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

The CD47-signal regulatory protein alpha (SIRPa) interaction is a therapeutic target for human solid tumors

Stephen B. Willingham, Jens-Peter Volkmer, Andrew J. Gentles, +41

Proceedings of the National Academy of Sciences

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

Dirk Grimm, Joyce Lee, Lora Wang, +4

Journal of Virology

Viral ReplicationIn Vivo Gene TherapySynthetic VirologyGeneticsImmunology +16

Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8

Katsuya Inagaki, Sally Fuess, Theresa A. Storm, +4

Molecular Therapy

Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo

Hiroyuki Nakai, Stephen R. Yant, Theresa A. Storm, +3

Journal of Virology

Disrupting the CD47-SIRPα anti-phagocytic axis by a humanized anti-CD47 antibody is an efficacious treatment for malignant pediatric brain tumors

Sharareh Gholamin, Siddhartha S. Mitra, Abdullah H. Feroze, +31

Science Translational Medicine

AAV serotype 2 vectors preferentially integrate into active genes in mice

Hiroyuki Nakai, Eugenio Montini, Sally Fuess, +3

Nature Genetics

Active GenesGeneticsPathogenesisViral PathogenesisImmunology +8

Rapid Uncoating of Vector Genomes Is the Key toEfficient Liver Transduction with Pseudotyped Adeno-Associated VirusVectors

Clare E. Thomas, Theresa A. Storm, Zan Huang, +1

Journal of Virology

Unrestricted Hepatocyte Transduction with Adeno-Associated Virus Serotype 8 Vectors in Mice

Hiroyuki Nakai, Sally Fuess, Theresa A. Storm, +3

Journal of Virology

Liver TransductionPortal Vein InjectionHepatologyMedicineLiver Physiology +12

Three differentiation states risk-stratify bladder cancer into distinct subtypes

Jens-Peter Volkmer, Debashis Sahoo, Robert Chin, +17

Proceedings of the National Academy of Sciences

Tumor BiologyUrologyEngineeringGenitourinary CancerTumor Heterogeneity +12