CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …

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 …

Acute myeloid leukaemia (AML) represents a set of heterogeneous myeloid malignancies,
and hallmarks include mutations in epigenetic modifiers, transcription factors and kinases …

Antiadhesion barriers such as films and hydrogels used to wrap repaired tendons are
important for preventing the formation of adhesion tissue after tendon surgery. However …

Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …

Mechanical activation of fibroblasts, caused by friction and transforming growth factor–β1
recognition, is one of the main causes of tissue adhesions. In this study, we developed a …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Abstract Hematopoietic Stem Cell (HSC) gene therapy (GT) may provide lifelong
reconstitution of the hematopoietic system with gene-corrected cells1. However, the effects …

Recombination activating genes (RAGs) are tightly regulated during lymphoid differentiation,
and their mutations cause a spectrum of severe immunological disorders. Hematopoietic …

The discovery of CRISPR has allowed site-specific genomic modification to become a reality
and this technology is now being applied in a number of human clinical trials. While this …