{"id":20799,"date":"2025-08-08T10:11:20","date_gmt":"2025-08-08T14:11:20","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=20799"},"modified":"2026-01-13T13:38:13","modified_gmt":"2026-01-13T18:38:13","slug":"gene-editing","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/uk\/2025\/08\/08\/gene-editing\/","title":{"rendered":"\u041d\u044c\u044e-\u0419\u043e\u0440\u043a\u0435\u0440 \u043f\u0440\u0435\u0434\u0441\u0442\u0430\u0432\u043b\u044f\u0454 \u0440\u0435\u0434\u0430\u0433\u0443\u0432\u0430\u043d\u043d\u044f \u0433\u0435\u043d\u0456\u0432 \u043f\u0440\u043e\u0433\u0435\u0440\u0456\u0457: PRF \u043d\u0430 \u0448\u043b\u044f\u0445\u0443 \u0434\u043e \u0412\u0418\u041a\u041b\u0418\u041a\u0410\u041d\u041d\u042f \u041f\u0420\u041e\u0413\u0415\u0420\u0406\u0407!"},"content":{"rendered":"

[et_pb_section fb_built=”1″ _builder_version=”4.27.4″ global_colors_info=”{}” theme_builder_area=”post_content”][et_pb_row _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”-74px|auto||auto|false|false” global_colors_info=”{}” theme_builder_area=”post_content”][et_pb_column type=”4_4″ _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}” theme_builder_area=”post_content”][et_pb_text admin_label=”Path & Alptug images” _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

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[\/et_pb_text][et_pb_text _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

As described in the August 11, 2025, front-page New Yorker article**, entitled How an Ultra-Rare Disease Accelerates Aging,\u00a0<\/em>\u00a0The Progeria Research Foundation is thrilled to announce the pioneering\u00a0Progeria\u00a0Gene Editing Program.<\/strong>\u00a0<\/p>\n

**Click here<\/strong><\/a> to read the full article.<\/p>\n

[\/et_pb_text][et_pb_testimonial quote_icon_color=”#FFFFFF” quote_icon_background_color=”#8FD2ED” admin_label=”KayleeQuote” _builder_version=”4.27.4″ _module_preset=”default” min_height=”83px” custom_margin=”0px||||false|false” custom_padding=”1px||12px||false|false” hover_enabled=”0″ border_radii=”on|35px|35px|35px|35px” border_width_all=”2px” border_color_all=”#00B2E2″ global_colors_info=”{}” theme_builder_area=”post_content” sticky_enabled=”0″]<\/p>\n

 <\/p>\n

“I\u2019ve been involved with PRF most of my life\u201d, says 22-year-old Kaylee from Ohio, who is prominently featured in the New Yorker article. \u201cI\u2019m really excited about the gene therapy they\u2019re working on!\u201d<\/strong><\/p>\n

[\/et_pb_testimonial][et_pb_text admin_label=”Kaylee image and Base editing text” _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

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Base editing therapy<\/strong>\u00a0uses CRISPR technology to target and correct a specific DNA base (or “letter”) in the genetic code. Because Progeria is caused by a change to a single letter in the DNA, it is perfectly suited for this type of gene therapy.<\/strong><\/p>\n

[\/et_pb_text][et_pb_text admin_label=”Progeria Gene Dream Team” _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”||0px||false|false” global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

The Progeria Gene Team <\/strong><\/span><\/h4>\n

PRF has been collaborating with an extraordinary team of world-renowned scientists and innovators. The team\u2019s tireless work and dedication is bringing this transformative therapy to reality:<\/p>\n

\"\"Top Left to Bottom Right:
Francis Collins, MD, PhD<\/strong>\u00a0\u2013 Longtime Progeria research partner and former Director of the National Institutes of Health (NIH)
Michael Erdos, PhD<\/strong>\u00a0\u2013 Associate Investigator, Molecular Genetics Section, National Human Genome Research Institute (NHGRI)
Leslie Gordon, MD, PhD<\/strong>\u00a0\u2013 Co-founder & Medical Director of PRF, and the world\u2019s foremost expert on Progeria
David Liu, PhD<\/strong>\u00a0\u2013 MIT\u2019s Broad Institute and Harvard University scientist, founder of\u00a0Base Editing<\/strong>\u00a0<\/strong>Gene Therapy Technology
*Sammy Basso<\/strong><\/a>, MS, Co-Founder of The Associazione Italiana Progeria Sammy Basso and PRF Ambassador, was an integral part of this team until his passing in October 2024.<\/p>\n

 <\/p>\n

[\/et_pb_text][et_pb_text admin_label=”Mouse Model” _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

From Mouse Model to Clinical Trials<\/strong><\/span><\/h4>\n

The\u00a0Progeria Gene Team\u00a0and their lab members first demonstrated that gene editing could dramatically increase the lifespan<\/strong> of the Progeria mouse model \u2014 by an astounding\u00a0140%<\/strong>\u00a0\u2014 a discovery that was published in one of the world\u2019s top scientific journals,\u00a0Nature<\/em><\/strong><\/a>. Building on this remarkable achievement, the team has refined the drug and is now focused on steps needed to start a clinical trial: drug manufacturing, engaging with the US FDA, and developing clinical trial protocols.<\/p>\n

The journey ahead will be multi-stage, technically complicated, and expensive. PRF is determined to meet every challenge to bring gene therapy to children and young adults with Progeria.<\/strong><\/p>\n

[\/et_pb_text][et_pb_text admin_label=”Community-Driven Mission” _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

A Community-Driven Mission<\/strong><\/span><\/h4>\n

The Progeria Community has been with us every step of the way.<\/strong> PRF\u2019s leadership team regularly hosts online meetings with Progeria families throughout the world, so they are our partners on this journey toward the cure.<\/p>\n

Moreover, the implications of this work may extend far beyond Progeria. In addition to curing Progeria, we are forging a path that other rare diseases can follow. <\/strong>Thus, this could be a groundbreaking leap forward not only for Progeria, but for other conditions that have long been overlooked.<\/i><\/p>\n

[\/et_pb_text][et_pb_testimonial quote_icon_color=”#FFFFFF” quote_icon_background_color=”#8FD2ED” admin_label=”Liu Quote” _builder_version=”4.27.4″ _module_preset=”default” min_height=”83px” custom_margin=”0px||||false|false” custom_padding=”1px||12px||false|false” border_radii=”on|35px|35px|35px|35px” border_width_all=”2px” border_color_all=”#00B2E2″ global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

 <\/p>\n

\u201cThe hope of our team is to use base editing to fix the root cause of progeria, providing a pathway to a potential cure for children with progeria, as well as advancing treatments for millions of other children with fatal genetic diseases.\u201d<\/strong><\/p>\n

[\/et_pb_testimonial][et_pb_text admin_label=”Liu & Sammy B” _builder_version=”4.27.4″ _module_preset=”default” global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

[\/et_pb_text][et_pb_text admin_label=”Your Support is Vital” _builder_version=”4.27.4″ _module_preset=”default” custom_margin=”8px|||||” global_colors_info=”{}” theme_builder_area=”post_content”]<\/p>\n

Your Support is Vital <\/strong><\/span><\/h4>\n

Gene therapy is the most promising and the most expensive type of drug to manufacture. The unwavering commitment of PRF\u2019s passionate supporters like you will make Progeria gene editing a reality.<\/strong><\/p>\n

 <\/p>\n

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Together, we are on the verge of something extraordinary. <\/strong><\/h4>\n

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<\/strong><\/span><\/h4>\n

Together, we WILL<\/em> find the cure!<\/strong><\/span><\/h4>\n

[\/et_pb_text][et_pb_button button_url=”https:\/\/www.progeriaresearch.org\/2025\/05\/27\/rdd-2025-cimus\/?form=donate” button_text=”Donate to Fund Progeria Gene Therapy!” button_alignment=”center” admin_label=”Donate button” _builder_version=”4.27.4″ _module_preset=”default” button_text_color=”#FFFFFF” button_bg_color=”#00B2E2″ button_border_color=”#00B2E2″ background_layout=”dark” custom_margin=”50px||||false|false” global_colors_info=”{}” theme_builder_area=”post_content”][\/et_pb_button][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>\n","protected":false},"excerpt":{"rendered":"

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\"\"David Liu,\u00a02025 Breakthrough Prize Laureate in the Life Sciences<\/a> for the development of base editing and prime editing, with Sammy Basso at the Brilliant Minds Conference in Sweden in 2023. They received a standing ovation for their presentation on base editing and progeria.<\/strong>
<\/i><\/p>\n