A transfer-RNA-derived small RNA regulates ribosome biogenesis

Hak Kyun Kim, Gabriele Fuchs, Shengchun Wang, +13

Nature

Rna BiologySmall RnaRna TransportGene ExpressionMedicine +3

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Leszek Lisowski, Allison Dane, Kirk Chu, +7

Nature

Allelic VariantXenotransplantationHepatologyVariant InterpretationMedicine +9

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Nicole K. Paulk, Katja Pekrun, Erhua Zhu, +17

Molecular Therapy

Risk and Prevention of Anti-factor IX Formation in AAV-Mediated Gene Transfer in the Context of a Large Deletion of F9

Paul Fields, Valder R. Arruda, Elina Armstrong, +5

Molecular Therapy

GeneticsAav VectorImmunologyImmune RegulationPathology +18

Posttranslational modifications of recombinant myotube-synthesized human factor IX

Valder R. Arruda, James N. Hagstrom, Jeffrey S. Deitch, +8

Blood

A tRNA-Derived Small RNA Regulates Ribosomal Protein S28 Protein Levels after Translation Initiation in Humans and Mice

Hak Kyun Kim, Jianpeng Xu, Kirk Chu, +6

Cell Reports

Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant

Sandra P. Melo, Leszek Lisowski, Elizaveta Bashkirova, +6

Molecular Therapy

miR-122 removal in the liver activates imprinted microRNAs and enables more effective microRNA-mediated gene repression

Paul N. Valdmanis, Hak Kyun Kim, Kirk Chu, +5

Nature Communications

Genomic Sequence and Transcription Start Site for the Human γ-Glutamyl Carboxylase

Sheue-Mei Wu, Darrel W. Stafford, L D Frazier, +5

Blood

RNA interference–induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice

Paul N. Valdmanis, Shuo Gu, Kirk Chu, +9

Nature Medicine

Hepatotoxicity ResultsMedicineLiver PhysiologyNatural SciencesImmunology +8