CORDIS provides links to public deliverables and publications of HORIZON projects.
Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .
Deliverables
Behavioural outcomes of Dp140 restoration in mdx52 using direct AON brain injection or AAVU7.
Behaviour: Dp71 restoration (opens in new window)Behavioural outcome of Dp71 restoration using AAV in Dp71-null and mdx3cv.
Mdx intellectual defects (opens in new window)Motor emotional and learning deficits shared or unique to the different mouse models
Differences between mouse models (opens in new window)Executive dysfunctions shared or unique to the different mouse models
Transcriptional changes in dystrophin absence (opens in new window)Characterization of transcriptional changes related to the loss of specific dystrophin isoforms.
First study approvals package (opens in new window)First study subject approvals package (prior to enrolment of first study subject). (i) Final version of study protocol as submitted to regulators/ethics committee(s), (ii) Registration number of clinical study in a WHO-or ICMJE- approved registry (with the possibility to post results), (iii) Approvals (ethics committees and national competent authority if applicable) required for invitation/enrolment of first subject in at least one clinical centre.
Communication, Dissemination and Exploitation plan (first release) (opens in new window)First release of the Communication, Dissemination and Exploitation Plan.
Neurobehavioural definition (opens in new window)Definition of the neurobehavioural co-morbidities of the 180 DMD and 90 BMD patients belonging to the 3 different genotype categories.
Genotype/phenotype correlations in mice (opens in new window)Genotype/ phenotype correlation in two mdx mouse models
First study subject approvals package (opens in new window)First study subject approvals package' (prior to enrolment of first study subject). (i) Final version of study protocol as submitted to regulators/ethics committee(s), (ii) Registration number of clinical study in a WHO-or ICMJE- approved registry (with the possibility to post results), (iii) Approvals (ethics committees and national competent authority if applicable) required for invitation/enrolment of first subject in at least one clinical centre.
Questionnaire definition (opens in new window)Definition of a questionnaire that will be distributed via UPPMD to national advocacy groups and registries
Mdx5cv validation (opens in new window)Validation of the mdx5cv mouse model
Report on a status of posting results (opens in new window)Report on status of posting results in the study registry(s) – For each clinical study, a report on the status of posting results in the study registry(s) must be submitted as a deliverable, including timelines if/when final posting of results is scheduled after end of funding period.We will post the result of the brain MRI studies, which will include the brain structure, perfusion and structural connectivity in DMD and BMD patients by month 53.
Dystrophin protein complex characterisation (opens in new window)Characterisation of dystrophin protein complexes in different brain regions from normal mouse brains
Mouse robust phenotypes (opens in new window)Duplication of robust phenotypes selected for WP4 in the different mouse models.
Behaviour: Dp427 restoration (opens in new window)Behavioural outcome of Dp427 restoration using direct AON brain injection or AAV-U7 in the mdx52.
Gene and proteomic knowledge map (opens in new window)Knowledge map of gene and proteomic changes associated with comorbidities in other than DMDBMD
Genotype/phenotype correlations in DMD/BMD brains (opens in new window)Genotype / phenotype correlations for brain structure, perfusion, functional connectivity in DMD and BMD patients
Post-natal brain restoration therapy (opens in new window)Effect of post-natal dystrophin restoration therapy.
Comorbidity ‘supercluster' formation (opens in new window)Formation of brain comorbidity supercluster and clinical trial advisory group on brain comorbidities
Mouse comorbidities (opens in new window)Comorbidities shared or unique to the different mouse models
Brain transcriptional changes (opens in new window)Characterization of transcriptional changes in different brain regions of mouse models lacking different dystrophin isoforms
Behaviour: Dp427 or Dp127+Dp140 restoration (opens in new window)Behavioural outcome of Dp427 or Dp127+Dp140 restoration in mdx52 using systemically administered new generations of AONs (tcDNA ).
WP5 - Midterm recruitment report (opens in new window)Midterm recruitment report Report for each clinical study to be scheduled for the point when 50 of the study population is expected to have been recruited
Report on status of posting results (opens in new window)Report on status of posting results in the study registry(s) – For each clinical study, a report on the status of posting results in the study registry(s) must be submitted as a deliverable, including timelines if/when final posting of results is scheduled after end of funding period. The end of the clinical deep phenotyping including the neurobehavioural and neurocognitive profile of the DMD and BMD patients will be delivered at month 47.
Neurocognitive profile definition (opens in new window)Definition of the neurocognitive profile of the 180 DMD patients and 90 BMD patients belonging to the 3 genotype categories.
Overlapping features related to co-morbidities (opens in new window)List of the overlapping features related to the co-morbidities identified in both DMD/BMD and in other disorders.
Omics datasets (opens in new window)Generation of proteomics and transcriptomics datasets to be used in WP7 to identify molecular networks related to cognition and emotional responses that are associated with dystrophin.
Dystrophin isoform localisation: mice (opens in new window)Characterisation of dystrophin isoform localisation in different brain regions from normal mouse brains.
Questionnaire on comorbidities to families (opens in new window)Questionnaire to families to ascertain severity of comorbidities
Prediction outcomes for different therapies (opens in new window)Outcome prediction for different dystrophin therapies
Dystrophin isoform localisation:humans (opens in new window)Characterisation of dystrophin isoform localisation in different brain regions from normal human brains
Protocol optimisation (opens in new window)Optimization of the neurocognitive and neurobehavioural protocols to be used in the study
Communication, Dissemination and Exploitation plan (second release) (opens in new window)Second release of the Communication, Dissemination and Exploitation Plan.
Factors responsible for behavioural changes (opens in new window)List of factors responsible for the behavioural observation in mouse and man.
WP6 - Midterm recruitment report (opens in new window)Midterm recruitment report Report for each clinical study to be scheduled for the point when 50 of the study population is expected to have been recruited
Communication, Dissemination and Exploitation plan (final release) (opens in new window)Final release of the Communication, Dissemination and Exploitation Plan.
The project website will be formally delivered in month 4, carrying general information about BIND and the planned public deliverables; it will then be updated at regular intervals, to report on activities, events, major achievements and to provide access to public deliverables.
Publications
Author(s):
Mary Chesshyre, Deborah Ridout, Yasumasa Hashimoto, Yoko Ookubo, Silvia Torelli, Kate Maresh, Valeria Ricotti, Lianne Abbott, Vandana Ayyar Gupta, Marion Main, Giulia Ferrari, Anna Kowala, Yung-Yao Lin, Francesco Saverio Tedesco, Mariacristina Scoto, Giovanni Baranello, Adnan Manzur, Yoshitsugu Aoki, Francesco Muntoni
Published in:
Journal of Cachexia, Sarcopenia and Muscle, 2022, ISSN 2190-5991
Publisher:
Springer Verlag
DOI:
10.1002/jcsm.12914
Author(s):
A. Saoudi, S. Barberat, O. Le Coz, O. Vacca, M. Doisy Caquant, Tensorer, E. Sliwinski, L. Garcia, F. Muntoni, C. Vaillend and A. Goyenvalle
Published in:
Molecular Therapy Nucelic Acids, 2023, ISSN 2162-2531
Publisher:
Nature Publishing Group
DOI:
10.1016/j.omtn.2023.03.009
Author(s):
Rachel E. Trimmer, William P.L. Mandy, Francesco Muntoni, Kate E. Maresh
Published in:
Neuromuscular Disorders, 2024, ISSN 0960-8966
Publisher:
Elsevier BV
DOI:
10.1016/j.nmd.2023.12.002
Author(s):
F. Zarrouki, S. Goutal, O. Vacca, L. Garcia, N. Tournier, A. Goyenvalle, and C. Vaillend
Published in:
International Journal of Molecular Sciences, 2022, ISSN 1422-0067
Publisher:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/ijms232012617
Author(s):
Saba Gharibi, Cyrille Vaillend, Angus Lindsay
Published in:
Progress in Neurobiology, 2024, ISSN 0301-0082
Publisher:
Pergamon Press Ltd.
DOI:
10.1016/j.pneurobio.2024.102590
Author(s):
M. Van Putten, M. Verhaeg, L. van der Pijl, D. van de Vijver, C. Tanganyika-de Winter, T. Stan, A. Aartsma-Rus
Published in:
Neuromuscular Disorders, Issue 43, 2024, Page(s) 104441.492, ISSN 0960-8966
Publisher:
Elsevier BV
DOI:
10.1016/j.nmd.2024.07.501
Author(s):
Jos Hendriksen, Pien Weerkamp, Ruben Miranda, Anna Kolesnik, Daniela Chieffo, David Skuse, Elizabeth Vroom, Chloe Geagan, Francesco Muntoni, Eugenio Mercuri
Published in:
Neuromuscular Disorders, Issue 44, 2024, Page(s) 104452, ISSN 0960-8966
Publisher:
Elsevier BV
DOI:
10.1016/j.nmd.2024.104452
Author(s):
Francesco Muntoni and Mary Chessyre (UCL)
Published in:
Journal of Cachexia, Sarcopenia and Muscle, 2022, ISSN 2190-6009
Publisher:
Wiley
DOI:
10.1002/jcsm.12914
Author(s):
Cyrille Vaillend, A. Saoudi, F. Zarrouki, C. Sebrié, C. Izabelle, A. Goyenvalle, C. Vaillend
Published in:
Disease Models and Mechanisms, 2021, ISSN 1754-8411
Publisher:
The company of Biologists
DOI:
10.1242/dmm.049028
Author(s):
Ophélie Vacca, Faouzi Zarrouki, Charlotte Izabelle, Mehdi Belmaati Cherkaoui, Alvaro Rendon, Deniz Dalkara, Cyrille Vaillend
Published in:
Cells, 2024, ISSN 2073-4409
Publisher:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/cells13080718
Author(s):
P. Weerkamp, E. Mol, D. Sweere, D. Schrans, R. J. Vermeulen, S. Klinkenberg, P. Hurks and J. Hendriksen
Published in:
Brain Sciences, 2022, ISSN 2076-3425
Publisher:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/brainsci12111544
Author(s):
Kate Maresh,Andriani Papageorgiou,
Deborah Ridout,
Neil Harrison,
William Mandy,
David Skuse,
Francesco Muntoni
Published in:
Plos one, 2022, ISSN 1932-6203
Publisher:
Public Library of Science
DOI:
10.1371/journal.pone.0264091
Author(s):
Mathilde Doisy, Ophélie Vacca, Claire Fergus, Talia Gileadi, Minou Verhaeg, Amel Saoudi, Thomas Tensorer, Luis Garcia, Vincent P Kelly, Federica Montanaro, Jennifer E Morgan, Maaike van Putten, Annemieke Aartsma-Rus , Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle
Published in:
Biomedicines, 2024, ISSN 2227-9059
Publisher:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/biomedicines11123243
Author(s):
P. Weerkamp, P. Collin, R. Maas, R. J. Vermeulen, S. Klinkenberg, J. Hendriksen
Published in:
Neuromuscular Disorders, 2022, ISSN 1873-2364
Publisher:
Elsevier
DOI:
10.1016/j.nmd.2021.10.008
Author(s):
Pien Weerkamp, Daniela Chieffo, Philippe Collin, Federica Moriconi, Andriani Papageorgiou, Isabella Vainieri, Ruben Miranda, Catherine Hankinson, Asmus Vogel, Sarah Poncet, Catherine Moss, Francesco Muntoni, Eugenio Mercuri, Jos Hendriksen
Published in:
European Journal of Paediatric Neurology, 2023, ISSN 1090-3798
Publisher:
W. B. Saunders Co., Ltd.
DOI:
10.1016/j.ejpn.2023.06.007
Author(s):
Daniela P R Chieffo, Federica Moriconi, Marika Pane, Simona Lucibello, Elisabetta Ferraroli, Giulia Norcia, Martina Ricci, Anna Capasso, Gianpaolo Cicala, Bianca Buchignani , Giorgia Coratti, Costanza Cutrona, Monia Pelizzari, Claudia Brogna, Jos G M Hendriksen, Francesco Muntoni, Eugenio Mercuri
Published in:
Journal of Clinical Medicine, 2023, ISSN 2077-0383
Publisher:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/jcm12020403
Author(s):
A. Saoudi, C. Fergu, T. Gileadi, F. Montanaro, JE Morgan, V. Kelly, T. Tensorer, L. Garcia, C. Vaillend, F. Muntoni and A. Goyenvalle
Published in:
Cells, 2023, ISSN 2073-4409
Publisher:
MDPI
DOI:
10.3390/cells12060908
Author(s):
Rubén Miranda, Léa Ceschi, Delphine Le Verger, Flora Nagapin, Jean-Marc Edeline, Rémi Chaussenot, Cyrille Vaillend
Published in:
Behavioral and Brain Functions, Issue 20, 2024, Page(s) https://behavioralandbrainfunctions.biomedcentral.com/articles/10.1186/s12993-024-00246-x, ISSN 1744-9081
Publisher:
BioMed Central
DOI:
10.1186/s12993-024-00246-x
Author(s):
Mathilde Doisy, Ophélie Vacca, Claire Fergus, Talia Gileadi, Minou Verhaeg, Amel Saoudi, Thomas Tensorer, Luis Garcia, Vincent P Kelly, Federica Montanaro, Jennifer E Morgan, Maaike van Putten, Annemieke Aartsma-Rus , Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle
Published in:
Biomedicines, 2023, ISSN 2227-9059
Publisher:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/biomedicines11123243
Author(s):
Language Development in Preschool Duchenne Muscular Dystrophy Boys
Published in:
Brain Sciences, 2022, ISSN 2076-3425
Publisher:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/brainsci12091252
Author(s):
Faouzi Zarrouki
Karima Relizani Flavien Bizot
Thomas Tensorer
Luis Garcia
Cyrille Vaillend
Aurélie Goyenvalle
Published in:
Ann Neurol., 2022, ISSN 0364-5134
Publisher:
John Wiley & Sons Inc.
DOI:
10.1002/ana.26409
Author(s):
Hashimoto Y, Kuniishi H, Sakai K, Fukushima Y, Du X, Yamashiro K, Hori K, Motohashi N, Imamura M, Hoshino M, Yamada M, Araki T, Sakagami H, Takeda S, Itaka K, Ichinohe N, Muntoni F, Sekiguchi M, Aoki Y.
Published in:
Prog Neurobiol., 2021, ISSN 0301-0082
Publisher:
Pergamon Press Ltd.
DOI:
10.1016/j.pneurobio.2022.102288
Author(s):
Pablo Perdomo-Quinteiro, Sergiu Sisminiuc, Paraskevi Sakellariou, Marco Roos, Pietro Spitali, Núria Queralt-Rosinach
Published in:
SWAT4HCLS 2023: The 14th International Conference on Semantic Web Applications and Tools for Health Care and Life Sciences, 2023, ISSN 1613-0073
Publisher:
CEUR Workshop Proceedings
Author(s):
Kate Maresh, Andriani Papageorgiou, Deborah Ridout, Neil A Harrison, William Mandy, David Skuse, Francesco Muntoni
Published in:
Research Article (peer reviewed), 2022, ISSN 1460-2156
Publisher:
Oxford University Press
DOI:
10.1093/brain/awac048
Author(s):
Minou Verhaeg, Lizette van der Pijl, Luna Mastenbroek, Esmee van der Linde, Angel van Uffelen, Urani Leka, Tiberiu Stan, Maaike van Putten, Luciano Censoni
Published in:
2024
Publisher:
SWEBAGS
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