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  • Having this new tool is really a breakthrough. It's a game-changer in the ability to study

  • disease and identify new treatments for disease. If you could take any cell in the body and

  • turn it into this immature stem cell-like state, if you could do it for animals you

  • could also do it for humans -- and if you could do it for humans, you could do it for

  • patients. And automatically, you have the ability to study the cellular and the molecular

  • basis of a person's disorder. And that's exactly what's happened over the last 6 or 7 years.

  • Human induced pluripotent stem cells, which is what we call them now, iPS cells, then

  • you can use these cells as a disease-in-a-dish, in order to identify new treatments. And you

  • can determine whether they might have adverse effects in the dish -- even before they go

  • into patients. People have been able to generate these cells from people with schizophrenia,

  • people with autism, people with bipolar disorder. And its really opened up a whole new world

  • of investigation to us. There are other ways in which people have been able to harness

  • an amazing capacity of these cells to self-organize into structures that look like the organs

  • that are in your own body. Recently there have been studies that have used these cells

  • to develop what are called organoids. And that sounds very science fictiony and Frankenstein-like.

  • But what they are is they are structures that the cells make all by themselves -- with a

  • little bit of coaxing from the scientists -- but they look like miniature brains. And

  • they have the organization of the brain. They have the cell types that are very similar

  • to the cell types in the brain, in the right organization. What that does is that allows

  • you to look whether the organization of these structures differs between cells from normal

  • non-disease subjects versus patients. And that's important for diseases like schizophrenia,

  • because the evidence suggests that these are developmental disorders. And to be able to

  • recapitulate -- to show the developmental process all over again -- in a dish is an

  • incredibly powerful tool. And allows you to discover whether there are mechanisms that

  • aren't functioning properly in these developing structures from patients. And it allows us

  • to look, in very fine detail, about what the exact defects are in disorders like autism

  • spectrum disorders, schizophrenia, bipolar disorder. Because all of these disorders fundamentally

  • are disorders of communication between these cells -- not just individually, but in circuits.

  • Because, fundamentally, these disorders are circuit disorders. The question is whether

  • you can actually recapitulate a predictive model of diseases using these simplistic cultures.

  • Where this technology will have the most relevance is not so much a doctor taking a cell or tissue

  • from a patient and turning it into cells -- because those technologies, or those procedures, will

  • take too long. You wouldn't be able to necessarily do a test on an individual patient using their

  • own cells. What you can do is understand the rules of the game. Are there certain patients

  • with say, a certain set of genes, a certain variant of genes, which would allow them to

  • respond in a certain way to particular drugs, while other patients who might have the same

  • disease may not respond in exactly the same way. So once you understand fully the genetics

  • of particular disorders, then you can use these cells to develop a whole set of drugs

  • that are based on whether they have a certain set of gene variants versus another set of

  • gene variants, then we come to a world of precision medicine. It's not personalized

  • medicine, in which a drug is tailored to any individual -- but it's a world in which you

  • can say "because you show these certain set of genes, you're more likely to respond to

  • drug X rather than drug Y." And you can develop diagnostic tests based on that.

Having this new tool is really a breakthrough. It's a game-changer in the ability to study

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iPS細胞とオルガノイド-SFと現実 (iPS Cells & Organoids - Sci-Fi vs Reality)

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    Study English に公開 2021 年 01 月 14 日
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