{"id":12020,"date":"2021-01-06T15:09:42","date_gmt":"2021-01-06T20:09:42","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=12020"},"modified":"2022-11-14T17:14:57","modified_gmt":"2022-11-14T22:14:57","slug":"kicking-off-the-new-year-with-exciting-research-news","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/bn\/2021\/01\/06\/kicking-off-the-new-year-with-exciting-research-news\/","title":{"rendered":"\u0989\u09a4\u09cd\u09a4\u09c7\u099c\u09a8\u09be\u09aa\u09c2\u09b0\u09cd\u09a3 \u0997\u09ac\u09c7\u09b7\u09a3\u09be \u0996\u09ac\u09b0 \u09b8\u0999\u09cd\u0997\u09c7 \u09a8\u09a4\u09c1\u09a8 \u09ac\u099b\u09b0 \u09ac\u09a8\u09cd\u09a7 \u09b2\u09be\u09a5\u09bf!"},"content":{"rendered":"

[et_pb_section fb_built=”1″ _builder_version=”4.16″ custom_padding=”0px|||||” global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”content base” _builder_version=”4.16″ background_size=”initial” background_position=”top_left” background_repeat=”repeat” global_colors_info=”{}”]<\/p>\n

\"\"<\/p>\n

Happy New Year! We hope everyone had a healthy, restful holiday.<\/p>\n

We are kicking off 2021 off with more exciting research news<\/strong>. In January, the science journal Nature<\/em> published breakthrough results demonstrating that genetic editing in a mouse model of Progeria corrected the mutation that causes Progeria in many cells,<\/strong> improved several key disease symptoms and dramatically increased lifespan<\/strong> in the mice.<\/p>\n

Co-funded by PRF and co-authored by PRF\u2019s Medical Director Dr. Leslie Gordon, the study found that with a single injection of a base editor programmed to correct the disease-causing mutation, mice survived 2.5 times longer than control untreated Progeria mice, to an age corresponding with the start of old age in healthy mice. Importantly, treated mice also retained healthy vascular tissue\u2014a significant finding, as loss of vascular integrity is a predictor of mortality in children with Progeria.<\/p>\n

The study was co-led by world expert in genetic editing, David Liu, PhD<\/a>, of the Broad Institute, MIT, Cambridge, MA, Jonathan Brown, Assistant Professor of Medicine in the Division of Cardiovascular Medicine at Vanderbilt University, and Francis Collins, MD, PhD, Director of the National Institutes of Health.<\/p>\n

[\/et_pb_text][et_pb_testimonial author=”Dr. Francis Collins” job_title=”Director of the National Institutes of Health. ” admin_label=”Dr. Francis Collins” _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]To see this dramatic response in our Progeria mouse model is one of the most exciting therapeutic developments I have been part of in 40 years as a physician-scientist,\u201d said Dr. Collins.
\n[\/et_pb_testimonial][et_pb_testimonial author=”David Lui, PhD” job_title=”Broad Institute, MIT” admin_label=”David Lui, PhD” _builder_version=”4.16″ _module_preset=”default” global_colors_info=”{}”]Five years ago, we were still finishing the development of the very first base editor,\u201d said Dr. Liu. \u201cIf you had told me then that within five years, a single dose of a base editor could address Progeria in an animal at the DNA, RNA, protein, vascular pathology, and lifespan levels, I would have said \u2018there\u2019s no way.\u2019 It’s a real testament to the dedication of the team that made this work possible.
\n[\/et_pb_testimonial][et_pb_text _builder_version=”4.18.1″ _module_preset=”default” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n

Additional preclinical studies are needed to investigate these results, which we hope will one day lead to a clinical trial. We look forward to keeping you informed of this exciting finding as further progress is made!<\/p>\n

Read more about this thrilling news here<\/a>.<\/p>\n

[\/et_pb_text][et_pb_text _builder_version=”4.18.1″ _module_preset=”default” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n

\n

As we enter 2021, we are grateful for partners like YOU, who make this kind of progress possible.<\/p>\n

Last year alone<\/em>, The Progeria Research Foundation:<\/strong><\/span><\/p>\n

\n

    \n
  • Achieved a major milestone with the first-ever FDA-approved treatment for Progeria, lonafarnib;<\/li>\n
  • Brought children with Progeria from around the world to Boston Children’s Hospital for the PRF-funded clinical trial (when travel was allowed due to COVID 19);<\/li>\n
  • Held our 10th International Progeria Scientific Workshop<\/em>, bringing together a record 370 registrants from 30 countries in our first-ever web-based scientific meeting;<\/li>\n
  • Continued in earnest with our key programmatic activities that will lead us to treatments and the future cure!<\/li>\n<\/ul>\n

    [\/et_pb_text][et_pb_text _builder_version=”4.18.1″ _module_preset=”default” hover_enabled=”0″ global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n

    \n

    Koblan LW, Erdos MR, Wilson C, Cabral\u00a0<\/sup>WA, Levy JM, Iong, ZM, Tavarez\u00a0<\/sup>UL, Davison L, Gete YG, Mao X, Newby\u00a0<\/sup>GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY,\u00a0Gordon<\/b>\u00a0LB,\u00a0 Cao K, Collins FS, Brown JD, Liu\u00a0DR.\u00a0<\/span>In Vivo\u00a0<\/i>Adenine Base Editing Rescues Hutchinson-Gilford Progeria Syndrome\u00a0<\/b>Nature. 2021 Jan 6<\/span><\/p>\n

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