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Showing results for ISSN matching 2329-0501

Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell single-cell cloning

JOURNAL ARTICLE published June 2023 in Molecular Therapy - Methods & Clinical Development

Authors: Sophie Broussau | Viktoria Lytvyn | Mélanie Simoneau | Claire Guilbault | Mélanie Leclerc | Nazila Nazemi-Moghaddam | Nathalie Coulombe | Seyyed Mehdy Elahi | Scott McComb | Rénald Gilbert

https://doi.org/10.1016/j.omtm.2023.02.013 Actions

Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment

JOURNAL ARTICLE published September 2021 in Molecular Therapy - Methods & Clinical Development

Authors: Susana Navarro | Oscar Quintana-Bustamante | Rebeca Sanchez-Dominguez | Sergio Lopez-Manzaneda | Isabel Ojeda-Perez | Aida Garcia-Torralba | Omaira Alberquilla | Kenneth Law | Brian C. Beard | Antonella Bastone | Michael Rothe | Mariela Villanueva | Juan C. Ramirez | Sara Fañanas-Baquero | Virginia Nieto-Romero | Andrea Molinos-Vicente | Sonia Gutierrez | Eileen Nicoletti | María García-Bravo | Juan A. Bueren | Jonathan D. Schwartz | Jose-Carlos Segovia

https://doi.org/10.1016/j.omtm.2021.07.006 Actions

Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing

JOURNAL ARTICLE published June 2020 in Molecular Therapy - Methods & Clinical Development

Research funded by Canadian Institutes of Health Research (MOP-125855,CFC#3172) | Cystic Fibrosis Canada (MOP-125855,CFC#3172) | Hospital for Sick Children (MOP-125855,CFC#3172)

Authors: Steven Erwood | Onofrio Laselva | Teija M.I. Bily | Reid A. Brewer | Alexandra H. Rutherford | Christine E. Bear | Evgueni A. Ivakine

https://doi.org/10.1016/j.omtm.2020.05.002 Actions

Rapid Lentiviral Vector Producer Cell Line Generation Using a Single DNA Construct

JOURNAL ARTICLE published December 2020 in Molecular Therapy - Methods & Clinical Development

Authors: Yu Hua Chen | Celeste Pallant | Christopher J. Sampson | Alessia Boiti | Sabine Johnson | Pijus Brazauskas | Philip Hardwicke | Michela Marongiu | Vanesa M. Marinova | Marlene Carmo | Nathan P. Sweeney | Ashkenaz Richard | Anthony Shillings | Peter Archibald | Eva Puschmann | Bernadette Mouzon | David Grose | Miriam Mendez-Tavio | Mao Xiang Chen | Stephen R.C. Warr | Tarik Senussi | Paul S. Carter | Sean Baker | Cindy Jung | Martijn H. Brugman | Steven J. Howe | Conrad A. Vink

https://doi.org/10.1016/j.omtm.2020.08.011 Actions

Targeted knockdown of the adenosine A2A receptor by lipid NPs rescues the chemotaxis of head and neck cancer memory T cells

JOURNAL ARTICLE published June 2021 in Molecular Therapy - Methods & Clinical Development

Authors: Hannah S. Newton | Ameet A. Chimote | Michael J. Arnold | Trisha M. Wise-Draper | Laura Conforti

https://doi.org/10.1016/j.omtm.2021.03.001 Actions

Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses

JOURNAL ARTICLE published December 2022 in Molecular Therapy - Methods & Clinical Development

Authors: Eduard H.T.M. Ebberink | Alisa Ruisinger | Markus Nuebel | Marco Thomann | Albert J.R. Heck

https://doi.org/10.1016/j.omtm.2022.11.003 Actions

AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency

JOURNAL ARTICLE published December 2022 in Molecular Therapy - Methods & Clinical Development

Authors: Lei Zhu | Brandon Tan | Selina S. Dwight | Brendan Beahm | Matt Wilsey | Brett E. Crawford | Becky Schweighardt | Jennifer W. Cook | Thomas Wechsler | William F. Mueller

https://doi.org/10.1016/j.omtm.2022.09.015 Actions

Expression of NMNAT1 in the photoreceptors is sufficient to prevent NMNAT1-associated retinal degeneration

JOURNAL ARTICLE published June 2023 in Molecular Therapy - Methods & Clinical Development

Research funded by National Eye Institute (EY012910,P30EY003790)

Authors: Emily E. Brown | Michael J. Scandura | Eric A. Pierce

https://doi.org/10.1016/j.omtm.2023.04.003 Actions

DNA-Loaded Cationic Liposomes Efficiently Function as a Vaccine against Malarial Proteins

JOURNAL ARTICLE published December 2017 in Molecular Therapy - Methods & Clinical Development

Research funded by FAPESP (2012/18617-1,2015/17174-7)

Authors: Wesley L. Fotoran | Rachele Santangelo | Beatriz N.M. de Miranda | Darrell J. Irvine | Gerhard Wunderlich

https://doi.org/10.1016/j.omtm.2017.08.004 Actions

Large-Scale Generation and Characterization of Homogeneous Populations of Migratory Cortical Interneurons from Human Pluripotent Stem Cells

JOURNAL ARTICLE published June 2019 in Molecular Therapy - Methods & Clinical Development

Research funded by New York Medical College/Westchester Medical Center Translational Stem Cell Center (MH107884,NYSTEM C32607GG)

Authors: Peiyan Ni | Haneul Noh | Zhicheng Shao | Qian Zhu | Youxin Guan | Joshua J. Park | Fatima Arif | James M. Park | Chiderah Abani | Cameron Beaudreault | Joy S. Park | Elizabeth Berry | Alexander Moghadam | Patric Stanton | John N. Hutchinson | Bill Andrews | Clare Faux | John Parnevelas | Leonard M. Eisenberg | Kyungjoon Park | Vadim Y. Bolshakov | Sangmi Chung

https://doi.org/10.1016/j.omtm.2019.04.002 Actions

Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering

JOURNAL ARTICLE published December 2023 in Molecular Therapy - Methods & Clinical Development

Research funded by Bundesministerium für Bildung und Forschung (16GW0322)

Authors: Reni Kitte | Martin Rabel | Reka Geczy | Stella Park | Stephan Fricke | Ulrike Koehl | U. Sandy Tretbar

https://doi.org/10.1016/j.omtm.2023.101139 Actions

Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard

JOURNAL ARTICLE published June 2023 in Molecular Therapy - Methods & Clinical Development

Research funded by National Institutes of Health (S10 OD018142-01)

Authors: Mario Mietzsch | Weijing Liu | Ke Ma | Antonette Bennett | Austin R. Nelson | Keely Gliwa | Paul Chipman | Xiaofeng Fu | Shane Bechler | Robert McKenna | Rosa Viner

https://doi.org/10.1016/j.omtm.2023.05.002 Actions

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

JOURNAL ARTICLE published September 2017 in Molecular Therapy - Methods & Clinical Development

Research funded by NIH (NS-90634,HL-122332) | Department of Defense (MD130014)

Authors: Chady H. Hakim | Nalinda B. Wasala | Xiufang Pan | Kasun Kodippili | Yongping Yue | Keqing Zhang | Gang Yao | Brittney Haffner | Sean X. Duan | Julian Ramos | Joel S. Schneider | N. Nora Yang | Jeffrey S. Chamberlain | Dongsheng Duan

https://doi.org/10.1016/j.omtm.2017.06.006 Actions

Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs

JOURNAL ARTICLE published June 2021 in Molecular Therapy - Methods & Clinical Development

Authors: Daniela Drago | Betsy Foss-Campbell | Keith Wonnacott | David Barrett | Adora Ndu

https://doi.org/10.1016/j.omtm.2021.04.001 Actions

STRIP2 Is Indispensable for the Onset of Embryonic Stem Cell Differentiation

JOURNAL ARTICLE published June 2017 in Molecular Therapy - Methods & Clinical Development

Research funded by German Research Foundation (SA 568/17-2)

Authors: Davood Sabour | Sureshkumar Perumal Srinivasan | Susan Rohani | Vilas Wagh | John Antonydas Gaspar | Darius Panek | Mostafa Abootorabi Ardestani | Michael Xavier Doss | Nicole Riet | Hinrich Abken | Jürgen Hescheler | Symeon Papadopoulos | Agapios Sachinidis

https://doi.org/10.1016/j.omtm.2017.04.001 Actions

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

JOURNAL ARTICLE published March 2019 in Molecular Therapy - Methods & Clinical Development

Research funded by European Union Research and Innovation Program (667751,658712) | European Research Council (617432)

Authors: Pasqualina Colella | Pauline Sellier | Helena Costa Verdera | Francesco Puzzo | Laetitia van Wittenberghe | Nicolas Guerchet | Nathalie Daniele | Bernard Gjata | Solenne Marmier | Severine Charles | Marcelo Simon Sola | Isabella Ragone | Christian Leborgne | Fanny Collaud | Federico Mingozzi

https://doi.org/10.1016/j.omtm.2018.11.002 Actions

Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice

JOURNAL ARTICLE published 2015 in Molecular Therapy - Methods & Clinical Development

Authors: Darin J Falk | Meghan S Soustek | Adrian Gary Todd | Cathryn S Mah | Denise A Cloutier | Jeffry S Kelley | Nathalie Clement | David D Fuller | Barry J Byrne

https://doi.org/10.1038/mtm.2015.7 Actions

Quantitative Hybrid Cardiac [18F]FDG-PET-MRI Images for Assessment of Cardiac Repair by Preconditioned Cardiosphere-Derived Cells

JOURNAL ARTICLE published September 2020 in Molecular Therapy - Methods & Clinical Development

Authors: Johannes Winkler | Dominika Lukovic | Julia Mester-Tonczar | Katrin Zlabinger | Alfred Gugerell | Noemi Pavo | András Jakab | Zsuzsanna Szankai | Denise Traxler | Claudia Müller | Andreas Spannbauer | Martin Riesenhuber | Ena Hašimbegović | James Dawkins | Matthias Zimmermann | Hendrik J. Ankersmit | Eduardo Marbán | Mariann Gyöngyösi

https://doi.org/10.1016/j.omtm.2020.06.008 Actions

Deferoxamine mesylate improves splicing and GAA activity of the common c.-32-13T>G allele in late-onset PD patient fibroblasts

JOURNAL ARTICLE published March 2021 in Molecular Therapy - Methods & Clinical Development

Research funded by Telethon (GGP14192)

Authors: Emanuele Buratti | Paolo Peruzzo | Luca Braga | Irene Zanin | Cristiana Stuani | Elisa Goina | Maurizio Romano | Mauro Giacca | Andrea Dardis

https://doi.org/10.1016/j.omtm.2020.11.011 Actions

Methods and clinical development of adenovirus-vectored vaccines against mucosal pathogens

JOURNAL ARTICLE published 2016 in Molecular Therapy - Methods & Clinical Development

Authors: Sam Afkhami | Yushi Yao | Zhou Xing

https://doi.org/10.1038/mtm.2016.30 Actions
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