The frontiers of precision medicine have been revolutionized by the development of
Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR)/Cas9 as an editing …

More than two decades have passed since genetically modified HIV was used for gene
delivery. Through continuous improvements these early marker gene-carrying HIVs have …

MicroRNAs are small, highly conserved non-coding RNA molecules involved in the
regulation of gene expression. MicroRNAs are transcribed by RNA polymerases II and III …

Lentiviruses such as HIV-1 traverse nuclear pore complexes (NPC) and infect terminally
differentiated non-dividing cells, but how they do this is unclear. The cytoplasmic NPC …

The first gene therapy clinical trials were initiated more than two decades ago. In the early
days, gene therapy shared the fate of many experimental medicine approaches and was …

The concept of gene therapy originated in the mid twentieth century and was perceived as a
revolutionary technology with the promise to cure almost any disease of which the molecular …

Lentiviral vectors (LVs) have emerged as potent and versatile vectors for ex vivo or in vivo
gene transfer into dividing and nondividing cells. Robust phenotypic correction of diseases …

The potential benefits of gene therapy for neurological diseases such as Parkinson's,
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …

Besides its broad application in research and biotechnology, genome editing (GE) has great
potential for clinical gene therapy, but delivery of GE tools remains a bottleneck. Whereas …

The CRISPR/Cas9 systems have revolutionized the field of genome editing by providing
unprecedented control over gene sequences and gene expression in many species …