gene therapy

In this webinar, Cole Cheng and Connie Rich will discuss the advantages of novel miniaturized plasmids for streamlining preclinical and clinical gene therapy development.

Even as CRISPR-based tools become a lab staple, scientists strive to tackle the associated technological challenges to improve their efficacy and safety.   

By postnatally providing a transcription factor important for brain development, researchers fixed abnormalities generated in utero in mice.

Discover how innovative production and clarification products improve lentiviral vector generation.

An optimized version of prime editing technology raises the possibility of a one-time treatment for cystic fibrosis.

Off-the-shelf and custom plasmid DNA and viral vector products help scientists take their gene therapy research to the next level.

An innovative helper plasmid improves adeno-associated virus quality and infectivity.

Standardized quality materials and reagents help scientists improve cell and gene therapy production.

As a trailblazer in rare disease research and treatment, Wendy Chung captures the big picture of rare disease genetics with the help of next generation sequencing.

An orthogonal method to cell culture speeds up testing for AAV and lentivirus vectors.

In this webinar, Douglas Marchuk and Viviana Gradinaru will discuss how scientists can overcome physiological barriers preventing gene therapies from reaching the brain. 

CRISPR-carrying lipid nanoparticles enabled researchers to correct a rare nonsense mutation in the lungs of a cystic fibrosis mouse model.

Base editors and prime editors help researchers perform more precise in vivo and ex vivo translational research.

With the help of directed evolution, scientists inch closer to developing viral vectors that can cross the human blood-brain barrier to deliver gene therapy.

Ten years ago, scientists put DNA barcodes in AAV vectors, creating an approach that simplified, expedited, and streamlined AAV screening. 

A risk-benefit analysis of gene editing tools in stem cells revealed that base and prime editing carry vulnerabilities similar to those of CRISPR-Cas9, but at a reduced rate. 

Take the leap from shake flasks to bioreactors.

Lauren Drouin shares how personal connections and scientific curiosities drive her work on gene therapy viral vectors. 

Scientists removed unnecessary sections of the Cas13 enzyme, creating a mini-enzyme that works and fits with other CRISPR elements into a single gene therapy vector.