In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas
(CRISPR-associated) genome editing systems have become one of the most robust …

The development over the past decade of methods for delivering genes to mammalian cells
has stimulated great interest in the possibility of treating human disease by gene-based …

We have previously shown correction of X-linked severe combined immunodeficiency [SCID-
X1, also known as γ chain (γc) deficiency] in 9 out of 10 patients by retrovirus-mediated γc …

Gene therapy has become a promising strategy for the treatment of many inheritable or
acquired diseases that are currently considered incurable. Non-viral vectors have attracted …

The possible use of pig organs and tissues as xenografts in humans is actively being
considered in biomedical research. We therefore examined whether pig endogenous …

Thirty years after initial publications of the concept of a chimeric antigen receptor (CAR), the
US Food and Drug Administration (FDA) approved the first anti-CD19 CAR T-cell therapy …

CRISPR/Cas9 genome editing platforms are widely applied as powerful tools in basic
research and potential therapeutics for genome regulation. The appropriate alternative of …

Gene therapy holds a great promise for the treatment of acquired and inherited diseases
with a genetic origin that are currently incurable. Non-viral gene delivery systems are …

Recent conceptual and technical improvements have resulted in clinically meaningful levels
of gene transfer into repopulating hematopoietic stem cells. At the same time, evidence is …

Recombinant viral vectors have allowed gene transfer to be developed as a promising
approach to the treatment of genetic diseases. Recently, gene therapy of children with X …