{"id":281,"date":"2024-04-14T08:35:22","date_gmt":"2024-04-14T08:35:22","guid":{"rendered":"https:\/\/presidentreport.biu.ac.il\/?p=281"},"modified":"2024-05-23T16:01:39","modified_gmt":"2024-05-23T16:01:39","slug":"one-small-cut-one-giant-leap-for-medicine","status":"publish","type":"post","link":"https:\/\/presidentreport.biu.ac.il\/index.php\/2024\/04\/14\/one-small-cut-one-giant-leap-for-medicine\/","title":{"rendered":"One Small Cut, One Giant Leap for Medicine"},"content":{"rendered":"\n
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Prof. Ayal Hendel in his Genome Editing and Gene Therapy Laboratory.<\/p> <\/div>\n<\/div>\n <\/div>\n <\/div>\n <\/div>\n

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The Mina and Everard Goodman Faculty of Life Sciences
The Institute of Nanotechnology and Advanced Materials<\/h5> <\/div>\n<\/div>\n\n
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One Small Cut,
One Giant Leap for Medicine<\/h3> <\/div>\n<\/div>\n\n
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A pioneer in the field of CRISPR\u2014a revolutionary technology that allows scientists to precisely cut and modify specific bits of DNA inside the cell\u2014Prof. Ayal Hendel developed a treatment for a rare genetic disorder, transforming what a death sentence into hope for a normal life.<\/h6> <\/div>\n<\/div>\n <\/div>\n <\/div>\n <\/div>\n
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Patient, Heal Thyself<\/h3>
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When David Vetter was born in 1971 with Severe Combined Immunodeficiency (SCID), a rare hereditary disorder that prevents the development of an immune system, doctors hoped his older sister could provide a bone-marrow transplant\u2014then, as now, the only effective treatment for the disease. When, however, she turned out not to be a match, Vetter was forced to live inside an isolator until his death at age 12, earning him and the disease the iconic \u201cBubble Boy\u201d nickname. Yet for most SCID patients who lack a matching bone-marrow donor\u2014or for whom a transplant is unsuccessful, because the donated bone marrow attacks its new host\u2014the average life span is much shorter: typically, under one year.<\/p>\n

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At least, it was until now. Genome-editing expert Prof. Ayal Hendel of the Mina and Everard Goodman Faculty of Life Sciences believes his new treatment for SCID will enable patients to literally heal themselves by using their own, \u201ccorrected\u201d stem cells to generate a healthy immune system. Together with physicians at Sheba Hospital, Hendel has successfully edited a SCID patient\u2019s blood stem cells using CRISPR, then returned them to his body; as a result, the patient reproduced new, \u201cnormal\u201d T cells that fight germs and protect against disease. Now in the pre-clinical stage, Hendel\u2019s breakthrough treatment is slated for human clinical trials in as little as 2-3 years. And if it\u2019s successful\u2014as was the case with the technology\u2019s recent treatment of sickle-cell anemia\u2014the phrase \u201cBubble Boy\u201d will one day soon become only the stuff of movies, or at least of recent memory.<\/p> <\/div>\n<\/div>\n <\/div>\n <\/div>\n <\/div>\n

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\u201cMore than anything, I hope this treatment for a rare genetic disease demonstrates that scientific innovation can go hand-in-hand with timeless Jewish values, including the sanctity of life above all else, and the idea that by saving a single person, we\u2019re actually saving a whole world,\u201d says Hendel. \u201cWith support from Jewish partners everywhere, Bar-Ilan can become an international leader in the fight against deadly genetic disease.\u201d<\/p>\n <\/div>\n<\/div>\n <\/div>\n

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An End to Sickle Cells: Editing Out a Deadly Disease<\/h3>
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When CRISPR first arrived on the scene, Prof. Ayal Hendel\u2014then a researcher at Stanford University School of Medicine\u2014was part of a team that developed a means of protecting the structural integrity of the CRISPR\u2019s RNA, the component that \u201cguides\u201d the technology toward the site on the\u00a0gene that needs to be cut. The resulting patent opened the door for CRISPR\u2019s use in treating monogenic diseases such as sickle-cell anemia, which affect nearly 20 million people worldwide. And indeed, Casgevy, the world\u2019s first CRISPR-based medicine, was approved as a therapy for sickle-cell anemia by the FDA last year.<\/p> <\/div>\n<\/div>\n <\/div>\n <\/div>\n <\/div>\n

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<\/a>\"image_print\"Print<\/span><\/a><\/div>","protected":false},"excerpt":{"rendered":"

Prof. Ayal Hendel in his Genome Editing and Gene Therapy Laboratory. The Mina and Everard Goodman Faculty of Life SciencesThe Institute of Nanotechnology and Advanced Materials One Small Cut,One Giant Leap for Medicine A pioneer in the field of CRISPR\u2014a revolutionary technology that allows scientists to precisely cut and modify specific bits of DNA inside […]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[5],"tags":[],"class_list":["post-281","post","type-post","status-publish","format-standard","hentry","category-research-innovation","has-post-title","has-post-date","has-post-category","has-post-tag","has-post-comment","has-post-author",""],"builder_content":"

Prof. Ayal Hendel in his Genome Editing and Gene Therapy Laboratory.<\/p>\n

The Mina and Everard Goodman Faculty of Life Sciences
The Institute of Nanotechnology and Advanced Materials<\/h5>\n

One Small Cut,
One Giant Leap for Medicine<\/h3>\n

A pioneer in the field of CRISPR\u2014a revolutionary technology that allows scientists to precisely cut and modify specific bits of DNA inside the cell\u2014Prof. Ayal Hendel developed a treatment for a rare genetic disorder, transforming what a death sentence into hope for a normal life.<\/h6>\n

Patient, Heal Thyself<\/h3>

When David Vetter was born in 1971 with Severe Combined Immunodeficiency (SCID), a rare hereditary disorder that prevents the development of an immune system, doctors hoped his older sister could provide a bone-marrow transplant\u2014then, as now, the only effective treatment for the disease. When, however, she turned out not to be a match, Vetter was forced to live inside an isolator until his death at age 12, earning him and the disease the iconic \u201cBubble Boy\u201d nickname. Yet for most SCID patients who lack a matching bone-marrow donor\u2014or for whom a transplant is unsuccessful, because the donated bone marrow attacks its new host\u2014the average life span is much shorter: typically, under one year.<\/p>

\u00a0<\/p>

At least, it was until now. Genome-editing expert Prof. Ayal Hendel of the Mina and Everard Goodman Faculty of Life Sciences believes his new treatment for SCID will enable patients to literally heal themselves by using their own, \u201ccorrected\u201d stem cells to generate a healthy immune system. Together with physicians at Sheba Hospital, Hendel has successfully edited a SCID patient\u2019s blood stem cells using CRISPR, then returned them to his body; as a result, the patient reproduced new, \u201cnormal\u201d T cells that fight germs and protect against disease. Now in the pre-clinical stage, Hendel\u2019s breakthrough treatment is slated for human clinical trials in as little as 2-3 years. And if it\u2019s successful\u2014as was the case with the technology\u2019s recent treatment of sickle-cell anemia\u2014the phrase \u201cBubble Boy\u201d will one day soon become only the stuff of movies, or at least of recent memory.<\/p>\n

\u201cMore than anything, I hope this treatment for a rare genetic disease demonstrates that scientific innovation can go hand-in-hand with timeless Jewish values, including the sanctity of life above all else, and the idea that by saving a single person, we\u2019re actually saving a whole world,\u201d says Hendel. \u201cWith support from Jewish partners everywhere, Bar-Ilan can become an international leader in the fight against deadly genetic disease.\u201d<\/p>\n\"Ayal\n\"Ayal\n\"One\n

An End to Sickle Cells: Editing Out a Deadly Disease<\/h3>

When CRISPR first arrived on the scene, Prof. Ayal Hendel\u2014then a researcher at Stanford University School of Medicine\u2014was part of a team that developed a means of protecting the structural integrity of the CRISPR\u2019s RNA, the component that \u201cguides\u201d the technology toward the site on the\u00a0gene that needs to be cut. The resulting patent opened the door for CRISPR\u2019s use in treating monogenic diseases such as sickle-cell anemia, which affect nearly 20 million people worldwide. And indeed, Casgevy, the world\u2019s first CRISPR-based medicine, was approved as a therapy for sickle-cell anemia by the FDA last year.<\/p>","_links":{"self":[{"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/posts\/281","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/comments?post=281"}],"version-history":[{"count":52,"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/posts\/281\/revisions"}],"predecessor-version":[{"id":2949,"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/posts\/281\/revisions\/2949"}],"wp:attachment":[{"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/media?parent=281"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/categories?post=281"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/presidentreport.biu.ac.il\/index.php\/wp-json\/wp\/v2\/tags?post=281"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}