Nature

A lentiviral vector for the production of T cells with an inducible transgene and a constitutively expressed tumour-targeting receptor

Vectors that facilitate the engineering of T cells that can better harness endogenous immunity and overcome suppressive barriers in the tumour microenvironment would help improve the safety and ...
The New England Journal of Medicine

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK ...
The New England Journal of Medicine

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

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 ...
Nature

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

Gene therapy mediated by recombinant adeno-associated virus (AAV) vectors is a promising treatment for systemic monogenic diseases. However, vector immunogenicity represents a major limitation to gene ...