To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

Cardiovascular disease remains the leading cause of morbidity and mortality in the
developed world. In recent decades, extraordinary effort has been devoted to defining the …

Prolonged expression of the CRISPR-Cas9 nuclease and gRNA from viral vectors may
cause off-target mutagenesis and immunogenicity. Thus, a transient delivery system is …

Despite the utility of CRISPR–Cas9 nucleases for genome editing, the potential for off-target
activity limits their application, especially for therapeutic purposes,. We developed a yeast …

Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic
genome editing but are severely constrained by cargo limits. Simultaneous delivery of …

CRISPR systems have emerged as transformative tools for altering genomes in living cells
with unprecedented ease, inspiring keen interest in increasing their specificity for perfectly …

Emerging clustered regularly interspaced short palindromic repeat/associated protein
(CRISPR/Cas) genome editing technology shows great potential in gene therapy. However …

Huntington's disease (HD) is a currently incurable and, ultimately, fatal neurodegenerative
disorder caused by a CAG trinucleotide repeat expansion within exon 1 of the huntingtin …

In recent years, CRISPR–Cas9 technology is widely acknowledged for having major
applications in the field of biotechnology for editing genome of any organism to treat a …

CRISPR–Cas9 is the state-of-the-art programmable genome-editing tool widely used in
many areas. For safe therapeutic applications in clinical medicine, its off-target effect must …