CRISPR-Cas9

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...

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...

Vertex Pharma and CRISPR Therapeutics are the first companies to seek FDA clearance for a gene-editing therapy. Vertex Pharmaceuticals and CRISPR Therapeutics have gotten closer to introducing exagamglogene autotemcel (exa-cel), a one-time treatment for sickle cell disease (SCD) and transfusion-dependent beta-...

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 ...

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...