Gene Therapy

The early steps of viral vector production influence cell and gene therapy success.

Neuroscientists safely delivered a critical gene that improved vision for two patients with Leber congenital amaurosis.

Bacterial contaminants in cell cultures pose a significant threat to the biopharmaceutical safety. How do scientists detect these hard-to-spot pests?

Samira Kiani combines her passion for art and design with synthetic biology to create safer CRISPR-based therapeutics for the future.

On a quest to bring new a new hemophilia gene therapy to the clinic, one research team leapt over hurdle after hurdle to produce enough viral therapeutics to treat a large number of patients.

Nanoparticles fuse with cells to deliver their gene or RNA therapy cargo, but some are better shuttles than others. Now, scientists have developed a way to find the best nanoparticles for the job.

Nanoparticles that successfully deliver drugs in mice often fail in humans. To understand how nanoparticles fare across different model species, scientists developed a high-throughput system to screen nanoparticles in mice with humanized livers with the hope of better predicting nanoparticles that work in humans.

While the blood-brain barrier is a significant obstacle to gene therapy, directed evolution of viral capsids helps ferry viral vectors across it.

Discover a novel approach to vaccine discovery using CRISPR engineering.

LIVE JUNE 23rd, 2022 at 2:30 PM
Uncover genomic and epigenomic variations within a group of cells using single cell sequencing.