Highlights•CAR T cells are associated with unique side-effects.•Cytokine release syndrome
(CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) and cytopenias are …

More than two decades have passed since genetically modified HIV was used for gene
delivery. Through continuous improvements these early marker gene-carrying HIVs have …

Introduction Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …

Wiskott-Aldrich syndrome (WAS) is characterized by microthrombocytopenia,
immunodeficiency, autoimmunity, and susceptibility to malignancies. In our hematopoietic …

Background In previous clinical trials involving children with X-linked severe combined
immunodeficiency (SCID-X1), a Moloney murine leukemia virus–based γ-retrovirus vector …

X-linked SCID (SCID-X1) is amenable to correction by gene therapy using conventional
gammaretroviral vectors. Here, we describe the occurrence of clonal T cell acute …

Cell-based therapies are fast-growing forms of personalized medicine that make use of the
steady advances in stem cell manipulation and gene transfer technologies. In this Review, I …

Gene therapies are gaining momentum as promising early successes in clinical studies
accumulate and examples of regulatory approval for licensing increase. Investigators are …

γ-Retroviral vectors (γRVs), which are commonly used in gene therapy, can trigger
oncogenesis by insertional mutagenesis. Here, we have dissected the contribution of vector …

Realizing the therapeutic potential of human induced pluripotent stem (iPS) cells will require
robust, precise and safe strategies for genetic modification, as cell therapies that rely on …