Efficient modification of <i>CCR5</i> in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

Blythe Sather, Guillermo S. Romano Ibarra, Karen Sommer, +12

Science Translational Medicine

AAV-mediated gene targeting methods for human cells

Iram Khan, Roli K. Hirata, David W. Russell

Nature Protocols

Aav-mediated GeneVirologyGene VectorMedicineCell Biology

Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors

Thomas R. Bauer, James M. Allen, Mehreen Hai, +8

Nature Medicine

Veterinary VaccineSuccessful TreatmentFoamy Virus VectorsPathogenesisViral Pathogenesis +6

Engineering Protein-Secreting Plasma Cells by Homology-Directed Repair in Primary Human B Cells

King L. Hung, Iana Meitlis, Malika Hale, +7

Molecular Therapy

Homology-directed RepairHuman CellProtein SecretionMedicineImmunology +7

Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

Nicholas Hubbard, David Hagin, Karen Sommer, +7

Blood

Engineering of Human Pluripotent Stem Cells by AAV-mediated Gene Targeting

Iram Khan, Roli K. Hirata, Pei-Rong Wang, +7

Molecular Therapy

Gene editing to induce FOXP3 expression in human CD4 ⁺ T cells leads to a stable regulatory phenotype and function

Yuchi Honaker, Nicholas Hubbard, Yufei Xiang, +14

Science Translational Medicine

Intraosseous Delivery of Lentiviral Vectors Targeting Factor VIII Expression in Platelets Corrects Murine Hemophilia A

Xuefeng Wang, Simon Shin, Andy F J Chiang, +4

Molecular Therapy

Cell TherapyImmunologyBlood CellBiomedical EngineeringImmune System +23

Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

Cathy X. Wang, Blythe Sather, Xuefeng Wang, +10

Blood

Normal Collagen and Bone Production by Gene-targeted Human Osteogenesis Imperfecta iPSCs

David R. Deyle, Iram Khan, Gaoying Ren, +4

Molecular Therapy