Article Link: Genome Modification Provides Hope for Gene Therapy – Rafael Casellas, NIH Scientist
NIH IRP (Intramural Research Program at the National Institutes of Health), Jun 13, 2013
>>Raphael Casellas, Ph.D.: The main goal of our laboratory is to understand how the B lymphocyte develops. And to do that we have taken an approach where we want to study the processes that are taking place in the nucleus of the B cell.
These are cells from the immune system that produce antibody molecules, these are the proteins that cross-react with pathogens when you get an infection. In the nucleus, where most of the action is happening in terms of what the B cell will do, we try to study what those signals getting from the outside are changing in the nucleus so that the cell can respond.
To understand how these processes are orchestrated, one thing that we’ve been trying to develop is a technique to modify the genome at will. And in order to do that we have to develop a process where we can introduce specific mutations or deletions across the genome at a very specific site.
So one technique we have been using is the TALEN(s) approach, which are small scissors that introduce DNA breaks at those specific sites so we can go in and make modifications.And what Kyongrim is doing now is she’s assembling those scissors so we can do this genome engineering.
>>Stevenson Nelson, Ph.D.: Essentially, anything that you do with a pipette, in terms of moving liquids, you can do with this machine. For our purposes the main function of this machine is to assemble part of the TALEN technology. It’s going to take — pick up samples from various locations, pull them together, and then allow us to essentially do digestion and ligation in the same reaction. So for us, it’s really just built to move a lot of different components one by one by one, to assemble them.
>>Raphael Casellas, Ph.D.: And here is where the data will be acquired from those experiments. And this is a deep sequencer. It means that it can sequence millions and millions of small short pieces of DNA. And the data has to be processed and analyzed. So those are the next stages in this whole experiment.
>> Stephen Fillini, Ph.D.: So this is a core switch. This is where all the elements of the system come in, both storage systems connect to this, and each of the end switches connect to this.
>> My name is Steve Fellini, I’m the systems architect of the Biowulf cluster at the NIH. It’s used entirely for scientific computing; it’s used by the intramural researchers at NIH for just about every sort of biomedical application you can think of.
>>Raphael Casellas, Ph.D.: So this is where all the data is being analyzed with bio-informatic tools. So this is the last stage before we actually pull all these discoveries together into a complete story.
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