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Gene therapy

Banana fruit (Musa sp.) DNA-magnetite nanoparticles: Synthesis, characterization, and biocompatibility assays on normal and cancerous cells

David Arregui-Almeida, Martín Coronel, Karina Analuisa, Carlos Bastidas-Caldes, Santiago Guerrero, Marbel Torres, Andrea Aluisa, Alexis Debut, Werner Brämer-Escamilla, Fernanda Pilaquinga

published 14 Oct 2024

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Dynamic BMP gene expression regulation in chick RPE during recovery from short term optical defocus and form-deprivation

Yan Zhang, Qiurong Zhu, Wulian Song, Grace May Chuang, Daniel Sun, Kiana Cheung, Andreana Chou, Andrea He, Elham Shoghi, Christine F. Wildsoet

published 11 Oct 2024

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In utero adeno-associated virus (AAV)-mediated gene delivery targeting sensory and supporting cells in the embryonic mouse inner ear

Carla Maria Barbosa Spinola, Jacques Boutet de Monvel, Saaid Safieddine, Ghizlène Lahlou, Raphaël Etournay

published 19 Jul 2024

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Clinician’s perspectives on gene therapy for Alzheimer’s disease: A qualitative study

Lilly Kelemen, Ishika Gupta, Zollie Yavarow, Samantha I. Smith, Kim G. Johnson, Nathan A. Boucher

published 18 Jul 2024

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Clinical and analytical validation of an 82-gene comprehensive genome-profiling panel for identifying and interpreting variants responsible for inherited retinal dystrophies

Jacqueline Chan, Jolyon Holdstock, John Shovelton, James Reid, Graham Speight, Duarte Molha, Venu Pullabhatla, Stephanie Carpenter, Ezam Uddin, Takanori Washio, Hiroko Sato, Yuuki Izumi, Reiko Watanabe, Hayato Niiro, Yoshiyuki Fukushima, Naoko Ashida, Takashi Hirose, Akiko Maeda

published 13 Jun 2024

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Electric pulse exposure reduces AAV8 dosage required to transduce HepG2 cells

Yizhou Yao, Robert W. Holdcraft, Susan C. Hagness, John H. Booske

published 30 Apr 2024

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A phase I clinical trial of oncolytic adenovirus mediated suicide and interleukin-12 gene therapy in patients with recurrent localized prostate adenocarcinoma

Shyam Nyati, Hans Stricker, Kenneth N. Barton, Pin Li, Mohamed Elshaikh, Haythem Ali, Stephen L. Brown, Clara Hwang, James Peabody, Svend O. Freytag, Benjamin Movsas, Farzan Siddiqui

published 15 Sep 2023

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AAV2/4-RS1 gene therapy in the retinoschisin knockout mouse model of X-linked retinoschisis

Brittni A. Scruggs, Sajag Bhattarai, Megan Helms, Ioana Cherascu, Adisa Salesevic, Elliot Stalter, Joseph Laird, Sheila A. Baker, Arlene V. Drack

published 07 Dec 2022

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Multiple criteria decision analysis for therapeutic innovations in a hemophilia care center: A pilot study of the organizational impact of innovation in hemophilia care management

Karen Beny, Amélie Dubromel, Benjamin du Sartz de Vigneulles, Valérie Gay, Florence Carrouel, Claude Negrier, Claude Dussart

published 09 Sep 2022

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Inhibition of experimental autoimmune uveitis by intravitreal AAV-Equine-IL10 gene therapy

Elizabeth Crabtree, Katy Uribe, Sara M. Smith, Darby Roberts, Jacklyn H. Salmon, Jacquelyn J. Bower, Liujiang Song, Prabhakar Bastola, Matthew L. Hirsch, Brian C. Gilger

published 18 Aug 2022

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mTOR inhibition as a novel gene therapeutic strategy for diabetic retinopathy

Steven Hyun Seung Lee, Joo Yong Lee, Jun-Sub Choi, Hee Jong Kim, Jin Kim, Seho Cha, Kyoung Jin Lee, Ha-Na Woo, Keerang Park, Heuiran Lee

published 16 Jun 2022

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Assessment of the percentage of full recombinant adeno-associated virus particles in a gene therapy drug using CryoTEM

Mathieu Colomb-Delsuc, Roman Raim, Christian Fiedler, Stefan Reuberger, Johannes Lengler, Rickard Nordström, Martin Ryner, Ioana Mihaela Folea, Barbara Kraus, Juan A. Hernandez Bort, Ida-Maria Sintorn

published 03 Jun 2022

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Improved targeting of human CD4⁺ T cells by nanobody-modified AAV2 gene therapy vectors

Martin V. Hamann, Niklas Beschorner, Xuan-Khang Vu, Ilona Hauber, Ulrike C. Lange, Bjoern Traenkle, Philipp D. Kaiser, Daniel Foth, Carola Schneider, Hildegard Büning, Ulrich Rothbauer, Joachim Hauber

published 20 Dec 2021

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