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Bertoni, Carmen
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1

Targeting a therapeutic LIF transgene to muscle via the imm..:

Welc, Steven S. ; Flores, Ivan ; Wehling-Henricks, Michelle...
Nature Communications.  10 (2019)  1 - p. , 2019
Link: https://doi.org/10.1038/..
 
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2

Clinical potential of ataluren in the treatment of Duchenne..:

Bertoni, Carmen ; Namgoong, John
Degenerative Neurological and Neuromuscular Disease.  , 2016
Link: https://doi.org/10.2147/..
 
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3

Contributors:

, In: Translating Gene Therapy to the Clinic,
Angelos, Mathew G. ; Baglieri, Jacopo ; Bertoni, Carmen... - p. xv-xvi , 2015
Link: https://doi.org/10.1016/..
 
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4

Genetic and Cell-Mediated Therapies for Duchenne Muscular D..:

, In: Translating Gene Therapy to the Clinic,
Baglieri, Jacopo ; Bertoni, Carmen - p. 147-171 , 2015
Link: https://doi.org/10.1016/..
 
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5

High Throughput Screening in Duchenne Muscular Dystrophy: F..:

Gintjee, Thomas ; Magh, Alvin ; Bertoni, Carmen
Biology.  3 (2014)  4 - p. 752-780 , 2014
Link: https://doi.org/10.3390/..
 
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6

Emerging gene editing strategies for Duchenne muscular dyst..:

Bertoni, Carmen
Frontiers in Physiology.  5 (2014)  - p. , 2014
Link: https://doi.org/10.3389/..
 
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7

Ex Vivo Gene Editing of the Dystrophin Gene in Muscle Stem ..:

Nik-Ahd, Farnoosh ; Bertoni, Carmen
Stem Cells.  32 (2014)  7 - p. 1817-1830 , 2014
Link: https://doi.org/10.1002/..
 
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8

A high-content, high-throughput siRNA screen identifies cyc..:

Khanjyan, Michael V. ; Yang, Jonathan ; Kayali, Refik..
Human Molecular Genetics.  22 (2013)  16 - p. 3283-3295 , 2013
Link: https://doi.org/10.1093/..
 
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9

Read-through compound 13 restores dystrophin expression and..:

Kayali, Refik ; Ku, Jin-Mo ; Khitrov, Gregory...
Human Molecular Genetics.  21 (2012)  18 - p. 4007-4020 , 2012
Link: https://doi.org/10.1093/..
 
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10

Arginine-rich cell-penetrating peptide dramatically enhance..:

Du, Liutao ; Kayali, Refik ; Bertoni, Carmen...
Human Molecular Genetics.  20 (2011)  16 - p. 3151-3160 , 2011
Link: https://doi.org/10.1093/..
 
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11

Site-directed gene repair of the dystrophin gene mediated b..:

Kayali, Refik ; Bury, Frederic ; Ballard, McIver.
Human Molecular Genetics.  19 (2010)  16 - p. 3266-3281 , 2010
Link: https://doi.org/10.1093/..
 
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12

34. Phi C31 Integrase System Enhances Dystrophin Gene Expre..:

Bertoni, Carmen ; Jarrahian, Sohail ; Wheeler, Thurman M....
Molecular Therapy.  13 (2006)  - p. S14-S15 , 2006
Link: https://doi.org/10.1016/..
 
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13

573. Enhanced Level of Gene Correction Mediated by Oligonuc..:

Bertoni, Carmen ; Rustagi, Arjun ; Rando, Thomas A.
Molecular Therapy.  13 (2006)  - p. S221 , 2006
Link: https://doi.org/10.1016/..
 
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14

Enhancement of Plasmid-Mediated Gene Therapy for Muscular D..:

Bertoni, Carmen ; Jarrahian, Sohail ; Wheeler, Thurman M....
Proceedings of the National Academy of Sciences of the United States of America.  103 (2006)  2 - p. 419-424 , 2006
Link: https://www.jstor.org/st..
 
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15

Enhancement of plasmid-mediated gene therapy for muscular d..:

Bertoni, Carmen ; Jarrahian, Sohail ; Wheeler, Thurman M....
Proceedings of the National Academy of Sciences.  103 (2005)  2 - p. 419-424 , 2005
Link: https://doi.org/10.1073/..
 
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