{"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\/nl\/2025\/08\/08\/gene-editing\/","title":{"rendered":"New Yorker besteedt aandacht aan progeria-genbewerking: PRF is op weg om PROGERIA te GENEZEN!"},"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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Zoals beschreven in het artikel op de voorpagina van de New Yorker van 11 augustus 2025**, getiteld Hoe een uiterst zeldzame ziekte veroudering versnelt,\u00a0<\/em>\u00a0De Progeria Research Foundation is verheugd de baanbrekende Progeria-studie aan te kondigen\u00a0Genbewerkingsprogramma.<\/strong>\u00a0<\/p>\n

**Klik hier<\/strong><\/a> om het volledige artikel te lezen.<\/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

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\u201c"Ik ben al bijna mijn hele leven betrokken bij PRF", zegt de 22-jarige Kaylee uit Ohio, die prominent in het artikel van The New Yorker voorkomt. "Ik ben ontzettend enthousiast over de gentherapie waar ze aan werken!"\u201d<\/strong><\/p>\n

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Basisbewerkingstherapie<\/strong>\u00a0maakt gebruik van CRISPR-technologie om een specifieke DNA-base (of "letter") in de genetische code te targetten en te corrigeren. Omdat Progeria wordt veroorzaakt door een verandering in \u00e9\u00e9n enkele letter in het DNA, is het bij uitstek geschikt voor dit type gentherapie.<\/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

Het Progeria-genenteam <\/strong><\/span><\/h4>\n

PRF werkt samen met een buitengewoon team van wereldberoemde wetenschappers en vernieuwers. Hun onvermoeibare werk en toewijding brengen deze transformatieve therapie tot leven:<\/p>\n

\"\"Van linksboven naar rechtsonder:
Francis Collins, MD, PhD<\/strong>\u00a0\u2013 Langdurige onderzoekspartner voor progeria en voormalig directeur van de National Institutes of Health (NIH)
Michael Erdos, PhD<\/strong>\u00a0\u2013 Associate Investigator, Sectie Moleculaire Genetica, National Human Genome Research Institute (NHGRI)
Leslie Gordon, MD, PhD<\/strong>\u00a0\u2013 Medeoprichter en medisch directeur van PRF, en 's werelds meest vooraanstaande expert op het gebied van Progeria
David Liu, PhD<\/strong>\u00a0\u2013 MIT's Broad Institute en Harvard University wetenschapper, oprichter van\u00a0Basisbewerking<\/strong>\u00a0<\/strong>Gentherapietechnologie
*Sammy Bas<\/strong><\/a>, MS, medeoprichter van The Associazione Italiana Progeria Sammy Basso en PRF-ambassadeur, was een integraal onderdeel van dit team tot aan zijn overlijden in oktober 2024.<\/p>\n

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Van muizenmodel tot klinische proeven<\/strong><\/span><\/h4>\n

Het Progeria Gene Team en hun laboratoriumleden hebben als eersten aangetoond dat genbewerking mogelijk is de levensduur drastisch verhogen<\/strong> van het Progeria-muismodel - door een verbazingwekkende\u00a0140%<\/strong>\u00a0\u2014 een ontdekking die werd gepubliceerd in een van 's werelds belangrijkste wetenschappelijke tijdschriften,\u00a0Natuur<\/em><\/strong><\/a>Voortbouwend op deze opmerkelijke prestatie heeft het team het medicijn verfijnd en richt het zich nu op de stappen die nodig zijn om een klinische proef te starten: het produceren van het medicijn, samenwerken met de Amerikaanse FDA en het ontwikkelen van protocollen voor klinische proeven.<\/p>\n

De reis die voor ons ligt, zal uit meerdere fasen bestaan, technisch ingewikkeld en duur zijn. PRF is vastbesloten om elke uitdaging aan te gaan om gentherapie beschikbaar te maken voor kinderen en jongvolwassenen met 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

Een gemeenschapsgedreven missie<\/strong><\/span><\/h4>\n

De Progeria-gemeenschap heeft ons bij elke stap bijgestaan.<\/strong> Het leiderschapsteam van PRF organiseert regelmatig online bijeenkomsten met progeriafamilies over de hele wereld. Zij zijn onze partners op deze reis naar een genezing.<\/p>\n

Bovendien kunnen de implicaties van dit werk veel verder reiken dan alleen Progeria. Naast het genezen van Progeria banen we een pad dat ook voor andere zeldzame ziekten geldt. <\/strong>Dit zou dus een enorme stap voorwaarts kunnen zijn, niet alleen voor Progeria, maar ook voor andere aandoeningen die lange tijd over het hoofd zijn gezien.<\/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

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"De hoop van ons team is om met behulp van base editing de grondoorzaak van progeria te achterhalen, wat een pad kan openen naar een mogelijke genezing voor kinderen met progeria en tevens de behandeling van miljoenen andere kinderen met dodelijke genetische ziekten kan verbeteren."<\/strong><\/p>\n

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Uw steun is van vitaal belang <\/strong><\/span><\/h4>\n

Gentherapie is het meestbelovende en duurste type medicijn om te produceren. Dankzij de niet aflatende inzet van PRF-fanaten zoals u wordt genbewerking voor progeria werkelijkheid.<\/strong><\/p>\n

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Samen staan we op de drempel van iets bijzonders. <\/strong><\/h4>\n

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Samen, wij ZULLEN<\/em> vind de remedie!<\/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>","protected":false},"excerpt":{"rendered":"

Op 11 augustus 2025 publiceerde deze toppublicatie een diepgaand artikel over de geschiedenis, prestaties en huidige focus van PRF op de gentherapie die ons dichter bij de genezing van progeria zou kunnen brengen. 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\"\"David Liu,\u00a0Winnaar van de Breakthrough Prize 2025 in de levenswetenschappen<\/a> voor de ontwikkeling van base editing en prime editing, met Sammy Basso op de Brilliant Minds Conference in Zweden in 2023. Ze kregen een staande ovatie voor hun presentatie over base editing en progeria.<\/strong>
<\/i><\/p>\n