{"id":2952,"date":"2019-03-27T13:16:28","date_gmt":"2019-03-27T04:16:28","guid":{"rendered":"http:\/\/163.180.4.222\/lab\/?p=2952"},"modified":"2019-03-27T13:16:28","modified_gmt":"2019-03-27T04:16:28","slug":"the-crispr-%ef%bb%bffix-that-could-combat-inherited-blood-disorders","status":"publish","type":"post","link":"https:\/\/biochemistry.khu.ac.kr\/lab\/?p=2952","title":{"rendered":"The CRISPR \ufefffix that could combat inherited blood disorders"},"content":{"rendered":"

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Researchers have finally identified a reliable way to edit the genes of blood stem cells.<\/h5>\n

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\"Scanning<\/p>\n

The elongated red blood cells of people with sickle-cell disease can block small blood vessels, reducing the flow of oxygen to nearby tissues. Credit: Eye of Science\/SPL<\/p>\n

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An enhanced version of the CRISPR\u2013Cas9 genome-editing tool can alter the DNA of bone-marrow stem cells, offering a potential treatment for certain blood diseases.<\/p>\n

Both sickle-cell disease and the illness \u03b2-thalassaemia are associated with misshapen red blood cells, a consequence of mutations in genes encoding the protein haemoglobin. Daniel Bauer at Boston Children\u2019s Hospital in Massachusetts and his colleagues isolated haematopoietic stem cells \u2014 which are found in the bone marrow and develop into blood cells \u2014 from the blood of people with either \u03b2-thalassaemia or sickle-cell disease.<\/p>\n

CRISPR\u2013Cas9 has been used to modify stretches of DNA in the genomes of many types of cell, but it hasn\u2019t worked reliably in haematopoietic stem cells. To get around this, the researchers used an altered version of CRISPR\u2019s DNA-cutting component to switch on a healthy haemoglobin gene already present in the stem cells\u2019 genomes.<\/p>\n

When the researchers infused the edited cells into mice, the human stem cells produced normal red blood cells, suggesting that the authors\u2019 strategy could eventually provide a treatment for haemoglobin disorders.<\/p>\n

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Nature Med.<\/i>\u00a0(2019)<\/a><\/p>\n<\/div>\n

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(\uc6d0\ubb38: \uc5ec\uae30<\/a>\ub97c \ud074\ub9ad\ud558\uc138\uc694~)<\/p>\n

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  Researchers have finally identified a reliable way to edit the genes of blood stem cells.   The elongated red blood cells of people with(more…)<\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"jetpack_post_was_ever_published":false,"_jetpack_newsletter_access":"","_jetpack_dont_email_post_to_subs":false,"_jetpack_newsletter_tier_id":0,"_jetpack_memberships_contains_paywalled_content":false,"_jetpack_memberships_contains_paid_content":false,"footnotes":"","jetpack_publicize_message":"","jetpack_publicize_feature_enabled":true,"jetpack_social_post_already_shared":true,"jetpack_social_options":{"image_generator_settings":{"template":"highway","default_image_id":0,"font":"","enabled":false},"version":2}},"categories":[33,34,29,30],"tags":[],"class_list":["post-2952","post","type-post","status-publish","format-standard","hentry","category-do-biology","category-lets-do-chemistry","category-lets-do-science","category-recent-science-news"],"aioseo_notices":[],"jetpack_publicize_connections":[],"jetpack_featured_media_url":"","jetpack-related-posts":[{"id":4084,"url":"https:\/\/biochemistry.khu.ac.kr\/lab\/?p=4084","url_meta":{"origin":2952,"position":0},"title":"Scientists use gene-edited stem cells to treat HIV \u2014 with mixed success","author":"biochemistry","date":"September 17, 2019","format":false,"excerpt":"\u00a0 \u00a0 Modified cells survived 19 months after transplant into an HIV-positive man in China, but the dose was not enough to reduce his viral load. \u00a0 \u00a0 HIV destroys the body's defences by attacking immune cells.\u00a0Credit: Steve Gschmeissner\/Science Photo Library \u00a0 \u00a0 For the first time, researchers have used\u2026","rel":"","context":"In "Let's Do Biology!"","block_context":{"text":"Let's Do Biology!","link":"https:\/\/biochemistry.khu.ac.kr\/lab\/?cat=33"},"img":{"alt_text":"","src":"","width":0,"height":0},"classes":[]},{"id":3524,"url":"https:\/\/biochemistry.khu.ac.kr\/lab\/?p=3524","url_meta":{"origin":2952,"position":1},"title":"Stem-cell and genetic therapies make a healthy marriage","author":"biochemistry","date":"May 16, 2019","format":false,"excerpt":"\u00a0 \u00a0 This scientific partnership could fight everything from blood diseases to HIV. \u00a0 A culture from human tissue showing neurons derived from stem cells.Credit: Daniel Schroen\/Cell Applications Inc\/SPL \u00a0 \u00a0 Aside from a 20-second exposure to the outside world at birth, David Vetter spent his entire life cocooned in\u2026","rel":"","context":"In "Essays on Science"","block_context":{"text":"Essays on Science","link":"https:\/\/biochemistry.khu.ac.kr\/lab\/?cat=32"},"img":{"alt_text":"","src":"","width":0,"height":0},"classes":[]},{"id":3988,"url":"https:\/\/biochemistry.khu.ac.kr\/lab\/?p=3988","url_meta":{"origin":2952,"position":2},"title":"The long shadow of a CRISPR scandal","author":"biochemistry","date":"August 3, 2019","format":false,"excerpt":"\u00a0 He Jiankui's controversial gene-editing experiment brought intense scrutiny to CRISPR scientists in China, and they're outraged. \u00a0 As He Jiankui strode to the podium at last year's summit on human genome editing in Hong Kong, China, more than 1 million people watched online. 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Humbert\u00a0et al.\u00a0used CRISPR-based gene editing to modify hematopoietic\u2026","rel":"","context":"In "Let's Do Biology!"","block_context":{"text":"Let's Do Biology!","link":"https:\/\/biochemistry.khu.ac.kr\/lab\/?cat=33"},"img":{"alt_text":"","src":"","width":0,"height":0},"classes":[]},{"id":4845,"url":"https:\/\/biochemistry.khu.ac.kr\/lab\/?p=4845","url_meta":{"origin":2952,"position":4},"title":"CRISPR tool modifies genes precisely by copying RNA into the genome & CRISPR: the movie","author":"biochemistry","date":"November 15, 2019","format":false,"excerpt":"\u00a0 \u00a0 The ultimate goal of genome editing is to be able to make any specific change to the blueprint of life. A \u2018search-and-replace\u2019 method for genome editing takes us a giant leap closer to this ambitious goal. \u00a0 \u00a0 Variation in the DNA sequences that constitute the blueprint of\u2026","rel":"","context":"In "Let's Do Biology!"","block_context":{"text":"Let's Do Biology!","link":"https:\/\/biochemistry.khu.ac.kr\/lab\/?cat=33"},"img":{"alt_text":"","src":"","width":0,"height":0},"classes":[]},{"id":2247,"url":"https:\/\/biochemistry.khu.ac.kr\/lab\/?p=2247","url_meta":{"origin":2952,"position":5},"title":"Precision genome engineering","author":"biochemistry","date":"December 3, 2018","format":false,"excerpt":"\u00a0 \u00a0 Genome editing through CRISPR-Cas systems has the potential to correct genetic mutations that occur in diseased cells, such as cancer cells. However, the ability to selectively activate CRISPR-Cas systems in diseased cells is important to ensure that gene editing only occurs where it is wanted. 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