Gene editing has emerged as a powerful approach for targeting the genetic causes of disease, but getting the editing ...

Gene therapy could potentially cure genetic diseases but it remains a challenge to package and deliver new genes to specific cells safely and effectively. Existing methods of engineering one of the ...

The landscape of gene editing applications is expanding quickly. More and more, researchers are faced with choosing the right tools and protocols for genome engineering, including overexpressing ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...

Research teams have created a versatile set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy. These delivery systems are ...

From gene therapy to vaccine development, plasmids are vital tools — but only if they’re designed, optimized, and verified with precision. Advances in codon optimization, long-read sequencing, and ...

For adeno-associated viral gene therapies to scale, scientists must first address safety and manufacturability challenges. Improving payload design and optimizing production systems can help ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ...