingozzi, Daniel Hui, Daniel Chung, Tonia S Rex,[...]. Mol Ther 2008
Jeannette Bennicelli, John Fraser Wright, Andras Komaromy, Jonathan B Jacobs, Bernd Hauck, Olga Zelenaia, Federico Mingozzi, Daniel Hui, Daniel Chung, Tonia S Rex,[...]. Mol Ther 2008
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Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model.
A Auricchio, G Kobinger, V Anand, M Hildinger, E O'Connor, A M Maguire, J M Wilson, J Bennett. Hum Mol Genet 2001
A Auricchio, G Kobinger, V Anand, M Hildinger, E O'Connor, A M Maguire, J M Wilson, J Bennett. Hum Mol Genet 2001
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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.
Gregory M Acland, Gustavo D Aguirre, Jean Bennett, Tomas S Aleman, Artur V Cideciyan, Jeannette Bennicelli, Nadine S Dejneka, Susan E Pearce-Kelling, Albert M Maguire, Krzysztof Palczewski,[...]. Mol Ther 2005
Gregory M Acland, Gustavo D Aguirre, Jean Bennett, Tomas S Aleman, Artur V Cideciyan, Jeannette Bennicelli, Nadine S Dejneka, Susan E Pearce-Kelling, Albert M Maguire, Krzysztof Palczewski,[...]. Mol Ther 2005
15
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
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
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DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice.
Zongchao Han, Shannon M Conley, Rasha S Makkia, Mark J Cooper, Muna I Naash. J Clin Invest 2012
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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
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
15
Novel AAV serotypes for improved ocular gene transfer.
Corinna Lebherz, Albert Maguire, Waixing Tang, Jean Bennett, James M Wilson. J Gene Med 2008
Corinna Lebherz, Albert Maguire, Waixing Tang, Jean Bennett, James M Wilson. J Gene Med 2008
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Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium.
G Le Meur, K Stieger, A J Smith, M Weber, J Y Deschamps, D Nivard, A Mendes-Madeira, N Provost, Y Péréon, Y Cherel,[...]. Gene Ther 2007
G Le Meur, K Stieger, A J Smith, M Weber, J Y Deschamps, D Nivard, A Mendes-Madeira, N Provost, Y Péréon, Y Cherel,[...]. Gene Ther 2007
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Towards mutation-independent silencing of genes involved in retinal degeneration by RNA interference.
S M Cashman, E A Binkley, R Kumar-Singh. Gene Ther 2005
S M Cashman, E A Binkley, R Kumar-Singh. Gene Ther 2005
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Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model.
Alessandra Tessitore, Fabiana Parisi, Michela Alessandra Denti, Mariacarmela Allocca, Umberto Di Vicino, Luciano Domenici, Irene Bozzoni, Alberto Auricchio. Mol Ther 2006
Alessandra Tessitore, Fabiana Parisi, Michela Alessandra Denti, Mariacarmela Allocca, Umberto Di Vicino, Luciano Domenici, Irene Bozzoni, Alberto Auricchio. Mol Ther 2006
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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
14
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
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
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
14
Strategems in vitro for gene therapies directed to dominant mutations.
S Millington-Ward, B O'Neill, G Tuohy, N Al-Jandal, A S Kiang, P F Kenna, A Palfi, P Hayden, F Mansergh, A Kennan,[...]. Hum Mol Genet 1997
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Prevalence of disease-causing mutations in families with autosomal dominant retinitis pigmentosa: a screen of known genes in 200 families.
Lori S Sullivan, Sara J Bowne, David G Birch, Dianna Hughbanks-Wheaton, John R Heckenlively, Richard Alan Lewis, Charles A Garcia, Richard S Ruiz, Susan H Blanton, Hope Northrup,[...]. Invest Ophthalmol Vis Sci 2006
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13
Functional cone rescue by RdCVF protein in a dominant model of retinitis pigmentosa.
Ying Yang, Saddek Mohand-Said, Aude Danan, Manuel Simonutti, Valérie Fontaine, Emmanuelle Clerin, Serge Picaud, Thierry Léveillard, José-Alain Sahel. Mol Ther 2009
Ying Yang, Saddek Mohand-Said, Aude Danan, Manuel Simonutti, Valérie Fontaine, Emmanuelle Clerin, Serge Picaud, Thierry Léveillard, José-Alain Sahel. Mol Ther 2009
13
In contrast to AAV-mediated Cntf expression, AAV-mediated Gdnf expression enhances gene replacement therapy in rodent models of retinal degeneration.
Prateek K Buch, Robert E MacLaren, Yanaí Durán, Kamaljit S Balaggan, Angus MacNeil, Frank C Schlichtenbrede, Alexander J Smith, Robin R Ali. Mol Ther 2006
Prateek K Buch, Robert E MacLaren, Yanaí Durán, Kamaljit S Balaggan, Angus MacNeil, Frank C Schlichtenbrede, Alexander J Smith, Robin R Ali. Mol Ther 2006
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AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa.
Alexander J Smith, Frank C Schlichtenbrede, Marion Tschernutter, James W Bainbridge, Adrian J Thrasher, Robin R Ali. Mol Ther 2003
Alexander J Smith, Frank C Schlichtenbrede, Marion Tschernutter, James W Bainbridge, Adrian J Thrasher, Robin R Ali. Mol Ther 2003
13
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.