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Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation Gene Ther. (IF 5.1) Pub Date : 2024-03-14 Maria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, Frauke Naumann-Winter, Enrico Costa, Darius Matusevicius, Dinah M. Duarte, Eva Malikova, Dinko Vitezic, Kristina Larsson, Armando Magrelli, Violeta Stoyanova-Beninska, Segundo Mariz
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A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice Gene Ther. (IF 5.1) Pub Date : 2024-03-12 Felix Krause, Katja Schmidtke, Mailton Franca de Vasconcelos, David Schmidt, Beyza Cansiz, Franziska Theisen, Melanie D. Mark, Max O. Rybarski
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Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate Gene Ther. (IF 5.1) Pub Date : 2024-03-01 Mathieu Guerriaud, Cyril Poupet, Zineb Lakhrif, Evelyne Kohli, Nathalie Moiré
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Analytical characterization of full, intermediate, and empty AAV capsids Gene Ther. (IF 5.1) Pub Date : 2024-02-19 Aisleen McColl-Carboni, Serena Dollive, Sarah Laughlin, Rudenc Lushi, Michael MacArthur, Shanshan Zhou, Jeffrey Gagnon, Christopher A. Smith, Brenda Burnham, Robert Horton, Dimpal Lata, Brianna Uga, Kalyani Natu, Emmanuela Michel, Celia Slater, Evan DaSilva, Robert Bruccoleri, Tim Kelly, James B. McGivney
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Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia Gene Ther. (IF 5.1) Pub Date : 2024-02-14 Ingrid Pabinger, Mila Ayash-Rashkovsky, Miguel Escobar, Barbara A. Konkle, María Eva Mingot-Castellano, Eric S. Mullins, Claude Negrier, Luying Pan, Kavitha Rajavel, Brian Yan, John Chapin
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Gene therapy corrects the neurological deficits of mice with sialidosis Gene Ther. (IF 5.1) Pub Date : 2024-02-07 Wuh-Liang Hwu, Karine Chang, Yu-Han Liu, Hao-Chun Wang, Ni-Chung Lee, Yin-Hsiu Chien
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Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses Gene Ther. (IF 5.1) Pub Date : 2024-01-26 María del Pilar Plastine, Sabrina Amalfi, María Gabriela López, María José Gravisaco, Oscar Taboga, Victoria Alfonso
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Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/− Gene Ther. (IF 5.1) Pub Date : 2024-01-26 Shannon M. McNamee, Natalie P. Chan, Monica Akula, Marielle O. Avola, Maiya Whalen, Kaden Nystuen, Pushpendra Singh, Arun K. Upadhyay, Margaret M. DeAngelis, Neena B. Haider
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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes Gene Ther. (IF 5.1) Pub Date : 2024-01-10 Brahim Chaqour, Thu T. Duong, Jipeng Yue, Tehui Liu, David Camacho, Kimberly E. Dine, Julian Esteve-Rudd, Scott Ellis, Jean Bennett, Kenneth S. Shindler, Ahmara G. Ross
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Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea Gene Ther. (IF 5.1) Pub Date : 2024-01-10 Hyeokgyo Jeong, Sujata Purja, Eunyoung Kim
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Ghrelin mediated cardioprotection using in vitro models of oxidative stress Gene Ther. (IF 5.1) Pub Date : 2024-01-04 Cindy Y. Kok, George Ghossein, Sindhu Igoor, Renuka Rao, Tracy Titus, Shinya Tsurusaki, James JH. Chong, Eddy Kizana
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CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering Gene Ther. (IF 5.1) Pub Date : 2024-01-04 Shahin Eghbalsaied, Clancy Lawler, Björn Petersen, Raul A. Hajiyev, Steve R. Bischoff, Stephen Frankenberg
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CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy Gene Ther. (IF 5.1) Pub Date : 2023-12-22 Emrah Gumusgoz, Sahba Kasiri, Mayank Verma, Jun Wu, Daniel Villarreal Acha, Ummay Marriam, Sharyl Fyffe-Maricich, Amy Lin, Xin Chen, Steven J. Gray, Berge A. Minassian
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Distributional comparison of different AAV vectors after unilateral cochlear administration Gene Ther. (IF 5.1) Pub Date : 2023-12-14 Shuang Han, Zhijiao Xu, Shengyi Wang, Honghai Tang, Shaowei Hu, Hui Wang, Guofang Guan, Yilai Shu
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Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver Gene Ther. (IF 5.1) Pub Date : 2023-11-27 Carola J. Maturana
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Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa Gene Ther. (IF 5.1) Pub Date : 2023-11-27 Nchangwi Syntia Munung, Obiageli E. Nnodu, Patrick Ohiani Moru, Akpaka A. Kalu, Benido Impouma, Marsha J. Treadwell, Ambroise Wonkam
Somatic gene therapy will be one of the most exciting practices of genetic medicine in Africa and is primed to offer a “new life” for persons living with sickle cell disease (SCD). Recently, successful gene therapy trials for SCD in the USA have sparked a ray of hope within the SCD community in Africa. However, the high cost, estimated to exceed 1.5 million USD, continues to be a major concern for
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Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement Gene Ther. (IF 5.1) Pub Date : 2023-11-20 A. Mateu-Bosch, E. Segur-Bailach, J. García-Villoria, S. Gea-Sorlí, I. Ruiz, J. del Rey, J. Camps, M. Guitart-Mampel, G. Garrabou, F. Tort, A. Ribes, C. Fillat
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Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability Gene Ther. (IF 5.1) Pub Date : 2023-11-15 Evgeny S. Nikitin, Tatiana Y. Postnikova, Elena Y. Proskurina, Anastasia A. Borodinova, Violetta Ivanova, Matvey V. Roshchin, Maria P. Smirnova, Ilya Kelmanson, Vsevolod V. Belousov, Pavel M. Balaban, Aleksey V. Zaitsev
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Approval and therapeutic value of gene therapies in the US and Europe Gene Ther. (IF 5.1) Pub Date : 2023-11-08 Kerstin N. Vokinger, Camille E. G. Glaus, Aaron S. Kesselheim
Gene therapies are a fast-growing area of innovation and hold promise for the treatment of many diseases currently with unmet medical need. To better understand the clinical importance of the current landscape of approved gene therapies, we conducted a systematic analysis of the approved gene therapies and their added therapeutic value. Through December 2022, 13 gene therapies have been approved in
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Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute Gene Ther. (IF 5.1) Pub Date : 2023-11-08 Lea Witkowsky, Matthew Norstad, Audrey R. Glynn, Melinda Kliegman
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Successes and challenges in clinical gene therapy Gene Ther. (IF 5.1) Pub Date : 2023-11-08 Donald B. Kohn, Yvonne Y. Chen, Melissa J. Spencer
Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provide enduring treatments for increasing number of diseases. There are active clinical trials approaching a variety of inherited and acquired disorders of different organ systems. Approaches include ex vivo modification of hematologic stem cells (HSC), T lymphocytes and other immune
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The estimated annual financial impact of gene therapy in the United States Gene Ther. (IF 5.1) Pub Date : 2023-11-08 Chi Heem Wong, Dexin Li, Nina Wang, Jonathan Gruber, Andrew W. Lo, Rena M. Conti
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A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies Gene Ther. (IF 5.1) Pub Date : 2023-10-30 Shadi Saleh, Omar Dabbous, Sean D. Sullivan, Dipen Ankleshwaria, Daiane Trombini, Mondher Toumi, Mahmoud Diaa, Anish Patel, Burcu Kazazoglu Taylor, Sean Tunis
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Implications of maternal-fetal health on perinatal stem cell banking Gene Ther. (IF 5.1) Pub Date : 2023-10-26 Dandan Zhu, Mehri Barabadi, Courtney McDonald, Gina Kusuma, Ishmael Miguel Inocencio, Rebecca Lim
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Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques Gene Ther. (IF 5.1) Pub Date : 2023-10-13 Alexander Kistner, Jessica A. Chichester, Lili Wang, Roberto Calcedo, Jenny A. Greig, Leah N. Cardwell, Margaret C. Wright, Julien Couthouis, Sunjay Sethi, Brian E. McIntosh, Kathleen McKeever, Samuel Wadsworth, James M. Wilson, Emil Kakkis, Barbara A. Sullivan
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p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma Gene Ther. (IF 5.1) Pub Date : 2023-10-13 Naomi Muramatsu, Misa Ichikawa, Tomoko Katagiri, Yumi Taguchi, Takashi Hatanaka, Tomoyuki Okuda, Hirokazu Okamoto
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Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo Gene Ther. (IF 5.1) Pub Date : 2023-09-26 Gabriela Toro Cabrera, Katharina E. Meijboom, Abbas Abdallah, Helene Tran, Zachariah Foster, Alexandra Weiss, Nicholas Wightman, Rachel Stock, Tania Gendron, Alisha Gruntman, Anthony Giampetruzzi, Leonard Petrucelli, Robert H. Brown, Christian Mueller
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Acoustically targeted noninvasive gene therapy in large brain volumes Gene Ther. (IF 5.1) Pub Date : 2023-09-12 Shirin Nouraein, Sangsin Lee, Vidal A. Saenz, Huckie C. Del Mundo, Joycelyn Yiu, Jerzy O. Szablowski
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Long-term effects of a fat-directed FGF21 gene therapy in aged female mice Gene Ther. (IF 5.1) Pub Date : 2023-09-12 Jacqueline M. Anderson, W. David Arnold, Wei Huang, Alissa Ray, Gregory Owendoff, Lei Cao
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A pilot study to determine the optimal dose of scAAVIL-1ra in a large animal model of post-traumatic osteoarthritis Gene Ther. (IF 5.1) Pub Date : 2023-09-11 P. Thampi, K. A. Seabaugh, L. M. Pezzanite, C. R. Chu, J. N. Phillips, J. C. Grieger, C. W. McIlwraith, R. J. Samulski, L. R. Goodrich
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Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic Gene Ther. (IF 5.1) Pub Date : 2023-08-23 Mark Singh, Andrew Brooks, Parto Toofan, Keith McLuckie
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Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases Gene Ther. (IF 5.1) Pub Date : 2023-08-18 Duncan J. Stewart, Albert Gianchetti, Dawn Byrnes, Howard C. Dittrich, Barb Thorne, Linda L. Manza, Rickey R. Reinhardt
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Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates Gene Ther. (IF 5.1) Pub Date : 2023-08-17 Tricia H. Burdo, Chen Chen, Rafal Kaminski, Ilker K. Sariyer, Pietro Mancuso, Martina Donadoni, Mandy D. Smith, Rahsan Sariyer, Maurizio Caocci, Shuren Liao, Hong Liu, Wenwen Huo, Huaqing Zhao, John Misamore, Mark G. Lewis, Vahan Simonyan, Ethan Y. Xu, Thomas J. Cradick, Jennifer Gordon, Kamel Khalili
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Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair Gene Ther. (IF 5.1) Pub Date : 2023-08-11 Jennifer A. Bell, Kevin Collon, Cory Mayfield, Matthew C. Gallo, Stephanie W. Chang, Osamu Sugiyama, Amy H. Tang, Roger Hollis, Shefali Chopra, Donald B. Kohn, Jay R. Lieberman
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Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery Gene Ther. (IF 5.1) Pub Date : 2023-08-09 Sarah Klinnert, Corinne D. Schenkel, Patrick C. Freitag, Huldrych F. Günthard, Andreas Plückthun, Karin J. Metzner
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CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease Gene Ther. (IF 5.1) Pub Date : 2023-08-04 Luis Fernando Narváez-Pérez, Francisco Paz-Bermúdez, José Arturo Avalos-Fuentes, Aurelio Campos-Romo, Benjamín Florán-Garduño, José Segovia
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Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice Gene Ther. (IF 5.1) Pub Date : 2023-07-27 Kaiyi Zhou, Meng Yuan, Jiabao Sun, Feixu Zhang, Xiaoying Zong, Zhanao Li, Dingyue Tang, Lichen Zhou, Jing Zheng, Xiao Xiao, Xia Wu
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Immunobiology of a rationally-designed AAV2 capsid following intravitreal delivery in mice Gene Ther. (IF 5.1) Pub Date : 2023-06-29 Michael Whitehead, Andrew Sage, Tom Burgoyne, Andrew Osborne, Patrick Yu-Wai-Man, Keith R. Martin
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Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo Gene Ther. (IF 5.1) Pub Date : 2023-06-15 Neda A. M. Nafchi, Ellie M. Chilcott, Sharon Brown, Heidi R. Fuller, Melissa Bowerman, Rafael J. Yáñez-Muñoz
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Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber Gene Ther. (IF 5.1) Pub Date : 2023-05-24 Kristina J. Chern, Kimicia Z. Issac, Zendorf D. Gumbs, Merissa E. O’Connor, Matthew S. Lawrence, Daniel M. Lipinski
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RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells Gene Ther. (IF 5.1) Pub Date : 2023-05-12 Lucie Bouquet, Elodie Bôle-Richard, Walid Warda, Mathieu Neto Da Rocha, Rim Trad, Clémentine Nicod, Rafik Haderbache, Delphine Genin, Christophe Ferrand, Marina Deschamps
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Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing Gene Ther. (IF 5.1) Pub Date : 2023-05-10 Martin Donnelley, Patricia Cmielewski, Emma Knight, Chantelle Carpentieri, Alexandra McCarron, Nathan Rout-Pitt, David Parsons, Nigel Farrow
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Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies Gene Ther. (IF 5.1) Pub Date : 2023-05-10 Daniel Stone, Martine Aubert, Keith R. Jerome
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Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings Gene Ther. (IF 5.1) Pub Date : 2023-04-25 Deepa H. Chand, Rui Sun, Karim A. Diab, Damien Kenny, Francis Fonyuy Tukov
Human gene replacement therapies such as onasemnogene abeparvovec (OA) use recombinant adeno-associated virus (rAAV) vectors to treat monogenic disorders. The heart and liver are known target organs of toxicity in animals; with cardiac and hepatic monitoring recommended in humans after OA dosing. This manuscript provides a comprehensive description of cardiac data from preclinical studies and clinical
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rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality Gene Ther. (IF 5.1) Pub Date : 2023-04-19 Seyedeh Zeinab Mirjalili Mohanna, Andrea J. Korecki, Elizabeth M. Simpson
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Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures Gene Ther. (IF 5.1) Pub Date : 2023-04-07 Esbjörn Melin, My Andersson, Casper R. Gøtzsche, Jenny Wickham, Yuzhe Huang, Julia Alicja Szczygiel, Arnie Boender, Søren H. Christiansen, Lars Pinborg, David P. D. Woldbye, Merab Kokaia
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Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor Gene Ther. (IF 5.1) Pub Date : 2023-03-28 Mikako Wada, Naoya Uchida, Guillermo Posadas-Herrera, Hiromi Hayashita-Kinoh, Yuji Tsunekawa, Yukihiko Hirai, Takashi Okada
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Induced pluripotent stem cell-derived dendritic cell vaccine therapy genetically modified on the ubiquitin-proteasome system Gene Ther. (IF 5.1) Pub Date : 2023-03-23 Shinta Tominaga, Toshiyasu Ojima, Motoki Miyazawa, Hiromitsu Iwamoto, Junya Kitadani, Shimpei Maruoka, Keiji Hayata, Hiroki Yamaue
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Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model Gene Ther. (IF 5.1) Pub Date : 2023-03-20 Simna SP, Rajendra N. Mitra, Min Zheng, Jared D. Chrispell, Kai Wang, Yong-Su Kwon, Ellen R. Weiss, Zongchao Han
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Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets Gene Ther. (IF 5.1) Pub Date : 2023-03-14 Apeksha Dave, Cara L. Berkowitz, Valerie L. Luks, Brandon M. White, Rohan Palanki, Marco D. Carpenter, John S. Riley, Sourav K. Bose, Haiying Li, Li Li, Pallavi V. Menon, Shiva Teerdhala, Mina Ebrahimi, Philip W. Zoltick, William H. Peranteau
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Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology Gene Ther. (IF 5.1) Pub Date : 2023-02-24 Barış Genç, Boram Nho, Hana Seung, Benjamin Helmold, Huiwon Park, Öge Gözütok, Seunghyun Kim, Jinil Park, Sanghyun Ye, Haneul Lee, Nayeon Lee, Seung-Shin Yu, Sunyoung Kim, Junghun Lee, Hande Özdinler
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Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail Gene Ther. (IF 5.1) Pub Date : 2023-02-24 Victor Solodushko, Brian Fouty
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Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9 Gene Ther. (IF 5.1) Pub Date : 2023-02-14 Pouiré Yaméogo, Catherine Gérard, Nathalie Majeau, Jacques P. Tremblay
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Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression Gene Ther. (IF 5.1) Pub Date : 2023-02-13 Rikke Hahn Kofoed, Elizabeth M. Simpson, Kullervo Hynynen, Isabelle Aubert
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Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy Gene Ther. (IF 5.1) Pub Date : 2023-02-10 Jiali Nie, Yu Han, Zhiyuan Jin, Weijian Hang, Hongyang Shu, Zheng Wen, Li Ni, Dao Wen Wang
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Reversible stabilization of DNA/PEI complexes by reducible click-linkage between DNA and polymer. A new polyplex concept for lowering polymer quantity Gene Ther. (IF 5.1) Pub Date : 2023-02-09 Delphine Maze, Chantal Pichon, Patrick Midoux
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Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis Gene Ther. (IF 5.1) Pub Date : 2023-02-02 Amira D. Rghei, Jacob G. E. Yates, Jordyn A. Lopes, Xuiaoyan Zhan, Matthew M. Guilleman, Yanlong Pei, Laura P. van Lieshout, Lisa A. Santry, Byram W. Bridle, Khalil Karimi, Brad Thompson, Leonardo Susta, James E. Crowe, Sarah K. Wootton
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Ptbp1 knockdown failed to induce astrocytes to neurons in vivo Gene Ther. (IF 5.1) Pub Date : 2023-02-01 Guixiang Yang, Zixiang Yan, Xiaoqing Wu, Meng Zhang, Chunlong Xu, Linyu Shi, Hui Yang, Kailun Fang
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Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters Gene Ther. (IF 5.1) Pub Date : 2023-01-13 Bart Nieuwenhuis, Elise Laperrousaz, James R. Tribble, Joost Verhaagen, James W. Fawcett, Keith R. Martin, Pete A. Williams, Andrew Osborne
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Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease Gene Ther. (IF 5.1) Pub Date : 2023-01-11 Jey M. Jeyakumar, Azadeh Kia, Lawrence C. S. Tam, Jenny McIntosh, Justyna Spiewak, Kevin Mills, Wendy Heywood, Elisa Chisari, Noemi Castaldo, Daniël Verhoef, Paniz Hosseini, Petya Kalcheva, Clement Cocita, Carlos J. Miranda, Miriam Canavese, Jaminder Khinder, Cecilia Rosales, Derralynn Hughes, Rose Sheridan, Romuald Corbau, Amit Nathwani