BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

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 …

Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …

HIV prevalence is increasing worldwide because people on antiretroviral therapy are living
longer, although new infections decreased from 3· 3 million in 2002, to 2· 3 million in 2012 …

AIDS remains incurable due to the permanent integration of HIV-1 into the host genome,
imparting risk of viral reactivation even after antiretroviral therapy. New strategies are …

Genome engineering with programmable nucleases depends on cellular responses to a
targeted double-strand break (DSB). The first truly targetable reagents were the zinc finger …

Purpose As a powerful technology for genome engineering, the CRISPR/Cas system has
been successfully applied to modify the genomes of various species. The purpose of this …

Current technology enables the production of highly specific genome modifications with
excellent efficiency and specificity. Key to this capability are targetable DNA cleavage …

The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases
has become a clinical standard but is limited by the availability of suitable matched donors …

Transplantation of hematopoietic stem cells (HSCs) with a naturally occurring CCR5
mutation confers a loss of detectable HIV-1 in the patient, making ablation of the CCR5 gene …