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患者から採取した初代細胞の疾患原因である遺伝子変異をゲノム編集により修正して、変異の修正による治療効果をin vitroで調べることができる。しかし初代細胞には分裂寿命があるため、治療に必要な細胞数の確保に限界があるなど、将来、治療につなげること難しい。一方、幹細胞は、自分と同じ細胞を作る(自己複製)能力と、別の種類の細胞に分化する能力を持ち、際限なく増殖できる細胞である。この幹細胞の特徴に着目したヒトiPSCsとゲノム編集を組み合わせた研究が進んでいる。患者の採取しやすい場所(たとえば血液や皮膚)から細胞を取り出し、まずは初期化する。患者iPSCsは特殊な培地で無限の増殖を行うことができ、培養条件を変えることで様々な組織特異的な細胞への分化が可能である。患者iPSCsは疾患原因である遺伝子変異を持っているので、このiPSCsをある特定の組織に分化させれば、疾患の特定の組織における病態形成の過程を調べることができる。

It is possible to use genome editing to correct genetic mutations that cause disease in primary cells collected from a patient, and then to investigate the therapeutic effect of that mutation correction in vitro. However, there are difficulties connecting this to future treatment, for example because of limits to securing the number of cells necessary for treatment since the primary cells have replicative senescence. On the other hand, stem cells have the ability to create identical cells (self-replication) and the ability to differentiate into other types of cells, and so can proliferate without limit. Research combining genome editing and human iPSCs while focusing on these characteristics of stem cells is making progress. Cells are extracted from a convenient sampling location (for example, the blood or skin) of a patient, and then start by being initialized. Patient iPSCs can proliferate indefinitely on specialized media, and changing the culturing conditions can cause differentiation into a variety of tissue-specific cells. Patient iPSCs have the same genetic mutations that cause their diseases, and so by differentiating these iPSCs into specific tissues, it is possible to investigate the process by which pathology develops in particular patient tissues.

It is possible to use genome editing in primary cells sampled from a patient to correct genetic mutations that cause disease in primary cells collected from a patient, and then to investigate the therapeutic effect of that mutation correctioncorrecting those mutations in vitro. However, there are difficulties connectingin using this to develop future treatmentinterventions, for example because of limitsthere is a limit to securing the number of cells necessary for treatment sincedue to the primary cells have replicative senescence. of primary cells. On the other hand, stem cells have the ability to createproduce identical cells (self-replication) and the ability to differentiate into other cell types of cells, and so can proliferate without limit. ResearchThere has been progress in research combining genome editing and human iPSCs while focusing on these characteristics of stem cells is making progress.. Cells are extractedtaken from a convenient sampling location (for example, the blood or skin) of a patient, and then start by beingfirst are initialized. Patient iPSCs can proliferate indefinitely on specialized media, and changingwith changes to the culturing conditions can cause differentiationdifferentiate into a variety of tissue-specific cells. Patient iPSCs from patients have the same genetic mutations that cause their diseases, and so by differentiatingcausing these iPSCs to differentiate into specific tissues, it is possible to investigate the process by which pathology develops in particular patient tissues.

It is possible to use genome editing in primary cells sampled from a patients to correct genetic mutations that causeresponsible for diseases, and then to investigate the therapeutic effects of the correctiong those mutations in vitro. However, this method could make it there are difficulties in to using this to develop future interventions;, for example, the replicative senescence of primary cells limits because there is a limit to securing the number of cells that can be obtained necessary for treatment due to the replicative senescence of primary cells. However,On the other hand, stem cells have the ability to have the ability to produce identical cells (self-replication) and the ability to differentiate into other cell types; they can therefore, and so can proliferate without limit. Studies onThere has been progress in research combining genome editing and human iPSCs, while focusing on these unique stem cell characteristics, have been conducted of stem cells. Patient Ccells are  collectedtaken from a convenient sampling location (for example, the blood or skin) of a patient, and are first are initialized. Patient iPSCs can proliferate indefinitely on specialized media, and with changes to the culturing conditions, can differentiate into differenta variety of tissue-specific cells. Patient iPSCs from patients carryhave the same genetic mutations responsible forthat cause theirir diseases;, and so therefore, by differentiatingcausing these iPSCs to differentiate into specific tissues, it is possible to investigate the pathogenesis process by which pathology develops in particular patient tissues.

It is possible to use gGenome editing in primary cells sampled from a patients can be used to correct genetic mutations that causeresponsible for diseases, and then to investigate the therapeutic effects of the correctiong those mutations in vitro. However, there are difficulties in using this to the development of  future interventions using this technique is difficult because,, for example, the replicative senescence of primary cells limits because there is a limit to securing the number of cells that can be obtained necessary for treatment due to the replicative senescence of primary cells. However,On the other hand, stem cells have the ability to have the ability to produce identical cells (self-replication) and the ability to differentiate into other cell types; they can, therefore,, and so can proliferate without limit. ResearchThere has been progress in research combining genome editing and human iPSCs, while focusing on these unique stem cell characteristics, has made considerable progress of stem cells. Patient Ccells are  collectedtaken from a convenient sampling sourcelocation (for example, the blood or skin) of a patient, and are first are initialized. Patient iPSCs can proliferate indefinitely on specialized media, and, with changes to the culturing conditions, can differentiate into differenta variety of tissue-specific cells. Patient iPSCs from patients carryhave the same genetic mutations responsible forthat cause their diseases;, and so therefore, by differentiatingcausing these iPSCs to differentiate into specific tissues, it is possible to investigate the  process by which pathology develops in underlying pathogenesis in these particular patient tissues.

患者から採取した初代細胞の疾患原因である遺伝子変異をゲノム編集により修正して、変異の修正による治療効果をin vitroで調べることができる。しかし初代細胞には分裂寿命があるため、治療に必要な細胞数の確保に限界があるなど、将来、治療につなげること難しい。一方、幹細胞は、自分と同じ細胞を作る(自己複製)能力と、別の種類の細胞に分化する能力を持ち、際限なく増殖できる細胞である。この幹細胞の特徴に着目したヒトiPSCsとゲノム編集を組み合わせた研究が進んでいる。患者の採取しやすい場所(たとえば血液や皮膚)から細胞を取り出し、まずは初期化する。患者iPSCsは特殊な培地で無限の増殖を行うことができ、培養条件を変えることで様々な組織特異的な細胞への分化が可能である。患者iPSCsは疾患原因である遺伝子変異を持っているので、このiPSCsをある特定の組織に分化させれば、疾患の特定の組織における病態形成の過程を調べることができる。

It is possible to use genome editing to correct genetic mutations that cause disease in primary cells collected from a patient, and then to investigate the therapeutic effect of that mutation correction in vitro. However, there are difficulties connecting this to future treatment, for example because of limits to securing the number of cells necessary for treatment since the primary cells have replicative senescence. On the other hand, stem cells have the ability to create identical cells (self-replication) and the ability to differentiate into other types of cells, and so can proliferate without limit. Research combining genome editing and human iPSCs while focusing on these characteristics of stem cells is making progress. Cells are extracted from a convenient sampling location (for example, the blood or skin) of a patient, and then start by being initialized. Patient iPSCs can proliferate indefinitely on specialized media, and changing the culturing conditions can cause differentiation into a variety of tissue-specific cells. Patient iPSCs have the same genetic mutations that cause their diseases, and so by differentiating these iPSCs into specific tissues, it is possible to investigate the process by which pathology develops in particular patient tissues.

It is possible to use genome editing in primary cells sampled from a patient to correct genetic mutations that cause disease in primary cells collected from a patient, and then to investigate the therapeutic effect of that mutation correctioncorrecting those mutations in vitro. However, there are difficulties connectingin using this to develop future treatmentinterventions, for example because of limitsthere is a limit to securing the number of cells necessary for treatment sincedue to the primary cells have replicative senescence. of primary cells. On the other hand, stem cells have the ability to createproduce identical cells (self-replication) and the ability to differentiate into other cell types of cells, and so can proliferate without limit. ResearchThere has been progress in research combining genome editing and human iPSCs while focusing on these characteristics of stem cells is making progress.. Cells are extractedtaken from a convenient sampling location (for example, the blood or skin) of a patient, and then start by beingfirst are initialized. Patient iPSCs can proliferate indefinitely on specialized media, and changingwith changes to the culturing conditions can cause differentiationdifferentiate into a variety of tissue-specific cells. Patient iPSCs from patients have the same genetic mutations that cause their diseases, and so by differentiatingcausing these iPSCs to differentiate into specific tissues, it is possible to investigate the process by which pathology develops in particular patient tissues.

It is possible to use genome editing in primary cells sampled from a patients to correct genetic mutations that causeresponsible for diseases, and then to investigate the therapeutic effects of the correctiong those mutations in vitro. However, this method could make it there are difficulties in to using this to develop future interventions;, for example, because there is a limit to securing the number of cells that can be obtained necessary for treatment is limited, owingdue to the replicative senescence of primary cells. However,On the other hand, stem cells canhave the ability to produce identical cells (self-replication) and the ability to differentiate into other cell types and can therefore, and so can proliferate without limit. There has been progress in research combining genome editing and human iPSCs, while focusing on these unique stem cell characteristics of stem cells. Cells are  collectedtaken from a convenient sampling location (for example, the blood or skin) of a patient, and are first are initialized. Patient iPSCs can proliferate indefinitely on specialized media, and with changes to the culturing conditions, can differentiate into a variety of tissue-specific cells. Patient iPSCs from patients carryhave the same genetic mutations responsible forthat cause theirir diseases, and so, by differentiatingcausing these iPSCs to differentiate into specific tissues, it is possible to investigate the pathogenesis process by which pathology develops in particular patient tissues.

GIt is possible to use genome editing in primary cells sampled from a patients can be used to correct genetic mutations that causeresponsible for diseases, and then to investigate the therapeutic effects of the correctiong those mutations in vitro. However, there are difficulties in using this to the development of future interventions using this technique is difficult because, , for example, because there is a limit to securing the number of cells that can be obtained necessary for treatment is limited owing to  due to the replicative senescence of primary cells. However, On the other hand, stem cells canhave the ability to produce identical cells (self-replication) and the ability to differentiate into other cell types and can, therefore,, and so can proliferate indefinitelywithout limit. There has been progress in research that combines ing genome editing and human iPSCs, while focusing on these unique stem cell characteristics of stem cells. Cells are  collectedtaken from a convenient sampling sourcelocation (for example, the blood or skin) of a patient, and are first are initialized. Patient iPSCs can proliferate indefinitely on specialized media, and, with changes to the culturing conditions, can differentiate into a variety of tissue-specific cells. Patient iPSCs from patients carryhave the same genetic mutations responsible forthat cause their diseases; thus,, and so  by differentiatingcausing these iPSCs to differentiate into specific tissues, it is possible to investigate the  process by which pathology develops in underlying pathogenesis in these particular patient tissues.

患者から採取した初代細胞の疾患原因である遺伝子変異をゲノム編集により修正して、変異の修正による治療効果をin vitroで調べることができる。しかし初代細胞には分裂寿命があるため、治療に必要な細胞数の確保に限界があるなど、将来、治療につなげること難しい。一方、幹細胞は、自分と同じ細胞を作る(自己複製)能力と、別の種類の細胞に分化する能力を持ち、際限なく増殖できる細胞である。この幹細胞の特徴に着目したヒトiPSCsとゲノム編集を組み合わせた研究が進んでいる。患者の採取しやすい場所(たとえば血液や皮膚)から細胞を取り出し、まずは初期化する。患者iPSCsは特殊な培地で無限の増殖を行うことができ、培養条件を変えることで様々な組織特異的な細胞への分化が可能である。患者iPSCsは疾患原因である遺伝子変異を持っているので、このiPSCsをある特定の組織に分化させれば、疾患の特定の組織における病態形成の過程を調べることができる。

It is possible to use genome editing to correct genetic mutations that cause disease in primary cells collected from a patient, and then to investigate the therapeutic effect of that mutation correction in vitro. However, there are difficulties connecting this to future treatment, for example because of limits to securing the number of cells necessary for treatment since the primary cells have replicative senescence. On the other hand, stem cells have the ability to create identical cells (self-replication) and the ability to differentiate into other types of cells, and so can proliferate without limit. Research combining genome editing and human iPSCs while focusing on these characteristics of stem cells is making progress. Cells are extracted from a convenient sampling location (for example, the blood or skin) of a patient, and then start by being initialized. Patient iPSCs can proliferate indefinitely on specialized media, and changing the culturing conditions can cause differentiation into a variety of tissue-specific cells. Patient iPSCs have the same genetic mutations that cause their diseases, and so by differentiating these iPSCs into specific tissues, it is possible to investigate the process by which pathology develops in particular patient tissues.

It is possible to use genome editing in primary cells sampled from a patient to correct genetic mutations that cause disease in primary cells collected from a patient, and then to investigate the therapeutic effect of that mutation correctioncorrecting those mutations in vitro. However, there are difficulties connectingin using this to develop future treatmentinterventions, for example because of limitsthere is a limit to securing the number of cells necessary for treatment sincedue to the primary cells have replicative senescence. of primary cells. On the other hand, stem cells have the ability to createproduce identical cells (self-replication) and the ability to differentiate into other cell types of cells, and so can proliferate without limit. ResearchThere has been progress in research combining genome editing and human iPSCs while focusing on these characteristics of stem cells is making progress.. Cells are extractedtaken from a convenient sampling location (for example, the blood or skin) of a patient, and then start by beingfirst are initialized. Patient iPSCs can proliferate indefinitely on specialized media, and changingwith changes to the culturing conditions can cause differentiationdifferentiate into a variety of tissue-specific cells. Patient iPSCs from patients have the same genetic mutations that cause their diseases, and so by differentiatingcausing these iPSCs to differentiate into specific tissues, it is possible to investigate the process by which pathology develops in particular patient tissues.

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