gene therapy

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

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

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. 

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.

In the race to deliver successful gene therapies, frontrunner AAVs come head to head with underdog lipid nanoparticles. 

A discovery that goes back to the first studies of translation has become the topic of biotech buzz.

An aqueduct connecting the brain to the ear may make gene therapy for hearing loss less invasive.

Gene therapy may be the first step toward curing a rare genetic epilepsy.

By delivering mRNA to the skin of mice in utero, researchers showed a proof-of-concept for shuttling gene therapies to skin cells before birth.

In both the laboratory and clinic, scientists harness viral genetic transfer capabilities to develop gene therapies that modulate cellular function.