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 …

Improvements in the gene transfer vectors used in therapeutic trials have led to substantial
clinical successes in patients with serious genetic conditions, such as immunodeficiency …

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 severe combined immunodeficiency (SCID-X1) is a profound deficiency of T, B, and
natural killer (NK) cell immunity caused by mutations in IL2RG encoding the common chain …

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 …

Zinc-finger nucleases (ZFNs) allow gene editing in live cells by inducing a targeted DNA
double-strand break (DSB) at a specific genomic locus. However, strategies for …

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 …

T cell-based adoptive immunotherapy has been shown to be a promising treatment for
various types of cancer. However, adoptive T cell therapy currently requires the custom …