CRISPR-Cas9

Aug 22, 2025

Vertex Pharmaceuticals and CRISPR Therapeutics have become the first companies to secure FDA approval for a CRISPR-based gene-editing therapy. Vertex Pharmaceuticals, in partnership with CRISPR Therapeutics, has achieved a historic milestone with the FDA approval of exagamglogene autotemcel (exa-cel, branded as ...

Aug 23, 2024

Neuromuscular disorders (NMD) encompass a broad spectrum of conditions impacting the peripheral nervous system, which comprises all motor and sensory nerves linking the brain and spinal cord to the body. These disorders comprise various classes of diseases, such as muscular dystrophies, myopathies, motor neuron dis...

Feb 05, 2024

Gene therapy is an innovative medical approach that manipulates an individual’s genetic material to prevent or treat diseases. It primarily involves introducing, modifying, or substituting genes within a patient’s cells. The central objective of gene therapy is to rectify genetic abnormalities, insert missing genes...

Jun 10, 2022

CRISPR technology or CRISPR/Cas9 technology is a genome-editing tool derived from the bacterial defense system against viruses and plasmids. The system comprises the Cas9 nuclease enzyme to create site-directed dsDNA (double-stranded DNA) break and a guide RNA, which is a predesigned 20 bp long RNA sequence within ...

Jan 09, 2018

Key to new Cancer drugs discovered in bacterial genomes Achievement is made by the scientists of The Scripps Research Institute (TSRI) Florida campus who had previously discovered LNM E1 compound used to kill prostate cancer. This time they have unlocked the capability of LNM family molecules by “mining” the in...