{"id":52290,"date":"2024-03-15T09:13:20","date_gmt":"2024-03-15T12:13:20","guid":{"rendered":"https:\/\/lnbio.cnpem.br\/divisoes-cientificas\/nucleos-avancados-saude\/recursos-terapia-genica\/"},"modified":"2026-01-16T08:12:42","modified_gmt":"2026-01-16T11:12:42","slug":"recursos-terapia-genica","status":"publish","type":"page","link":"https:\/\/lnbio.cnpem.br\/en\/divisoes-cientificas\/nucleos-avancados-saude\/recursos-terapia-genica\/","title":{"rendered":"Gene Editing Technology Center (NuTEG)"},"content":{"rendered":"

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Gene Editing Technology Center (NuTEG)<\/span><\/h1>\n

[\/vc_column_text][vc_column_text css=”.vc_custom_1696512384777{margin-left: -15px !important;border-left-width: -15px !important;padding-left: -15px !important;}”]\t\t\t\r\n\t\t\t

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  3. Gene Editing Technology Center...<\/span><\/li>\t\t\t\t\t<\/ol>\r\n\t\t\t\t\t
    <\/div>\r\n\t\t\t\t<\/div>\r\n\t\t\t<\/div>\r\n\t\t\t[\/vc_column_text][\/vc_column][vc_column width=”1\/6″][\/vc_column][\/vc_row][vc_row full_width=”stretch_row” gap=”5″ css=”.vc_custom_1710932812600{padding-top: 4rem !important;padding-bottom: 4rem !important;background-image: url(https:\/\/lnbio.cnpem.br\/wp-content\/uploads\/2024\/03\/grafismo-organico-lnbio-cinza.png?id=35463) !important;background-position: center !important;background-repeat: no-repeat !important;background-size: cover !important;}”][vc_column][vc_row_inner][vc_column_inner][vc_column_text css=””]Gene therapy is an innovative treatment and one component of what are known as advanced therapy products (ATP). For more than a decade this area has been evolving worldwide, bringing promising advances in the treatment of human diseases.<\/p>\n

    In late 2023, regulatory agencies in the US and UK approved the first CRISPR\/Cas9-based gene therapy (CASGEVY\u2122), to treat hematologic diseases such as sickle cell anemia and beta thalassemia. Today this treatment has shown excellent results and is expanding globally.<\/p>\n

    In Brazil, ANVISA has already approved 9 gene therapy products, including Zolgensma\u00ae (to treat spinal muscular atrophy, SMA), Kymriah\u00ae (for some types of leukemia and lymphoma), Roctavian\u00ae (for hemophilia A) and Luxturna\u00ae (for hereditary retinal dystrophy). In 2025, Brazil\u2019s Unified Health System (SUS) treated two Brazilian patients with Zolgensma\u00ae for the first time.<\/p>\n

    The LNBio Gene Editing Technology Center (NuTEG) is using advanced technologies like CRISPR\/Cas9, prime editing and base editing to develop gene editing protocols in order to treat human diseases that do not yet have cures. The team has also been working to optimize encapsulation and delivery of these advanced therapy products (ATP) to develop safer and more effective treatments.[\/vc_column_text][\/vc_column_inner][\/vc_row_inner][\/vc_column][\/vc_row][vc_row][vc_column][vc_empty_space][\/vc_column][\/vc_row][vc_row][vc_column][vc_custom_heading text=”Main findings and scientific publications” font_container=”tag:h3|text_align:left” use_theme_fonts=”yes”][vc_row_inner][vc_column_inner width=”1\/3″]\n\t

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    MEF2C repressor variant deregulation leads to cell cycle re-entry and development of heart failure<\/h3>\t\t\t
    Pereira, A.H.M.; Cardoso, A.C.; Consonni, S.R.; Oliveira, R.R.; Saito, A.; Vaggione, M.L.B.; Matos-Souza, J.R.; Carazolle, M.F.; Gon\u00e7alves, A.; Fernandes, J.L.; Ribeiro, G.C.A.; Lopes, M.M.; Molkentin, J.D.; Franchini, K.G<\/div>\t\t<\/div>\n\t\t<\/span>\n\t<\/a>\n\n\t\t<\/div>\n\n\t[\/vc_column_inner][vc_column_inner width=”1\/3″]\n\t
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    Intracellular hyaluronic acid-binding protein 4 (HABP4): a candidate tumor suppressor in colorectal cancer<\/h3>\t\t\t
    Melo-Hanchuk, T.D.; Colleti, C.; Saito*, A.; Mendes, M.C.S.; Cavalheira, J.B.C.; Vassallo, J.; Kobarg, J<\/div>\t\t<\/div>\n\t\t<\/span>\n\t<\/a>\n\n\t\t<\/div>\n\n\t[\/vc_column_inner][vc_column_inner width=”1\/3″]\n\t
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    Obesity-Linked PPAR\u03b3 Ser273 Phosphorylation Promotes Beneficial Effects on the Liver, despite Reduced Insulin Sensitivity in Mice<\/h3>\t\t\t
    TERRA, MAIARA FERREIRA ; GARC\u00cdA-AR\u00c9VALO, MARTA ; AVELINO, THAYN\u00c1 MENDON\u00c7A ; DEGAKI, KARINA Y. ; DE CARVALHO, MURILO ; TORRES, FELIPE RAFAEL ; SAITO, ANGELA ; FIGUEIRA, ANA CAROLINA MIGLIORINI<\/div>\t\t<\/div>\n\t\t<\/span>\n\t<\/a>\n\n\t\t<\/div>\n\n\t[\/vc_column_inner][\/vc_row_inner][\/vc_column][\/vc_row][vc_row full_width=”stretch_row” css=”.vc_custom_1696434766725{background-color: #ffffff !important;background-position: center !important;background-repeat: no-repeat !important;background-size: cover !important;}”][vc_column][vc_empty_space height=”32″][\/vc_column][\/vc_row][vc_row full_width=”stretch_row” css=”.vc_custom_1696515988446{background-color: #eff2f5 !important;background-position: center !important;background-repeat: no-repeat !important;background-size: cover !important;}”][vc_column][vc_empty_space height=”16px”][vc_custom_heading text=”meet our team” font_container=”tag:h3|text_align:left” use_theme_fonts=”yes” css=””][\/vc_column][\/vc_row][vc_row full_width=”stretch_row” content_placement=”middle” css=”.vc_custom_1696516377387{background-color: #eff2f5 !important;background-position: center !important;background-repeat: no-repeat !important;background-size: cover !important;}”][vc_column width=”1\/4″]
    \"\"<\/span><\/div>

    \u00c2ngela Saito<\/h2>
    Lead researcher<\/div>

    See Lattes CV<\/a><\/p><\/div>

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    Angelica Jardim Costa<\/h2>
    Adjunct Researcher<\/div>

    See Lattes CV<\/a><\/p><\/div>

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    Caroline Mantovani da Luz<\/h2>
    Specialist<\/div>

    See Lattes CV<\/a><\/p><\/div>

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    Raphael Morales Neto<\/h2>
    Specialist<\/div>

    See Lattes CV<\/a><\/p><\/div>

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    Gabriel Viliod Vieira<\/h2>
    Specialist<\/div>

    See Lattes CV<\/a><\/p><\/div>

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    Karina Yumi Degaki<\/h2>
    Analyst<\/div>

    See Lattes CV<\/a><\/p><\/div>

    <\/div><\/div><\/div><\/div>[\/vc_column][vc_column width=”1\/4″][\/vc_column][vc_column width=”1\/4″][\/vc_column][\/vc_row][vc_row full_width=”stretch_row” css=”.vc_custom_1696434766725{background-color: #ffffff !important;background-position: center !important;background-repeat: no-repeat !important;background-size: cover !important;}”][vc_column][vc_empty_space height=”40″][\/vc_column][\/vc_row][vc_row full_width=”stretch_row” css=”.vc_custom_1705339428945{background-color: #ffffff !important;background-position: center !important;background-repeat: no-repeat !important;background-size: cover !important;}”][vc_column][vc_row_inner][vc_column_inner][vc_custom_heading text=”All groups in advanced health technologies” font_container=”tag:h3|text_align:left|color:%23111111″ use_theme_fonts=”yes”][vc_separator color=”green” align=”align_left” border_width=”5″ el_width=”10″ css_animation=”slideInLeft”][vc_empty_space][\/vc_column_inner][\/vc_row_inner][\/vc_column][\/vc_row][vc_row full_width=”stretch_row” css=”.vc_custom_1710441827678{background-color: #ffffff !important;background-position: center !important;background-repeat: no-repeat !important;background-size: cover !important;}”][vc_column][vc_row_inner][vc_column_inner]