海外マルファン情報

米国マルファン症候群患者団体The Marfan Foundationからの情報を中心に、マルファン症候群や関連疾患についての海外情報を翻訳して発信します。

Entries from 2020-11-01 to 1 month

マルファン関連疾患における整形外科的所見 ~側弯症~

Keeping Your Spine, Limbs, and Feet Healthy in Marfan and Related Conditions 3:51-4:42 On the spine, I'd like to cover scoliosis, kyphosis and slipping, spondylolisthesis. I think that these are all things you've probably heard of, but I'l…

マルファン関連疾患における整形外科的所見 ~はじめに~

Keeping Your Spine, Limbs, and Feet Healthy in Marfan and Related Conditions 3:04-3:50 The topic of my talk is to talk about their healthy limb, feet and spine and cooperating with Adam, we, the thing I'd like to focus on would be the spin…

血管以外の臓器系へのロサルタンの影響について

CRISPR, Stem Cells, Gene Modifiers – The Future of Marfan Research (August 27 2020) E3 Summit Q:Is there any more research being planned on the effects of losartan on other organ systems? 血管以外の臓器系に対してロサルタンの影響を調べる研…

ゲノム編集と将来のマルファン症候群治療 ~最後に~

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…

ゲノム編集と将来のマルファン症候群治療 ~治療薬の新たな選択肢~

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,…

ゲノム編集と将来のマルファン症候群治療 ~ERK抑制遺伝子~

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 …

ゲノム編集と将来のマルファン症候群治療 ~マウスを用いた仮説の検証~

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…

ゲノム編集と将来のマルファン症候群治療 ~MAP3K4遺伝子について~

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…

ゲノム編集と将来のマルファン症候群治療 ~新たな仮説~

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…

ゲノム編集と将来のマルファン症候群治療 ~ERKの不活性化にむけて~

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 …

マルファン関連疾患向け運動ガイドライン

運動はマルファン症候群の方にも効果があります マルファン症候群では運動に修正が必要なのはなぜか? 運動の種類 METs(代謝当量)とは? マルファン症候群の方が運動する際に知っておくべきこと 医師が気にかけることは? マルファン症候群の方が安全に運…

ゲノム編集と将来のマルファン症候群治療 ~iPS細胞を用いたマルファン症候群の研究~

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…

ゲノム編集と将来のマルファン症候群治療 ~幹細胞を用いた心筋治療の可能性~

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…

ゲノム編集と将来のマルファン症候群治療 ~大動脈基部でのFBN2遺伝子活性化に向けた研究~

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-…

ゲノム編集と将来のマルファン症候群治療 ~CRISPRaによるFBN2の活性化~

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…

ゲノム編集と将来のマルファン症候群治療 ~胚性遺伝子のオン・オフ~

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…

ゲノム編集と将来のマルファン症候群治療 ~マルファン症候群におけるゲノム編集の課題~

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…

ゲノム編集と将来のマルファン症候群治療 ~ゲノム編集に適した組織~

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…

ゲノム編集と将来のマルファン症候群治療 ~体内でゲノム編集を行う場合の課題~

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…

ゲノム編集と将来のマルファン症候群治療 ~修正された細胞による様々な可能性~

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…

ゲノム編集と将来のマルファン症候群治療 ~修正した細胞を体内に戻す際の課題~

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…

ゲノム編集と将来のマルファン症候群治療 ~細胞修正後のプロセス~

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…