Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

Interactions between newly integrate DNA and the host genome limit the reliability and
safety of transgene integration for therapeutic cell engineering and other applications …

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

Introduction Wiskott-Aldrich syndrome (WAS) is a primary immunodeficiency characterized
by eczema, thrombocytopenia, infections, and a high risk of developing autoimmunity and …

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

The success of adoptive T cell gene transfer for treatment of cancer and HIV is predicated on
generating a response that is both durable and safe. We report long-term results from three …

The β-haemoglobinopathies are the most prevalent inherited disorders worldwide. Gene
therapy of β-thalassaemia is particularly challenging given the requirement for massive …

Hematopoietic stem/progenitor cells (HSPCs) are capable of supporting the lifelong
production of blood cells exerting a wide spectrum of functions. Lentiviral vector HSPC gene …

Background The outcomes of gene therapy to correct congenital immunodeficiencies are
unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked …

Mouse haematopoietic stem cells (HSCs) undergo a postnatal transition in several
properties, including a marked reduction in their self-renewal activity. We now show that the …