Inborn errors of immunity (IEI) are diseases caused by genetic mutations that affect the
immune system's ability to fight pathogens, cope with the microbiota or regulate�…

Background The DNA-repair enzyme Artemis is essential for rearrangement of T-and B-cell
receptors. Mutations in DCLRE1C, which encodes Artemis, cause Artemis-deficient severe�…

Primary immunodeficiencies (PID) are rare, complex diseases that can be characterised by
a spectrum of phenotypes, from increased susceptibility to infections to autoimmunity�…

Cell therapies that rely on engineered immune cells can be enhanced by achieving uniform
and controlled transgene expression in order to maximize T-cell function and achieve�…

Advanced Therapy Medicinal Products (ATMPs) comprising cell, gene, and tissue-
engineered therapies have demonstrated enormous therapeutic benefits. However, their�…

Abstract We present Dystrophic Epidermolysis Bullosa Cell Therapy (DEBCT), a scalable
platform producing autologous organotypic iPS cell-derived induced skin composite (iSC)�…

Although IL-2 was first recognized as growth factor for T cells, it is now also appreciated to
be a key regulator of T cells through its effects on regulatory T cells (Treg). The IL-2 receptor�…

X-linked severe combined immunodeficiency (X-SCID) is a primary immunodeficiency that is
caused by mutations in the interleukin-2 receptor gamma (IL2RG) gene. Some patients�…

Patients with inborn errors of immunity (IEI) have been transplanted for more than 50 years.
Many long-term survivors have ongoing medical issues showing the need for further�…

Retroviral gene therapy has emerged as a promising therapeutic modality for multiple
inherited and acquired human diseases. The capability of delivering curative treatment or�…