Recent advances in the generation, purification and cellular delivery of RNA have enabled
development of RNA-based therapeutics for a broad array of applications. RNA therapeutics …

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 …

Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs)
engineered with integrating vectors is a promising treatment for monogenic diseases, but …

CD3δ SCID is a devastating inborn error of immunity caused by mutations in CD3D,
encoding the invariant CD3δ chain of the CD3/TCR complex necessary for normal …

Molecular, cellular, and clinical studies of human inborn errors of immunity have
revolutionized our understanding of their pathogenesis, considerably broadened their …

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 …

The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system has
revolutionized the field of gene editing. Continuous efforts in developing this technology …

Hematopoietic stem-cell gene therapy has proven to be an effective treatment for several
primary immunodeficiencies, and yet companies in this space are withdrawing from the EU …

Twenty genetic therapies have been approved by the US Food and Drug Administration to
date, a number that now includes the first CRISPR genome-editing therapy for sickle cell …

Despite the ups and downs in the field over three decades, the science of gene therapy has
continued to advance and provide enduring treatments for increasing number of diseases …