AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice

<i>LMO2</i> -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

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Life Sciences, Genetics, Immunology +17

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

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Life Sciences, Hematology, Molecular Genetics +9

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

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Medicine, Molecular Genetics, Oncology +14

The Liver: Biology and Pathobiology.

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Life Sciences, Biochemistry, Cell Biology +17

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

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Life Sciences, Molecular Biology, Molecular Genetics +15