Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Catherine S. Manno, Glenn F. Pierce, Valder R. Arruda, +28

Nature Medicine

InflammationAav-factor IxHost ResponseAutoimmune DiseaseSuccessful Transduction +9

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Amit C. Nathwani, Edward G. D. Tuddenham, Savita Rangarajan, +29

New England Journal of Medicine

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

Dirk Grimm, Konrad L. Streetz, Catherine L. Jopling, +6

Nature

MedicineGeneticsNatural SciencesRna BiologyMicrorna Detection +6

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Amit Nathwani, Ulreke M. Reiss, Edward G. D. Tuddenham, +27

New England Journal of Medicine

RNA interference in adult mice

Anton P. McCaffrey, Leonard Meuse, Thu-Thao T. Pham, +3

Nature

Rna InterferenceGeneticsRna BiologyGene ExpressionMedicine +1

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

Mark A. Kay, Catherine S. Manno, Margaret V. Ragni, +12

Nature Genetics

Factor IxImmunogeneticsAutoimmune DiseaseImmunodeficienciesPathogenesis +9

Robust expansion of human hepatocytes in Fah−/−/Rag2−/−/Il2rg−/− mice

Hisaya Azuma, Nicole K. Paulk, Aarati R. Ranade, +7

Nature Biotechnology

InflammationHepatologyLiver PhysiologyImmunologyLiver Disease +5

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Catherine S. Manno, Ah Keng Chew, S Hutchison, +17

Blood

Human tRNA-derived small RNAs in the global regulation of RNA silencing

Dirk Haussecker, Yong Huang, Ashley Lau, +3

RNA

A nonviral minicircle vector for deriving human iPS cells

Fangjun Jia, Kitchener D. Wilson, Ning Sun, +9

Nature Methods

Human CellMedicineCell EngineeringCell BiologyCell Signaling +1