Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

To elucidate principles operating in native biological systems and to develop novel
biotechnologies, synthetic biology aims to build and integrate synthetic gene circuits within …

Understanding how transcriptional enhancers control over 20,000 protein-coding genes to
maintain cell-type-specific gene expression programs in all human cells is a fundamental …

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 …

Developmental gene expression is often controlled by distal regulatory DNA elements called
enhancers. Distant enhancer action is restricted to structural chromosomal domains that are …

A picture is beginning to emerge from a variety of organisms that for a subset of genes, the
most important sequences that regulate expression are situated not in the promoter but …

Transcriptional dysregulation of the MYC oncogene is among the most frequent events in
aggressive tumor cells, and this is generally accomplished by acquisition of a super …

Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent
and severe monogenic disorder, SCD has been long considered a logical candidate for …

Spearheaded by the therapeutic use of chimeric antigen receptors (CARs) targeting CD19,
synthetic immunology has entered the clinical arena. CARs are recombinant receptors for …