On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …

Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an
excellent safety record. They provide evidence for the long-sought promise of gene therapy …

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

Genome sequencing studies have shown that human malignancies often bear mutations in
four or more driver genes, but it is difficult to recapitulate this degree of genetic complexity in …

Background Severe combined immunodeficiency due to adenosine deaminase (ADA)
deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency. Methods We …

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

Previously, several individuals with X-linked SCID (SCID-X1) were treated by gene therapy
to restore the missing IL-2 receptor γ (IL2RG) gene to CD34+ BM precursor cells using …

Cancer therapy has entered a new era, transitioning from unspecific chemotherapeutic
agents to increasingly specific immune-based therapeutic strategies. Among these, chimeric …

Wiskott‐Aldrich syndrome (WAS) is a rare X‐linked primary immunodeficiency characterized
by microthrombocytopenia, eczema, recurrent infections, and an increased incidence of …

The first gene therapy clinical trials were initiated more than two decades ago. In the early
days, gene therapy shared the fate of many experimental medicine approaches and was …