Publications by authors named "Jonathan Yen"

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A mouse model for hemoglobin SC disease recapitulates characteristic human pathologies.
Jinbin Zhai, James B Papizan, Yu Yao, Elizabeth D Arnold, Jing Yu, Jonathan S Yen

Blood Adv· December 2025

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Engraftment and persistence of base-edited hematopoietic stem cells in nonhuman primates.
Stefan Radtke, Emily Fields, Kyle Swing, Greta Kanestrom, Jonathan S Yen

Sci Transl Med· August 2025

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Preclinical development of lentiviral vector gene therapy for Diamond-Blackfan anemia syndrome.
Senthil Velan Bhoopalan, Thiyagaraj Mayuranathan, Nana Liu, Kalin Mayberry, Yu Yao, Jonathan S Yen

Mol Ther· July 2025

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BCL11A-deficient human erythropoiesis is impaired in vitro and after xenotransplantation into mice.
Yoonjeong Jang, Ruopeng Feng, Lance E Palmer, Thiyagaraj Mayuranathan, Yu Yao, Jonathan S Yen

Blood Adv· June 2025

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Multiplex base editing to protect from CD33 directed drugs for immune and gene therapy.
Florence Borot, Olivier Humbert, Jeffrey T Ehmsen, Emily Fields, Sajeev Kohli, Jonathan S Yen

Nat Commun· May 2025

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FLT1 and other candidate fetal haemoglobin modifying loci in sickle cell disease in African ancestries.
Ambroise Wonkam, Kevin Esoh, Rachel M Levine, Valentina Josiane Ngo Bitoungui, Khuthala Mnika, Jonathan S Yen

Nat Commun· March 2025

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34 cell therapy to induce fetal hemoglobin for sickle cell disease.
Varun Katta, Kiera O'Keefe, Yichao Li, Thiyagaraj Mayuranathan, Cicera R Lazzarotto, Jonathan S Yen

Mol Ther· October 2024

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Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.
Xizhen Lian, Sumanta Chatterjee, Yehui Sun, Sean A Dilliard, Stephen Moore, Jonathan S Yen

Nat Nanotechnol· September 2024

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Potent and uniform fetal hemoglobin induction via base editing.
Thiyagaraj Mayuranathan, Gregory A Newby, Ruopeng Feng, Yu Yao, Kalin D Mayberry, Jonathan S Yen

Nat Genet· July 2023

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Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
Kelcee A Everette, Gregory A Newby, Rachel M Levine, Kalin Mayberry, Yoonjeong Jang, Jonathan S Yen

Nat Biomed Eng· May 2023

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Gene Therapy and Gene Editing for β-Thalassemia.
Georgios E Christakopoulos, Raul Telange, Jonathan Yen, Mitchell J Weiss

Hematol Oncol Clin North Am· April 2023

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Editing human hematopoietic stem cells: advances and challenges.
Senthil Velan Bhoopalan, Jonathan S Yen, Rachel M Levine, Akshay Sharma

Cytotherapy· March 2023

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Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene Therapy.
Florence Borot, Olivier Humbert, Gregory A Newby, Emily Fields, Sajeev Kohli, Jonathan S Yen

bioRxiv· February 2023

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An RPS19-edited model for Diamond-Blackfan anemia reveals TP53-dependent impairment of hematopoietic stem cell activity.
Senthil Velan Bhoopalan, Jonathan S Yen, Thiyagaraj Mayuranathan, Kalin D Mayberry, Yu Yao

JCI Insight· January 2023

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Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations.
Kaitly J Woodard, Phillip A Doerfler, Kalin D Mayberry, Akshay Sharma, Rachel Levine, Jonathan Yen

Dis Model Mech· June 2022

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Base editing of haematopoietic stem cells rescues sickle cell disease in mice.
Gregory A Newby, Jonathan S Yen, Kaitly J Woodard, Thiyagaraj Mayuranathan, Cicera R Lazzarotto

Nature· July 2021

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Directed evolution of adenine base editors with increased activity and therapeutic application.
Nicole M Gaudelli, Dieter K Lam, Holly A Rees, Noris M Solá-Esteves, Luis A Barrera, Jonathan Yen

Nat Biotechnol· July 2020

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TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells.
Jonathan Yen, Michael Fiorino, Yi Liu, Steve Paula, Scott Clarkson

Sci Rep· November 2018

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Degradable and biocompatible hydrogels bearing a hindered urea bond.
Hanze Ying, Jonathan Yen, Ruibo Wang, Yang Lai, Jer-Luen-Aaron Hsu

Biomater Sci· November 2017

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Selective in vivo metabolic cell-labeling-mediated cancer targeting.
Hua Wang, Ruibo Wang, Kaimin Cai, Hua He, Yang Liu, Jonathan Yen

Nat Chem Biol· April 2017

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CD44 Mediated Nonviral Gene Delivery into Human Embryonic Stem Cells via Hyaluronic-Acid-Coated Nanoparticles.
Jonathan Yen, Hanze Ying, Hua Wang, Lichen Yin, Fatih Uckun

ACS Biomater Sci Eng· March 2016

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Redox-Responsive Self-Assembled Chain-Shattering Polymeric Therapeutics.
Kaimin Cai, Jonathan Yen, Qian Yin, Yang Liu, Ziyuan Song

Biomater Sci· July 2015

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Non-invasive, real-time reporting drug release in vitro and in vivo.
Yanfeng Zhang, Qian Yin, Jonathan Yen, Joanne Li, Hanze Ying

Chem Commun (Camb)· April 2015

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Bioorthogonal Oxime Ligation Mediated Cancer Targeting.
Li Tang, Qian Yin, Yunxiang Xu, Qin Zhou, Kaimin Cai, Jonathan Yen

Chem Sci· April 2015

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Enhanced Non-Viral Gene Delivery to Human Embryonic Stem Cells via Small Molecule-Mediated Transient Alteration of Cell Structure.
Jonathan Yen, Lichen Yin, Jianjun Cheng

J Mater Chem B· January 2014

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