Outcomes – Ardat

Title:

AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain

Authors:

Helena Costa-Verdera, Vasco Meneghini, Zachary Fitzpatrick, Monah Abou Alezz, Emily Fabyanic, Xin Huang, Yulia Dzhashiashvili, Avantika Ahiya, Elisabeth Mangiameli, Erika Valeri, Giovanni Crivicich, Silvia Piccolo, Ivan Cuccovillo, Roberta Caccia, Ying Kai Chan, Bérangère Bertin, Giuseppe Ronzitti, Esteban A. Engel, Ivan Merelli, Federico Mingozzi, Angela Gritti, Klaudia Kuranda, Anna Kajaste-Rudnitski

Published by:

Nature Communications

Published online:

18 April 2025

Title:

Liver fibrosis negatively impacts in vivo gene transfer to murine hepatocytes

Authors:

Chiara Simoni, Justine Nozi, Francesco Starinieri, Tiziana La Bella, Elisabetta Manta, Camilla Negri, Mauro Biffi, Rossana Norata, Martina Rocchi, Francesca Sanvito, Giuseppe Ronzitti, Elena Barbon, Alessio Cantore

Published by:

Nature Communications

Published online:

10 March 2025

Title:

A systematic screening assay identifies efficient small guide RNAs for CRISPR activation

Authors:

Elin Arvidsson, Diana Duarte Lobo, Ermelinda Sabarese, Fabio Duarte, Rui Jorge Nobre, Luis Quintino, Cecilia Lundberg

Published by:

Frontiers in Bioengineering and Biotechnology

Published online:

23 January 2025

Title:

Polyfunctional T cells and unique cytokine clusters imprint the anti rAAV2/rAAV9 vector immune response

Authors:

Stephan J. Holtkamp, Florian R. Lagoda, Adam Lister, Pradeep Harish, Ulrike Kleymann, Theresa Pesch, Chai Fen Soon, Munir Pirmohamed, Dean Naisbitt, Mark Trautwein

Published by:

Frontiers in Immunology

Published online:

25 November 2024

Title:

Polyfunctional T cells and unique cytokine clusters imprint the anti rAAV2/rAAV9 vector immune response Frontiers in Immunology

Authors:

Stephan Jonas Holtkamp, Florian R. Lagoda, Adam Lister, Pradeep Harish, Ulrike Kleymann, Theresa Pesch, Chai Fen, Munir Pirmohamed, Dean Naisbitt, Mark Trautwein

Published by:

Frontiers in Immunology

Published online:

4 November 2024

Title:

Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47

Authors:

Jessica P Wiseman, Joseph M Scarrott, João Alves-Cruzeiro, Afshin Saffari, Cedric Böger, Evangelia Karyka, Emily Dawes, Alexandra K Davies, Paolo M Marchi, Emily Graves, Fiona Fernandes, Zih-Liang Yang Ian Coldicott, Jennifer Hirst, Christopher P Webster, J Robin Highley, Neil Hackett, Adrienn Angyal Thushan de Silva, Adrian Higginbottom, Pamela J Shaw, Laura Ferraiuolo Darius Ebrahimi-Fakhari, Mimoun Azzouz

Published by:

EMBO Mol Med

Published online:

2 October 2024

Title:

A microvascularized in vitro liver model for disease modeling and drug discovery

Authors:

Flavio Bonanini, Roelof Dinkelberg, Manuel Caro Torregrosa, Nienke Kortekaas, Tessa M S Hagens1, Stéphane Treillard, Dorota Kurek, Vincent van Duinen, Paul Vulto and Kristin Bircsak

Published by:

IOP Publishing Ltd

Published online:

24 October 2024

Title:

A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders

Authors:

Besarte Vrellaku1, Ilda Sethw Hassan2, Rebecca Howitt3, Christopher P. Webster Eli Harriss Fraser McBlane Corinne Betts Jorge Schettini Mattia Lion John E. Mindur Michael Duerr Pamela J. Shaw Janine Kirby Mimoun Azzouz2, Laurent Servais

Published by:

Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.

Published online:

July 21, 2024

Title:

The IgG-degrading enzyme, Imlifidase, restores the therapeutic activity of FVIII in inhibitor-positive hemophilia A mice

Authors:

Melissa Bou-Jaoudeh Sandrine Delignat Victoria Daventure Jan Astermark Hervé Lévesque Jordan D. Dimitrov Claire Deligne Valérie Proulle Sébastien Lacroix-Desmazes

Published by:

Ferrata Storti Foundation, Pavia, Italy

Pubmed

36655430

Print ISSN

0390-6078

Online ISSN

1592-8721

Title:

Imlifidase, a new option to optimize the management of patients with hemophilia A on emicizumab

Authors:

Melissa Bou-Jaoudeh Angelina Mimoun Sandrine Delignat Ivan Peyron Ladislas Capdevila Victoria Daventure Claire Deligne Jordan D. Dimitrov Olivier D. Christophe Cécile V. Denis Peter J. Lenting Valérie Proulle Sébastien Lacroix-Desmazes

Published by:

Ferrata Storti Foundation, Pavia, Italy

Published online:

July 17, 2023

Manuscript handled by:

David Lillicrap