CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 53:13-55:55 Final slide, there's lots of good work trying to identify other sources of modification of Marfan syndrome, making it worse or making…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 52:17-53:12 So based upon this, based upon both our hypothesis constrained arduous work but also now by this new technology of mapping modifiers,…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 49:29-53:12 We also found that losartan is tremendously protective even in these 129 mice, so it completely prevented aneurysms from forming and …

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 47:32-49:29 Now around the same time, we recognize that one strain of mouse called the black six (BL6) mouse had very mild aneurysms when we intr…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 43:49-47:31 So fortunately in collaboration with Bart Loeys, Anne De Peapa and Julie DeBacker and others, we've had the good fortune of observing…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 40:30-43:48 And one method to do this was to try to understand families with Marfan syndrome like the one that I'm showing here. 先ほど述べた従来…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 38:20-40:29 So I'm going to end the talk by talking about studying modifiers. 最後は、遺伝子の修飾についてお話します。 So over the course of the …

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 36:57-38:19 It's clear that the ability to make stem cells has already led to significant advances. We're not quite ready to use them therapeutic…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 35:59-36:55 Now there are also some further advanced methods using stem cells that might be applicable to Marfan syndrome. 幹細胞を使った方法にも…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 34:03-35:57 We're also considering how we might deliver this activation machinery to the aorta and specifically to the base of the aorta, the so-…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 32:07-34:06 Now it turns out that there is a fetal form of fibrillin-1, it's a protein called fibrillin-2, just as we've been discussing, it norm…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 29:36-32:06 So I'm going to now tell you about what I think might be a an exciting twist on this type of therapeutic intervention. これまでに述べ…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 27:16-29:35 So what about Marfan syndrome? Let's consider what we have to achieve. Our target tissues are: the arterial system, you know, again w…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 24:29-27:15 So liver is really the poster child for cell and vector-based therapies. Why is liver so promising? 肝臓は細胞ベースの治療やベクター…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 23:25-24:28 So a lot of the same considerations for this genome editing inside the body. 体内でゲノム編集を行う場合も同様に、以下のような課題があ…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 20:52-23:24 So the easiest solution to imagine for this type of situation is that it's sufficient to have a blob of corrected cells that go somew…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 18:25-20:50 Now the final step is to put those cells back and expect them to do something good to not cause mischief but rather to benefit the pa…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 16:58-18:24 So once, but if you're starting with a culture dish in a lab and you're doing this correction process, even if only one percent of th…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 14:28-16:57 So the second major hurdle was to come up with an efficient way to modify the DNA, to correct the DNA. There are many different metho…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 13:11-14:27 So the first major obstacle was to come up with a way of making these stem cells and importantly they're not just any stem cell. They…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 11:20-13:10 So what's involved in the process of correcting a cell outside of the body and then giving it back? Well, first up on the upper left …

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 10:15-11:19 So what is genome editing? So it's a global term that's meant to imply purposeful modification of the DNA within a target cell type o…

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit 7:22-10:14 So tonight we're going to talk about three topics. With little side discussions of some modifications of these methods, but in essence…