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Items: 1 to 20 of 1630

1.
Immune-driven gene expression loss following intramuscular AAV delivery to non-human primates is only transient
Malo Journou, Marie Devaux, Nicolas Jaulin, Virginie Pichard, Mercedes Segovia, Aurélie Moreau, Johanne Le Duff, Maria Cristina Cuturi, Mickaël Guilbaud, Oumeya Adjali
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101409. Published online 2025 Jan 19. doi: 10.1016/j.omtm.2025.101409
PMCID:
PMC11914520
ArticlePDF–4.4M
2.
Cathepsin B inhibition blocks amyloidogenesis in the mouse models of neurological lysosomal diseases MPS IIIC and sialidosis
Gustavo M. Viana, Xuefang Pan, Shuxian Fan, TianMeng Xu, Alexandra Wyatt, Alexey V. Pshezhetsky
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101432. Published online 2025 Feb 11. doi: 10.1016/j.omtm.2025.101432
PMCID:
PMC11910108
ArticlePDF–7.8M
3.
Nonclinical study of ixo-vec gene therapy for nAMD supports efficacy for a human dose of 6E10 vg/eye and staggered dosing of fellow eyes
Kris Poulsen, Kelly Hanna, Julio Nieves, Ngoc Nguyen, Pallavi Sharma, Ruslan Grishanin, Romu Corbau, Szilárd Kiss
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101430. Published online 2025 Feb 10. doi: 10.1016/j.omtm.2025.101430
PMCID:
PMC11910100
ArticlePDF–4.0M
4.
Cell-penetrating peptide-grafted AAV2 capsids for improved retinal delivery via intravitreal injection
Jiang-Hui Wang, Mengtian Cui, Hao Liu, Peiyi Guo, Jackson McGowan, Shun-Yun Cheng, Dominic J. Gessler, Jun Xie, Claudio Punzo, Phillip W.L. Tai, Guangping Gao
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101426. Published online 2025 Feb 3. doi: 10.1016/j.omtm.2025.101426
PMCID:
PMC11872077
ArticlePDF–3.6M
5.
Modeling Pkd1 gene-targeted strategies for correction of polycystic kidney disease
Almira Kurbegovic, Rey Christian Pacis, Marie Trudel
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101422. Published online 2025 Jan 28. doi: 10.1016/j.omtm.2025.101422
Expression of Concern about:
Mol Ther Methods Clin Dev. 2023 Jun 8; 29: 366–380.
PMCID:
PMC11815702
ArticlePDF–165K
6.
A novel platform for engineered AAV-based vaccines
Sabrina Babutzka, Miranda Gehrke, Anastasia Papadopoulou, Maria Diedrichs-Möhring, Maria Giannaki, Lena Hennis, Bastian Föhr, Cale Kooyman, Andreas Osterman, Evangelia Yannaki, Gerhild Wildner, Hermann Ammer, Stylianos Michalakis
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101418. Published online 2025 Jan 22. doi: 10.1016/j.omtm.2025.101418
PMCID:
PMC11850754
ArticlePDF–4.5M
7.
Human striatal progenitor cells that contain inducible safeguards and overexpress BDNF rescue Huntington’s disease phenotypes
Danielle A. Simmons, Sridhar Selvaraj, Tingshuo Chen, Gloria Cao, Talita Souto Camelo, Tyne L.M. McHugh, Selena Gonzalez, Renata M. Martin, Juste Simanauskaite, Nobuko Uchida, Matthew H. Porteus, Frank M. Longo
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101415. Published online 2025 Jan 22. doi: 10.1016/j.omtm.2025.101415
PMCID:
PMC11848452
ArticlePDF–7.5M
8.
Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot study
Huiping Li, Shu Liu, Caimei Lin, Yuchao Wu, Xiuping Wu, Yukun Huang, Yajun Wu, Xiubin Tong, Xiu Xu
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101413. Published online 2025 Jan 21. doi: 10.1016/j.omtm.2025.101413
PMCID:
PMC11847261
ArticlePDF–2.0M
9.
Probing aspects of extracellular vesicle associated AAV allows increased vector yield and insight into its transduction and immune-evasive properties
Ming Cheng, Demitri de la Cruz, Adam V. Crain, Paula Espinoza, Carrie Ng, Zachary C. Elmore, Aravind Asokan, Casey A. Maguire
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101407. Published online 2025 Jan 17. doi: 10.1016/j.omtm.2025.101407
PMCID:
PMC11851286
ArticlePDF–3.7M
10.
Polo-like kinase inhibitors increase AAV production by halting cell cycle progression
Kaylin Fisher, Francis Grafton, Francesca Ispaso, Joshua Tworig, Rupert Derler, Florian Sonntag, Markus Hörer, Andreas Schulze, Christopher A. Reid, Mohammad A. Mandegar
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101412. Published online 2025 Jan 17. doi: 10.1016/j.omtm.2025.101412
PMCID:
PMC11834096
ArticlePDF–4.0M
11.
Ensuring patient access to gene therapies for rare diseases: Navigating reimbursement and coverage challenges
Diane Berry, Carolyn Hickey, Lisa Kahlman, James Long, Christina Markus, Caitlin K. McCombs
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101403. Published online 2025 Jan 17. doi: 10.1016/j.omtm.2024.101403
PMCID:
PMC11786838
ArticlePDF–233K
12.
Reducing off-target expression of mRNA therapeutics and vaccines in the liver with microRNA binding sites
Brian J. Parrett, Satoko Yamaoka, Michael A. Barry
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101402. Published online 2024 Dec 19. doi: 10.1016/j.omtm.2024.101402
PMCID:
PMC11758401
ArticlePDF–3.0M
13.
Characterization and effective expansion of CD4−CD8− TCRαβ+ T cells from individuals living with type 1 diabetes
J. Ernesto Fajardo-Despaigne, Félix Lombard-Vadnais, Adam-Nicolas Pelletier, Aïnhoa Olazabal, Lucie Boutin, Sarah Pasquin, Valérie Janelle, Laurent Legault, Jean-Sébastien Delisle, Erin E. Hillhouse, Lise Coderre, Sylvie Lesage
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101400. Published online 2024 Dec 17. doi: 10.1016/j.omtm.2024.101400
PMCID:
PMC11772147
ArticlePDF–4.3M
14.
Lipid nanoparticle encapsulation of a Delta spike-CD40L DNA vaccine improves effectiveness against Omicron challenge in Syrian hamsters
Levi Tamming, Diana Duque, Anh Tran, Casey Lansdell, Grant Frahm, Jianguo Wu, Emily E.F. Fekete, Marybeth Creskey, Sathya N. Thulasi Raman, Emmanuel Laryea, Wanyue Zhang, Annabelle Pfeifle, Caroline Gravel, Andrew Stalker, Anwar M. Hashem, Wangxue Chen, Matthew Stuible, Yves Durocher, David Safronetz, Jingxin Cao, Lisheng Wang, Simon Sauve, Michael Rosu-Myles, Xu Zhang, Michael J.W. Johnston, Xuguang Li
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101388. Published online 2024 Dec 11. doi: 10.1016/j.omtm.2024.101388
Corrects:
Mol Ther Methods Clin Dev. 2024 Sep 12; 32(3): 101325.
PMCID:
PMC11699230
CorrectionPDF–170K
15.
Silencing hepatic PCSK9 via novel chimeric AAV8 mitigates the progression of atherosclerosis by inhibiting inflammation in ApoE−/− mice
Xiaocui Chen, Minghui Sun, Xiang Ma, Yitong Ma, Bangdang Chen
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101390. Published online 2024 Dec 5. doi: 10.1016/j.omtm.2024.101390
PMCID:
PMC11787523
ArticlePDF–4.1M
16.
Increased functional potency of multi-edited CAR-T cells manufactured by a non-viral transfection system
Aine O’Sullivan, Sarah Case, Aisling McCrudden, Emer Hackett, Louise Gallagher, Darren Martin, Gillian P. Johnson, Kirti Mahadik, Thomas Kienzle, Jude Kevin Lim, Aya Nashat, Kartik Srinivasan, Mark W. Lowdell, Lisa O’Flynn, Jamie Frankish
Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1): 101389. Published online 2024 Dec 5. doi: 10.1016/j.omtm.2024.101389
PMCID:
PMC11730244
ArticlePDF–4.2M
17.
Size-exclusion chromatography as a multi-attribute method for process and product characterization of adeno-associated virus
Sri Hari Raju Mulagapati, Arun Parupudi, Tomasz Witkos, Nick Bond, Xiaoyu Chen, Thomas Linke, Guoling Xi, Albert Ethan Schmelzer, Wei Xu
Mol Ther Methods Clin Dev. 2024 Dec 12; 32(4): 101382. Published online 2024 Nov 19. doi: 10.1016/j.omtm.2024.101382
PMCID:
PMC11647602
ArticlePDF–3.0M
18.
Quantitative proteomic analysis of residual host cell protein retention across adeno-associated virus affinity chromatography
Thomas M. Leibiger, Lie Min, Kelvin H. Lee
Mol Ther Methods Clin Dev. 2024 Dec 12; 32(4): 101383. Published online 2024 Nov 18. doi: 10.1016/j.omtm.2024.101383
PMCID:
PMC11650319
ArticlePDF–3.0M
19.
High-affinity chimeric antigen receptor signaling induces an inflammatory program in human regulatory T cells
Russell W. Cochrane, Rob A. Robino, Bryan Granger, Eva Allen, Silvia Vaena, Martin J. Romeo, Aguirre A. de Cubas, Stefano Berto, Leonardo M.R. Ferreira
Mol Ther Methods Clin Dev. 2024 Dec 12; 32(4): 101385. Published online 2024 Nov 18. doi: 10.1016/j.omtm.2024.101385
PMCID:
PMC11647616
ArticlePDF–4.0M
20.
mRNA vaccines encoding membrane-anchored RBDs of SARS-CoV-2 mutants induce strong humoral responses and can overcome immune imprinting
Hareth A. Al-Wassiti, Stewart A. Fabb, Samantha L. Grimley, Ruby Kochappan, Joan K. Ho, Chinn Yi Wong, Chee Wah Tan, Thomas J. Payne, Asuka Takanashi, Chee Leng Lee, Rekha Shandre Mugan, Horatio Sicilia, Serena L.Y. Teo, Julie McAuley, Paula Ellenberg, James P. Cooney, Kathryn C. Davidson, Richard Bowen, Marc Pellegrini, Steven Rockman, Dale I. Godfrey, Terry M. Nolan, Lin-fa Wang, Georgia Deliyannis, Damian F.J. Purcell, Colin W. Pouton
Mol Ther Methods Clin Dev. 2024 Dec 12; 32(4): 101380. Published online 2024 Nov 15. doi: 10.1016/j.omtm.2024.101380
PMCID:
PMC11646785
ArticlePDF–5.1M

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