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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
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Genetic and virulence characteristics of a Raoultella planticola isolate resistant to carbapenem and tigecycline
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Genomic and transcriptomic profiling reveals distinct molecular subsets associated with outcomes in mantle cell lymphoma
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Multifaceted characterization of the signatures and efficacy of mesenchymal stem/stromal cells in acquired aplastic anemia
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Customized blood-brain barrier shuttle peptide to increase AAV9 vector crossing the BBB and augment transduction in the brain
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Yuan, Yi, Li, Ying, Wang, Guangxi, Li, Chengwen, Xiang, Li, She, Junping, Yang, Yan, Zhong, Fangcai, Zhang, Luhua
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Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
Asokan, Aravind, Conway, Julia C, Phillips, Jana L, Li, Chengwen, Hegge, Julia, Sinnott, Rebecca, Yadav, Swati, DiPrimio, Nina, Nam, Hyun-Joo, Agbandje-McKenna, Mavis, McPhee, Scott, Wolff, Jon, Samulski, R Jude
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Minimal residual disease monitoring in childhood Philadelphia chromosome-positive acute lymphoblastic leukemia: prognostic significance and correlation between multiparameter flow cytometry and real-time quantitative polymerase chain reaction
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Pei, Xiaolei, He, Ting, Hall, Nikita E., Gerber, David, Samulski, R. Jude, Li, Chengwen
Published in Virology (New York, N.Y.) (01.05.2018)
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The impact of clone size on the prognostic value of chromosome aberrations by fluorescence in situ hybridization in multiple myeloma
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Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial
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Published in Human gene therapy (01.02.2015)
Published in Human gene therapy (01.02.2015)
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