CRISPR is becoming an indispensable tool in biological research. Once known as the
bacterial immune system against invading viruses, the programmable capacity of the Cas9 …

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 …

RNA-based therapeutics, such as small-interfering (siRNAs), microRNAs (miRNAs),
antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR–Cas9, have …

The CRISPR-Cas9 RNA-guided DNA endonuclease has contributed to an explosion of
advances in the life sciences that have grown from the ability to edit genomes within living …

Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Programmable DNA cleavage using CRISPR–Cas9 enables efficient, site-specific genome
engineering in single cells and whole organisms. In the research arena, versatile CRISPR …

The development of a method for genome editing based on CRISPR–Cas9 technology was
awarded The Nobel Prize in Chemistry in 2020, less than a decade after the discovery of all …

With the recent development of CRISPR technology, it is becoming increasingly easy to
engineer the genome. Genome-editing systems based on CRISPR, as well as transcription …

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 …

Genome editing is a powerful new tool for making precise alterations to an organism's
genetic material. Recent scientific advances have made genome editing more efficient …