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Combining CRISPR/Cas mediated terminal resolution with a novel genetic workflow to achieve high-diversity adenoviral libraries Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-18 Julian Fischer, Ariana Fedotova, Lena Jaki, Erwan Sallard, Anja Erhardt, Jonas Fuchs, Zsolt Ruzsics
While recombinant Adenoviruses are widely used in both laboratory and medical gene transfer, library-based applications using this vector platform are not readily available. Recently, we developed a new method, the CRISPR/Cas9 mediated terminal resolution aiding high-efficiency rescue of recombinant Adenoviruses from recombinant DNA. Here we report on a genetic workflow that allows construction of
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Strengthening health systems for access to gene therapy in rare genetic disorders Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-13 Sonal Bhatia, Yann Le Cam, Juan Carrion, Lauren Diamond, Paul Fennessy, Safiyya Gassman, Felix Gutzwiller, Stephen Kagan, Diana Pankevich, Jennifer Young Maloney, Nitin Mahadev, Martin Schulz, Durhane Wong-Rieger, Paolo Morgese
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AAV-delivered hepato-adrenal cooperativity in steroidogenesis: implications for gene therapy for congenital adrenal hyperplasia Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-12 Lara E. Graves, Eva B. van Dijk, Erhua Zhu, Sundar Koyyalamudi, Tiffany Wotton, Dinah Sung, Shubha Srinivasan, Samantha L. Ginn, Ian E. Alexander
Despite the availability of life-saving corticosteroids for 70 years, treatment for adrenal insufficiency is not able to recapitulate physiological diurnal cortisol secretion and results in numerous complications. Gene therapy is an attractive possibility for monogenic adrenocortical disorders such as congenital adrenal hyperplasia, however, requires further development of gene transfer/editing technologies
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Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-12 Hao Liu, Yue Zhang, Mitchell Yip, Lingzhi Ren, Jialing Liang, Xiupeng Chen, Nan Liu, Ailing Du, Jiaming Wang, Hao Chang, Hyejin Oh, Chen Zhou, Ruxiao Xing, Mengyao Xu, Peiyi Guo, Dominic Gessler, Jun Xie, Phillip WL. Tai, Guangping Gao, Dan Wang
Recombinant adeno-associated virus (rAAV)-based gene therapy is entering clinical and commercial stages at an unprecedented pace. Triple transfection of HEK293 cells is currently the most widely used platform for rAAV manufacturing. Here, we develop low-cis triple transfection that reduces the transgene plasmid usage by 10- to 100-fold, and overcomes several major limitations associated with standard
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Novel AAV variants with improved tropism for human Schwann cells Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-11 Matthieu Drouyer, Tak-Ho Chu, Elodie Labit, Florencia Haase, Renina Gale Navarro, Deborah Nazareth, Nicole Rosin, Jessica Merjane, Suzanne Scott, Marti Cabanes-Creus, Adrian Westhaus, Erhua Zhu, Rajiv Midha, Ian E. Alexander, Jeff Biernaskie, Samantha L. Ginn, Leszek Lisowski
Gene therapies and associated technologies are transforming biomedical research and enabling novel therapeutic options for patients living with debilitating and incurable genetic disorders. The vector system based on recombinant adeno-associated viral vectors (AAVs) has shown great promise in recent clinical trials for genetic diseases of multiple organs, such as the liver and the nervous system. Despite
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Non-canonical capsid engineering highlights new possibilities for AAV vectorology Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-06 Zehan Zhang, John R. Counsell
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The very stable lentiviral vector Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-05 Gwendolyn K. Binder, Chien-Chung Chen
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Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success? Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-05 Stefano Rivella
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Embryo and fetal gene editing: Technical challenges and progress toward clinical applications Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-04 Citra N.Z. Mattar, Wei Leong Chew, Poh San Lai
Gene modification therapies (GMTs) are slowly but steadily making progress toward clinical application. As the majority of rare diseases have an identified genetic cause, and as rare diseases collectively affect 5% of the global population, it is increasingly important to devise gene correction strategies to address the root causes of the most devastating of these diseases and to provide access to
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A robust and flexible baculovirus-insect cell system for AAV vector production with improved yield, capsid ratios and potency Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-04 Yoko Marwidi, Hoang-Oanh B. Nguyen, David Santos, Tenzin Wangzor, Sumita Bhardwaj, Gabriel Ernie, Gregg Prawdzik, Garrett Lew, David Shivak, Michael Trias, Jada Padilla, Hung Tran, Kathleen Meyer, Richard Surosky, Alex Michael Ward
Manufacturing of adeno-associated viruses (AAV) for gene and cell therapy applications has increased significantly and spurred development of improved mammalian and insect cell-based production systems. We developed a baculovirus-based insect cell production system—the SGMO Helper—with a novel gene architecture and greater flexibility to modulate the expression level and content of individual Rep and
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Measurement solutions and standards for advanced therapy Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-02 Lili Wang, Samantha Maragh, Edward Kwee, Jamie L. Almeida, Sierra D. Miller, Tara Eskandari, Sheng Lin-Gibson
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A positive take on negative selection for CAR-T manufacturing Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-03-01 Rimas J. Orentas
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Manufacturing DNA in E. coli yields higher-fidelity DNA than in vitro enzymatic synthesis Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-28 Steven J. Hersch, Siddarth Chandrasekaran, Jamie Lam, Nafiseh Nafissi, Roderick A. Slavcev
Biotechnologies such as gene therapy have brought DNA vectors to the forefront of pharmaceuticals. The quality of starting material plays a pivotal role in determining final product quality. Here, we examined the fidelity of DNA replication using enzymatic methods () compared to plasmid DNA produced in . Next-generation sequencing approaches rely on polymerases, which have inherent limitations in sensitivity
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Peptide-encoding gene transfer to modulate intracellular protein-protein interactions Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-28 Toshihiko Taya, Daisuke Kami, Fumiya Teruyama, Satoaki Matoba, Satoshi Gojo
Peptide drug discovery has great potential, but the cell membrane is a major obstacle when the target is an intracellular protein-protein interaction (PPI). It is difficult to target PPIs with small molecules; indeed, there are no intervention tools that can target any intracellular PPI. In this study, we developed a platform that enables the introduction of peptides into cells via mRNA-based gene
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Endovascular transplantation of mRNA-enhanced mesenchymal stromal cells results in superior therapeutic protein expression in swine heart Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-27 Jonathan Al-Saadi, Mathias Waldén, Mikael Sandell, Jesper Sohlmér, Rikard Grankvist, Ida Friberger, Agneta Andersson, Mattias Carlsten, Kenneth Chien, Johan Lundberg, Nevin Witman, Staffan Holmin
Heart failure has a poor prognosis and no curative treatment exists. Clinical trials are investigating gene- and cell-based therapies to improve cardiac function. The safe and efficient delivery of these therapies to solid organs is challenging. Herein, we demonstrate the feasibility of using an endovascular intramyocardial delivery approach to safely administer mRNA drug products and perform cell
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Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-27 Isabella Elias Yonezawa Ogusuku, Vera Herbel, Simon Lennartz, Caroline Brandes, Eva Argiro, Caroline Fabian, Carola Hauck, Conny Hoogstraten, Sabrina Veld, Lois Hageman, Karin Teppert, Georgia Koutsoumpli, Marieke Griffioen, Nadine Mockel-Tenbrinck, Thomas Schaser, Rosa de Groot, Ian C.D. Johnston, Dominik Lock
Acute myeloid leukemia (AML) is a heterogeneous malignancy that requires further therapeutic improvement, especially for the elderly and for subgroups with poor prognosis. A recently discovered T cell receptor (TCR) targeting mutant nucleophosmin 1 (ΔNPM1) presents an attractive option for the development of a cancer antigen-targeted cellular therapy. Manufacturing of TCR-modified T cells, however
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Genome editing in clinical practice: A model study for next-gen hematopoietic cell transplants in hematologic malignancies Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-26 Patrick Derigs, Carsten Müller-Tidow
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B cell focused transient immune suppression protocol for efficient AAV readministration to the liver Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-20 Jyoti Rana, Roland W. Herzog, Maite Muñoz-Melero, Kentaro Yamada, Sandeep R.P. Kumar, Anh K. Lam, David M. Markusic, Dongsheng Duan, Cox Terhorst, Barry J. Byrne, Manuela Corti, Moanaro Biswas
Adeno-associated virus (AAV) vectors are used for correcting multiple genetic disorders. Although the goal is to achieve lifelong correction with a single vector administration, the ability to redose would enable the extension of therapy in cases in which initial gene transfer is insufficient to achieve a lasting cure, episomal vector forms are lost in growing organs of pediatric patients, or transgene
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Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-20 Manuela Braun, Claudia Lange, Philipp Schatz, Brian Long, Johannes Stanta, Boris Gorovits, Edit Tarcsa, Vibha Jawa, Tong-Yuan Yang, Wibke Lembke, Nicole Miller, Fraser McBlane, Louis Christodoulou, Daisy Yuill, Mark Milton
Recombinant adeno-associated virus (AAV) vectors are the leading delivery vehicle used for gene therapies. Anti-AAV antibodies (AAV Abs) can interact with the viral capsid component of an AAV-based gene therapy (GT). Therefore, patients with preexisting AAV Abs (seropositive patients) are often excluded from GT trials to prevent treatment of patients who are unlikely to benefit or may have a higher
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Glutaredoxin-1 modulates the NF-κB signaling pathway to activate inducible nitric oxide synthase in experimental necrotizing enterocolitis Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-19 Yunfei Zhang, Mei Yan, Yingying Xia, Yingbin Yue, Shuli Wang, Yuhui Hu, Genjian Lai, Quanjiang Wu, Qianyang Liu, Xin Ding, Chunbao Guo
Inducible nitric oxide synthase (iNOS), regulated by nuclear factor kappa B (NF-κB), is crucial for intestinal inflammation and barrier injury in the progression of necrotizing enterocolitis (NEC). The NF-κB pathway is inhibited by S-glutathionylation of inhibitory κB kinase β (IKKβ), which can be restored by glutaredoxin-1 (Grx1). Thus, we aim to explore the role of Grx1 in experimental NEC. Wild-type
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Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-19 Junping Zhang, Xiangping Yu, Matthew Chrzanowski, Jiahe Tian, Derek Pouchnik, Ping Guo, Roland W. Herzog, Weidong Xiao
The unique palindromic inverted terminal repeats (ITRs) and single-stranded nature of adeno-associated virus (AAV) DNA are major hurdles to current sequencing technologies. Due to these characteristics, sequencing noncanonical AAV genomes present in AAV vector preparations remains challenging. To address this limitation, we developed thorough molecule configuration analysis of noncanonical AAV genomes
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Rationally engineered novel AAV capsids for intra-articular gene delivery Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-14 Wenjun Li, Susi Liu Feng, Lizette Herrschaft, R. Jude Samulski, Chengwen Li
Intra-articular adeno-associated virus (AAV) gene therapy has been explored as a potential strategy for joint diseases. However, concerns of low transduction efficacy, off-target expression, and neutralizing antibodies (Nabs) still need to be addressed. In this study, we demonstrated that AAV6 was the best serotype to transduce joints after screening serotypes 1 to 9. To develop a more effective AAV
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Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-13 Christina Amatya, Katherine A. Weissler, Vicki Fellowes, Norris Lam, Lauren C. Cutmore, Danielle A. Natrakul, Steven L. Highfill, James N. Kochenderfer
T cells expressing anti-CD19 chimeric antigen receptors (CARs) have activity against chronic lymphocytic leukemia (CLL), but complete response rates range from 18% to 29%, so improvement is needed. Peripheral blood mononuclear cells (PBMCs) of CLL patients often contain high levels of CLL cells that can interfere with CAR T cell production, and T cells from CLL patients are prone to exhaustion and
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First use of adeno-associated viruses in the human inner ear Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-10 Lukas D. Landegger
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A gene silencing-based approach to tackle fatty liver disease Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-09 Pavel Strnad, Christina Schrader, Nicola Brunetti-Pierri
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Continuous manufacturing of lentiviral vectors using a stable producer cell line in a fixed-bed bioreactor Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-09 Dale J. Stibbs, Pedro Silva Couto, Yasuhiro Takeuchi, Qasim A. Rafiq, Nigel B. Jackson, Andrea C.M.E. Rayat
Continuous manufacturing of lentiviral vectors (LVs) using stable producer cell lines could extend production periods, improve batch-to-batch reproducibility, and eliminate costly plasmid DNA and transfection reagents. A continuous process was established by expanding cells constitutively expressing third-generation LVs in the iCELLis Nano fixed-bed bioreactor. Fixed-bed bioreactors provide scalable
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Navigating toward gene therapy in Marfan syndrome: A hope for halting aortic aneurysm Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-08 Gustavo Egea
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Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-06 Melissa Pille, John M. Avila, So Hyun Park, Cuong Q. Le, Haipeng Xue, Filomeen Haerynck, Lavanya Saxena, Ciaran Lee, Elizabeth J. Shpall, Gang Bao, Bart Vandekerckhove, Brian R. Davis
Wiskott-Aldrich syndrome (WAS) is a severe X-linked primary immunodeficiency resulting from a diversity of mutations distributed across all 12 exons of the gene. encodes a hematopoietic-specific and developmentally regulated cytoplasmic protein (WASp). The objective of this study was to develop a gene correction strategy potentially applicable to most WAS patients by employing nuclease-mediated, site-specific
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Extracellular vesicles ameliorates sleep deprivation induced anxiety-like behavior and cognitive impairment in mice Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-05 Yiqin Lin, Zhenming Kang, Changsheng Su, Shunyuan Li, Wenqin Xie
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A novel FOXP3 knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-05 Yohei Sato, Abinaya Nathan, Suzette Shipp, John Fraser Wright, Keri Marie Tate, Prachi Wani, Maria-Grazia Roncarolo, Rosa Bacchetta
Abstract not available
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A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-03 Junping Zhang, Dylan A. Frabutt, Matthew Chrzanowski, Ning Li, Lohra M. Miller, Jiahe Tian, Patrick L. Mulcrone, Anh K. Lam, Benjamin E. Draper, Martin F. Jarrold, Roland W. Herzog, Weidong Xiao
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All-in-one IQ toggle switches with high versatilities for fine-tuning of transgene expression in mammalian cells and tissues Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-02 Jeongkwan Hong, Kyung-Cheol Sohn, Hye-Won Park, Hyoeun Jeon, Eunjin Ju, Jae-Geun Lee, Jeong-Soo Lee, Jaerang Rho, Gang Min Hur, Hyunju Ro
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Gesicles packaging dCas9-VPR ribonucleoprotein complexes can combine with vorinostat and promote HIV proviral transcription Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-02 Michaela A. Fisher, Waj Chaudhry, Lee A. Campbell
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Large-scale generation of IL-12 secreting macrophages from human pluripotent stem cells for cancer therapy Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-02 Baoqiang Kang, Qi Xing, Yuhua Huang, Huaisong Lin, Jiaojiao Peng, Zhishuai Zhang, Mingquan Wang, Xinrui Guo, Xing Hu, Shuoting Wang, Junwei Wang, Minghui Gao, Yanling Zhu, Guangjin Pan
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AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-02-02 Bhavya S. Doshi, Benjamin J. Samelson-Jones, Timothy C. Nichols, Elizabeth P. Merricks, Joshua L. Siner, Robert A. French, Ben J. Lee, Valder R. Arruda, Mary Beth Callan
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Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-30 Miles C. Smith, Lalitha R. Belur, Andrea D. Karlen, Olivia Erlanson, Justin Furcich, Troy C. Lund, Davis Seelig, Kelley F. Kitto, Carolyn A. Fairbanks, Kwi Hye Kim, Nick Buss, R. Scott McIvor
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Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-30 Meghan Blackwood, Alisha M. Gruntman, Qiushi Tang, Debora Pires-Ferreira, Darcy Reil, Oleksandr Kondratov, Damien Marsic, Sergei Zolotukhin, Gwladys Gernoux, Allison M. Keeler, Christian Mueller, Terence R. Flotte
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Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74 Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-23 Rachael A. Potter, Ellyn L. Peterson, Danielle Griffin, Grace Cooper Olson, Sarah Lewis, Kyle Cochran, Jerry R. Mendell, Louise R. Rodino-Klapac
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Expanded specific T cells to hypomutated regions of the SARS-CoV-2 using mRNA electroporated antigen-presenting cells Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-22 Elizabeth Ogando-Rivas, Paul Castillo, Changlin Yang, Vrunda Trivedi, Dingpeng Zhang, Fernanda Pohl-Guimarães, Ruixuan Liu, Arnav Barpujari, Kate M. Candelario, Hector Mendez-Gomez, Elias J. Sayour, Duane A. Mitchell
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AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-22 Joshua C. Chang, Molly R. Ryan, Marie C. Stark, Su Liu, Pravinkumar Purushothaman, Fria Bolan, Caitlin A. Johnson, Mark Champe, Hui Meng, Michael W. Lawlor, Sarah Halawani, Lucie V. Ngaba, David R. Lynch, Crystal Davis, Elena Gonzalo-Gil, Cathleen Lutz, Fabrizia Urbinati, Bala Medicherla, Carlos Fonck
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Liter-scale manufacturing of shelf-stable plasmid DNA/PEI transfection particles for viral vector production Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-22 Yizong Hu, Brendan A. Eder, Jinghan Lin, Sixuan Li, Yining Zhu, Tza-Huei Wang, Ting Guo, Hai-Quan Mao
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Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-17 Bijina Balakrishnan, Xinhua Yan, Marshall D. McCue, Olivia Bellagamba, Aaron Guo, Felicity Winkler, Jason Thall, Lisa Crawford, Rain Dimen, Sara Chen, Sean McEnaney, Yiman Wu, Mike Zimmer, Joe Sarkis, Paolo G.V. Martini, Patrick F. Finn, Kent Lai
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Recombinant AAV genome size effect on viral vector production, purification, and thermostability Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-17 Nermin Ibreljic, Benjamin E. Draper, Carl W. Lawton
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Extracellular vesicle depletion and UGCG overexpression mitigate the cell density effect in HEK293 cell culture transfection Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-17 Pol Pérez-Rubio, Jesús Lavado-García, Laia Bosch-Molist, Elianet Lorenzo Romero, Laura Cervera, Francesc Gòdia
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Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-17 Mathieu Mével, Virginie Pichard, Mohammed Bouzelha, Dimitri Alvarez-Dorta, Pierre-Alban Lalys, Nathalie Provost, Marine Allais, Alexandra Mendes, Elodie Landagaray, Jean-Baptiste Ducloyer, Estelle Toublanc, Anne Galy, Nicole Brument, Gaëlle M. Lefevre, Sébastien G. Gouin, Carolina Isiegas, Guylène Le Meur, Thérèse Cronin, Caroline Le Guiner, Michel Weber, Philippe Moullier, Eduard Ayuso, David Deniaud
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High-titer manufacturing of SARS-CoV-2 Spike-pseudotyped VSV in stirred-tank bioreactors Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-17 Hayley M. Todesco, Chris Gafuik, Cini M. John, Erin L. Roberts, Breanna S. Borys, Alexis Pawluk, Michael S. Kallos, Kyle G. Potts, Douglas J. Mahoney
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New perspectives for gene therapy of the X-linked form of Charcot-Marie-Tooth disease Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-12 Rafael Balada Caballé, Mario Bortolozzi
Abstract not available
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Long-term stability of clinical-grade lentiviral vectors for cell therapy Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-10 Julie K. Jadlowsky, Rachel Leskowitz, Stephen McKenna, Jayashree Karar, Yujie Ma, Anlan Dai, Gabriela Plesa, Fang Chen, Kathleen Alexander, Jennifer Petrella, Nan Gong, Wei-Ting Hwang, Olivia Farrelly, Julie Barber-Rotenberg, Shannon Christensen, Vanessa E. Gonzalez, Anne Chew, Joseph A. Fraietta, Carl H. June
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Improving cell-specific recombination using AAV vectors in the murine CNS by capsid and expression cassette optimization Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-10 Hayato Kawabata, Ayumu Konno, Yasunori Matsuzaki, Yumika Sato, Mika Kawachi, Ryo Aoki, Saki Tsutsumi, Shota Togai, Ryosuke Kobayashi, Takuro Horii, Izuho Hatada, Hirokazu Hirai
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Can mitochondria brown the lower-limb adipocytes? Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-05 Ilias P. Doulamis, Aspasia Tzani
Abstract not available
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Genetic surgery for a cystic fibrosis-causing splicing mutation Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-02 Mattijs Bulcaen, Marianne S. Carlon
Abstract not available
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TAZing down metabolic mayhem: siRNAs against liver inflammation and fibrosis in humanized mice Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-01 Julian Weihs, Milad Rezvani
Abstract not available
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Pulmonary macrophage transplant therapy in parenchymal lung diseases Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2024-01-01 Marissa O’Callaghan, Cormac McCarthy
Abstract not available
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Nonviral delivery of nCas9 for “safe harbor” integration to treat MPS IVA Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2023-12-29 Li Ou
Abstract not available
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Efficient and sustained FOXP3 locus editing in hematopoietic stem cells as a therapeutic approach for IPEX syndrome Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2023-12-26 Swati Singh, Cole M. Pugliano, Yuchi Honaker, Aidan Laird, M. Quinn DeGottardi, Ezra Lopez, Stefan Lachkar, Claire Stoffers, Karen Sommer, Iram F. Khan, David J. Rawlings
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Perspectives of the Friedreich ataxia community on gene therapy clinical trials Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2023-12-18 Shandra J. Trantham, Mackenzi A. Coker, Samantha Norman, Emma Crowley, Julie Berthy, Barry J. Byrne, Sub Subramony, XiangYang Lou, Manuela Corti
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AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2023-12-14 Yoshiko Murakami, Saori Umeshita, Kae Imanishi, Yoshichika Yoshioka, Akinori Ninomiya, Takehiko Sunabori, Shibi Likhite, Masato Koike, Kathrin C. Meyer, Taroh Kinoshita
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Manufacture of CD22 CAR T cells following positive versus negative selection results in distinct cytokine secretion profiles and γδ T cell output Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2023-12-11 Hannah W. Song, Mehdi Benzaoui, Alka Dwivedi, Sarah Underwood, Lipei Shao, Sooraj Achar, Vesna Posarac, Victoria A. Remley, Michaela Prochazkova, Yihua Cai, Ping Jin, Robert P. Somerville, David F. Stroncek, Grégoire Altan-Bonnet, Nirali N. Shah, Christopher D. Chien, Naomi Taylor, Steven L. Highfill
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The comparability tales: A phase-appropriate roadmap for CGT drug product development Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2023-12-05 American Society of Gene and Cell Therapy (ASGCT) CMC Committee Members, Nathalie Clément, Sadik H. Kassim, Daniel Leblanc, Katy Spink, John Tomtishen
Cell and gene therapies (CGTs) have revolutionized patient outcomes and provided care options for previously untreatable conditions. The clinical and commercial progress of CGT therapies is hindered by chemistry, manufacturing, and control (CMC) challenges. This article summarizes recommendations from the 2023 Annual Meeting CMC sessions wherein speakers advocated for science-driven comparability strategies
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Lipid nanoparticle-encapsulated DNA vaccine robustly induce superior immune responses to the mRNA vaccine in Syrian hamsters Mol. Ther. Methods Clin. Dev. (IF 4.7) Pub Date : 2023-12-05 Hung-Chun Liao, Kuan-Yin Shen, Chung-Hsiang Yang, Fang-Feng Chiu, Chen-Yi Chiang, Kit Man Chai, Wan-Chun Huang, Hui-Min Ho, Yi-Hua Chen, Min-Syuan Huang, Ching-Len Liao, Hsin-Wei Chen, Ming-Hsi Huang, Shih-Jen Liu