Feasibility of blood testing combined with PET-CT to screen for cancer and guide intervention

Anne Marie Lennon, Adam H. Buchanan, Isaac Kinde, +37

Science

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Hojun Li, Virginia Haurigot, Yannick Doyon, +17

Nature

Vivo GenomeKnockout MouseEngineeringGenome SurgeryGenetics +10

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Salima Hacein‐Bey‐Abina, Sung‐Yun Pai, H. Bobby Gaspar, +45

New England Journal of Medicine

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

Salima Hacein-Bey Abina, H. Bobby Gaspar, Johanna Blondeau, +29

JAMA

The host genomic environment of the provirus determines the abundance of HTLV-1–infected T-cell clones

Nicolas Gillet, Nirav Malani, Anat Melamed, +7

Blood

Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

Eirini P. Papapetrou, Gabsang Lee, Nirav Malani, +11

Nature Biotechnology

Induced Pluripotent Stem CellGenomic Safe HarborsGeneticsStem Cell EngineeringHematology +8

<i>piggyBac</i> transposase tools for genome engineering

Xianghong Li, Erin R. Burnight, Ashley L. Cooney, +10

Proceedings of the National Academy of Sciences

Assessing the potential for AAV vector genotoxicity in a murine model

Hojun Li, Nirav Malani, Shari R Hamilton, +9

Blood

HIV Integration Targeting: A Pathway Involving Transportin-3 and the Nuclear Pore Protein RanBP2

Karen E. Ocwieja, Troy Brady, Keshet Ronen, +11

PLoS Pathogens

Viral ReplicationImmunologyGag ReplacementMolecular BiologyGenome-wide Sirna Screens +18

BET proteins promote efficient murine leukemia virus integration at transcription start sites

Amit Sharma, Ross C. Larue, Matthew Plumb, +12

Proceedings of the National Academy of Sciences

Viral ReplicationImmunologyMolecular BiologyImmunodominanceBet Proteins +15