In the last few weeks, excitement regarding a potential new approach (CRISPR/Cas9) to treating DMD has grown. Science recently published several individual labs' work demonstrating the ability to correct the basal genetic defect in DMD in the mouse model of DMD. These exciting results have been shared in several prominent media outlets, including the New York Times, The Guardian, and the BBC.
Though I'm no expert, CRISPR/Cas9 certainly is one of the most revolutionary developments in science and medicine in the last year, and will be something to keep a sharp eye on moving forward. Different than all previous therapies, CRISPR/Cas9 edits the actual genome (DNA), rather than transcriptome (RNA), or protein, leading to permanent changes within the host. This opens up many more questions than answers. Is it ethical to modify the genome or, how can this be used to treat patients with unique and individual mutations?
Unleashing the capacity of CRISPR/Cas9 is leading to a new epoch of science and medicine, and it will be exciting to see how we can utilize it to treat the muscular dystrophies.
Though a few days late, I thought it'd be worthwhile to share a few reflections of the first year of this website. Like all years, 2015 had its share of ups and downs, but for the most part was a very good year.
The undeniably most significant happening of the year was going to Nepal for a month with Rosha after the earthquake. Having our boots on the ground, literally kilometers from the epicenter, and being able to really pitch in our combined efforts to try and help with the recovery was one of the most meaningful things I've done in my life. As beautiful of a country as Nepal is, in the heaven's of Earth, the people are even more beautiful. The hospitality and generosity that was given to us, from many who have next to nothing, is something I'll never forget.
Grad school is trudging along, just as it has been the previous three years. I'm anticipating to defend my studies in March/April, so that is something certainly worth looking forward to. Beyond grad school, I'll be jumping back into the med school, starting as an MS-3 come May. I'm equally petrified and excited for med school to return, as I haven't worked with patients in quite sometime, but look forward to the human-to-human interactions that it offers.
I was privileged to participate in a number of conferences and research meetings throughout the year. Meeting, and getting to talk to Francis Collins was certainly the highpoint of my year in regards to professionally networking.
Scattered throughout the year were trips with friends, family, and loved ones to a number of places. From graduations to road trips, experiencing this journey called "life" with the people closest to me make all of this journey entirely worthwhile.
It's been a good year and I wish everyone the best for 2016!
I'm a current MD-PhD candidate, working hard to help treat and manage muscular dystrophies