Lara Roach, Post 2

Greetings from The Farm! I am now half-way through my summer here in Palo Alto. So much has happened since my last post! I have been working on developing a protocol for treadmill testing, which will provide functional analysis of the gene therapy approach developed in the lab for Duchenne Muscular Dystrophy. I will be measuring the time until the mice reach exhaustion before and after receiving a plasmid-based therapy aimed at rescuing the production of dystrophin missing in these diseased mice. I will also be performing a technique called eccentric contraction after the treadmill testing to determine the force the muscle can withstand. Since dystrophic mice have muscle degeneration and weakening, they withstand less force than wild-type mice. Our hope is that the gene therapy will increase dystrophin production and also the functional abilities of the mice to run longer and withstand more force. Since the gene therapy approach is plasmid based, involves site-specific integration into the genome, and can be delivered efficiently into muscle through a vein injection, this project is advancing the field of gene therapy for muscular dystrophy and could potentially lead to clinical trials in the future.

            Outside of lab, I have explored Palo Alto as well as San Francisco with the peers in my summer program. We had a barbeque for the 4^th of July, visited Fisherman’s Warf and Ghirardelli Square in SF, and saw Pixar’s Inside Out. Next weekend we will be travelling to Santa Cruz to explore the beach and boardwalk. I cannot believe the summer is already half over, but I am looking forward to making the most out of the next 4 weeks.

Until next time,

Lara

A video of a muscular dystrophic mouse running on the treadmill. I have optimized the running speed and will begin testing mice who will receive gene therapy.

Ghirardelli Square in San Francisco