Spotlight on Gene Therapy: finding a Cure

Florie Borel presented our lab's work at the 20th Annual Basic Research/Morningside Graduate School of Biomedical Sciences Retreat, held at the University of Massachusetts Amherst Conference Center.

The first poster "Therapeutic rAAVrh.10-mediated SOD1 silencing in adult SOD1G93A mice and non-human primates" describes preclinical studies done in ALS mice and non-human primates to evaluate a gene therapy treatment for amyotrophic lateral sclerosis.

The second poster "Simultaneous disruption of five Serpina1 genes in mice using CRISPR/Cas9 to generate the first model of alpha-one antitrypsin deficiency, the leading cause of genetic COPD" describes the generation and characterization of new mouse model of alpha-one antitrypsin deficiency.

Below, second author Brynn Cardozo presenting the AAT poster.