Human gene therapy

Keyphrases

Publications

volume 35, number 13-14, 2024

Julie Vendomèle, Gaëlle Anne Chauveau, Deniz Dalkara, Anne Galy, Sylvain Fisson
Peripheral Cellular Immune Responses Induced by Subretinal Adeno-Associated Virus Gene Transfer Can Be Restrained by the Subretinal-Associated Immune Inhibition Mechanism. Human gene therapy 35 (13-14) (2024)
Maite Muñoz-Melero, Moanaro Biswas
Role of FoxP3 + Regulatory T Cells in Modulating Immune Responses to Adeno-Associated Virus Gene Therapy. Human gene therapy 35 (13-14) (2024)
Sara Rose Krivoshik, Lindsey Dzielak, April R Masters, Jennifer Hall, Alison J Johnson
Development of an Enzyme-Linked Immunosorbent Spot Assay for the Assessment of Adeno-Associated Virus Peptides to Examine Immune Safety. Human gene therapy 35 (13-14) (2024)
Di Cao, Barry J Byrne, Ype P de Jong, Cox Terhorst, Dongsheng Duan, Roland W Herzog, Sandeep R P Kumar
Innate Immune Sensing of Adeno-Associated Virus Vectors. Human gene therapy 35 (13-14) (2024)
Mark A Brimble, Christopher L Morton, Stephen M Winston, Isaiah L Reeves, Yunyu Spence, Pei-Hsin Cheng, Junfang Zhou, Amit C Nathwani, Paul G Thomas, Aisha Souquette, Andrew M Davidoff
Pre-Existing Immunity to a Nucleic Acid Contaminant-Derived Antigen Mediates Transaminitis and Resultant Diminished Transgene Expression in a Mouse Model of Hepatic Recombinant Adeno-Associated Virus-Mediated Gene Transfer. Human gene therapy 35 (13-14) (2024)
Elizabeth Kropf, David M Markusic, Anna Majowicz, Federico Mingozzi, Klaudia Kuranda
Complement System Response to Adeno-Associated Virus Vector Gene Therapy. Human gene therapy 35 (13-14) (2024)

volume 35, number 15-16, 2024

Alex Philippidis
Roche, Ascidian Launch Up-to-$1.8B RNA Exon Editing Collaboration. Human gene therapy 35 (15-16) (2024)
Jan C Klapwijk, Alberto Del Rio Espinola, Silvana Libertini, Philippe Collin, Mick D Fellows, Susan Jobling, Anthony M Lynch, HansJoerg Martus, Catherine Vickers, Andreas Zeller, Luca Biasco, Martijn H Brugman, Frederic D Bushmann, Toni Cathomen, Hildegrund C J Ertl, Richard Gabriel, Guangping Gao, Julie K Jadlowsky, Ian Kimber, Thomas A Lanz, Bruce L Levine, Kenneth P Micklethwaite, Masafumi Onodera, Daniella M Pizzurro, Simon Reed, Michael Rothe, Denise E Sabatino, Jesse J Salk, Axel Schambach, Michael Themis, Jing Yuan
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article. Human gene therapy 35 (15-16) (2024)
Yangmin Wang, Meilin Liu, Xinjian Lin, Haozheng Wang, Na Dong, Hengshen Liu, Hongwei Shao, Wen-Feng Zhang
Genome Editing of Mammalian Cells Through RNA Transcript-Mediated Homologous Recombination Repair. Human gene therapy 35 (15-16) (2024)
Jiajv Chen, Wei Li
The Legal Status and Improvement Path of Human Genetic Data in Gene Therapy in China. Human gene therapy 35 (15-16) (2024)
Charis L Himeda, Takako I Jones, Peter L Jones
Have a Little Heart (or Not): Highly Minimized Skeletal Muscle Regulatory Cassettes with Low or No Activity in the Heart. Human gene therapy 35 (15-16) (2024)
Robert E MacLaren, Isabelle Audo, M Dominik Fischer, Rachel M Huckfeldt, Byron L Lam, Mark E Pennesi, Robert Sisk, James A Gow, Jiang Li, Kan Zhu, So-Fai Tsang
An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study. Human gene therapy 35 (15-16) (2024)

volume 35, number 17-18, 2024

Alex Philippidis
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD. Human gene therapy 35 (17-18) (2024)
Gloria Gonzalez Aseguinolaza
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy . Human gene therapy 35 (17-18) (2024)
Alberto Auricchio
Introduction to ESGCT 2024 Special Issue. Human gene therapy 35 (17-18) (2024)

volume 35, number 3-4, 2024

Zeyu Han, Xianyanling Yi, Jin Li, Dazhou Liao, Guangping Gao, Jianzhong Ai
Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis. Human gene therapy 35 (3-4) (2024)
Zeyu Han, Xianyanling Yi, Jin Li, Dazhou Liao, Guangping Gao, Jianzhong Ai
Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis. Human gene therapy 35 (3-4) (2024)
Rui Li, Ling Zhang, Chen Peng, Yanmei Lu, Zhihao Liu, Xiao Xu, Changyi Wang, Ruijie Hu, Wuping Tan, Liping Zhou, Yueyi Wang, Lilei Yu, Yuhong Wang, Baopeng Tang, Hong Jiang
Chronic Expression of Interleukin-10 Transgene Modulates Cardiac Sympathetic Ganglion Resulting in Reduced Ventricular Arrhythmia. Human gene therapy 35 (3-4) (2024)
Cameron V Lin, Clementine A D Thomas, Thanh L Huynh, David T Wei, Jaime N Young, Anahid S Aivazian, Abigail McInnes, Jixiang Xu, Sarah E Cook, Jessica Vazquez, Ricardo A Maselli
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice. Human gene therapy 35 (3-4) (2024)
Nathan Yingling, Miguel Sena-Esteves, Heather L Gray-Edwards
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market. Human gene therapy 35 (3-4) (2024)
Alex Philippidis
Danaher Partners with Innovative Genomics Institute, Doudna, and Urnov on Beacon for CRISPR Cures. Human gene therapy 35 (3-4) (2024)
Rui Li, Ling Zhang, Chen Peng, Yanmei Lu, Zhihao Liu, Xiao Xu, Changyi Wang, Ruijie Hu, Wuping Tan, Liping Zhou, Yueyi Wang, Lilei Yu, Yuhong Wang, Baopeng Tang, Hong Jiang
Chronic Expression of Interleukin-10 Transgene Modulates Cardiac Sympathetic Ganglion Resulting in Reduced Ventricular Arrhythmia. Human gene therapy 35 (3-4) (2024)
Zeyu Han, Xianyanling Yi, Jin Li, Dazhou Liao, Guangping Gao, Jianzhong Ai
Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis. Human gene therapy 35 (3-4) (2024)

volume 35, number 5-6, 2024

Barry J Byrne, Terence R Flotte, Roland W Herzog, Arun Srivastava
Kenneth I. Berns, MD, PhD [1938-2024]. Human gene therapy 35 (5-6) (2024)
Hongyu Yu, Fang Sun, Yan Xu, Hao Yang, Chongyu Tian, Cong Li, Yimin Kang, Lei Hao, Penghui Yang
Combination Immunotherapy of Oncolytic Flu-Vectored Virus and Programmed Cell Death 1 Blockade Enhances Antitumor Activity in Hepatocellular Carcinoma. Human gene therapy 35 (5-6) (2024)
Lars Thalmann, Natalia Martin-Gonzalez, Dominik Brücher, Andreas Plückthun, Pedro J de Pablo, Maarit Suomalainen, Urs F Greber
Gutless Helper-Dependent and First-Generation HAdV5 Vectors Have Similar Mechanical Properties and Common Transduction Mechanisms. Human gene therapy 35 (5-6) (2024)
Alex Philippidis
Akouos Therapy Restores 11-Year-Old Boy's Hearing After 1 Month. Human gene therapy 35 (5-6) (2024)
Yuka Hayashi, Yoshihide Sehara, Ryota Watano, Kenji Ohba, Yuki Takayanagi, Yoshio Sakiyama, Kazuhiro Muramatsu, Hiroaki Mizukami
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model. Human gene therapy 35 (5-6) (2024)
Louise Medaer, Koenraad R P Veys, Rik Gijsbers
Current Status and Prospects of Viral Vector-Based Gene Therapy to Treat Kidney Diseases. Human gene therapy 35 (5-6) (2024)
Xuehan Sun, Chen Liang, Yangcan Chen, Tongtong Cui, Jiabao Han, Moyu Dai, Ying Zhang, Qi Zhou, Wei Li
Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa. Human gene therapy 35 (5-6) (2024)

volume 35, number 7-8, 2024

Alex Philippidis
Orchard Therapeutics Gains First U.S. Approval for a Metachromatic Leukodystrophy Gene Therapy. Human gene therapy 35 (7-8) (2024)
Michelle Mendiola Pla, Dawn E Bowles
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation. Human gene therapy 35 (7-8) (2024)
Michelle Mendiola Pla, Dawn E Bowles
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation. Human gene therapy 35 (7-8) (2024)
Michelle Mendiola Pla, Dawn E Bowles
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation. Human gene therapy 35 (7-8) (2024)
Rajeev Rai, Zohar Steinberg, Marianna Romito, Federica Zinghirino, Yi-Ting Hu, Nathan White, Asma Naseem, Adrian J Thrasher, Giandomenico Turchiano, Alessia Cavazza
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells. Human gene therapy 35 (7-8) (2024)
Alessandro Romano, Alessandra Mortellaro
The New Frontiers of Gene Therapy and Gene Editing in Inflammatory Diseases. Human gene therapy 35 (7-8) (2024)
Michelle Mendiola Pla, Dawn E Bowles
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation. Human gene therapy 35 (7-8) (2024)
Alessandro Romano, Alessandra Mortellaro
The New Frontiers of Gene Therapy and Gene Editing in Inflammatory Diseases. Human gene therapy 35 (7-8) (2024)
Rajeev Rai, Zohar Steinberg, Marianna Romito, Federica Zinghirino, Yi-Ting Hu, Nathan White, Asma Naseem, Adrian J Thrasher, Giandomenico Turchiano, Alessia Cavazza
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells. Human gene therapy 35 (7-8) (2024)

volume 35, number 9-10, 2024

Tadatoshi Sato, Sachin Chaugule, Matthew B Greenblatt, Guangping Gao, Jae-Hyuck Shim
Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus. Human gene therapy 35 (9-10) (2024)
Chujiao Lin, Matthew B Greenblatt, Guangping Gao, Jae-Hyuck Shim
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases. Human gene therapy 35 (9-10) (2024)
Rebecca Xicluna, Allan Avenel, Céline Vandamme, Marie Devaux, Nicolas Jaulin, Célia Couzinié, Johanne Le Duff, Alicia Charrier, Mickaël Guilbaud, Oumeya Adjali, Gwladys Gernoux
Prevalence Study of Cellular Capsid-Specific Immune Responses to AAV2, 4, 5, 8, 9, and rh10 in Healthy Donors. Human gene therapy 35 (9-10) (2024)
Yangyang Zheng, Xin Xu, Ruoyue Fan, Haolang Jiang, Qingguo Guo, Xuefei Han, Ying Liu, Guangzuo Luo
Intravitreal Delivery of rAAV2-hSyn-hRS1 Results in Retinal Ganglion Cell-Specific Gene Expression and Retinal Improvement in the Rs1 -KO Mouse. Human gene therapy 35 (9-10) (2024)
Tadatoshi Sato, Sachin Chaugule, Matthew B Greenblatt, Guangping Gao, Jae-Hyuck Shim
Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus. Human gene therapy 35 (9-10) (2024)
Chujiao Lin, Matthew B Greenblatt, Guangping Gao, Jae-Hyuck Shim
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases. Human gene therapy 35 (9-10) (2024)
Yangyang Zheng, Xin Xu, Ruoyue Fan, Haolang Jiang, Qingguo Guo, Xuefei Han, Ying Liu, Guangzuo Luo
Intravitreal Delivery of rAAV2-hSyn-hRS1 Results in Retinal Ganglion Cell-Specific Gene Expression and Retinal Improvement in the Rs1 -KO Mouse. Human gene therapy 35 (9-10) (2024)
Chujiao Lin, Matthew B Greenblatt, Guangping Gao, Jae-Hyuck Shim
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases. Human gene therapy 35 (9-10) (2024)
Tadatoshi Sato, Sachin Chaugule, Matthew B Greenblatt, Guangping Gao, Jae-Hyuck Shim
Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus. Human gene therapy 35 (9-10) (2024)
Alex Philippidis
Verve Pauses Enrollment in Base Editing Trial after Adverse Events. Human gene therapy 35 (9-10) (2024)
Alex Philippidis
Verve Pauses Enrollment in Base Editing Trial after Adverse Events. Human gene therapy 35 (9-10) (2024)