Engineering retroviruses into vectors of therapeutic value has been a highly pursued effort. To date, the promise of retroviral-mediated gene delivery to treat or even cure genetic diseases has been ...
The location of integration of a gene therapy has been crucial for the safety and efficacy of the treatment to cure infants with X-linked severe combined immunodeficiency. Identified integrome ...
In previous clinical trials involving children with X-linked severe combined immunodeficiency (SCID-X1), a Moloney murine leukemia virus–based γ-retrovirus vector expressing interleukin-2 receptor ...
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