Rescuing Cognitive Deficits in Neurodevelopmental Disorders by Gene Editing in Brain Development: the Case of Down Syndrome

Project Information

GenEdiDS

Grant agreement ID: 725563

DOI

10.3030/725563

Project closed

EC signature date 26 April 2017

Start date 1 October 2017

End date 30 September 2023

Funded under

• EXCELLENT SCIENCE - European Research Council (ERC)

Total cost

€ 2 000 000,00

EU contribution

€ 2 000 000,00

Coordinated by

FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA
Italy

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 .

Publications

Peer reviewed articles (11)

Rescuing Over-activated Microglia Restores Cognitive Performance in Juvenile Animals of the Dp(16) Mouse Model of Down Syndrome

Author(s): Bruno Pinto, Giovanni Morelli, Mohit Rastogi, Annalisa Savardi, Amos Fumagalli, Andrea Petretto, Martina Bartolucci, Emilio Varea, Tiziano Catelani, Andrea Contestabile, Laura E. Perlini, Laura Cancedda
Published in: Neuron, 2020, ISSN 0896-6273
Publisher: Cell Press
DOI: 10.1016/j.neuron.2020.09.010

Viral vector-based gene therapy in neurological disease: The future is now

Author(s): Jared B. Smith1* Laura Cancedda2 Sergiusz Jozwiak3 Andrew C. Mercer1
Published in: Front. Neurol, Issue Volume 14 - 2023, 2023, ISSN 1664-2295
Publisher: Frontiers Research Foundation
DOI: 10.3389/fneur.2023.1153681

Design, Synthesis, In Vitro and In Vivo Characterization of Selective NKCC1 Inhibitors for the Treatment of Core Symptoms in Down Syndrome

Author(s): Marco Borgogno, Annalisa Savardi, Jacopo Manigrasso, Alessandra Turci, Corinne Portioli, Giuliana Ottonello, Sine Mandrup Bertozzi, Andrea Armirotti, Andrea Contestabile, Laura Cancedda*, and Marco De Vivo*
Published in: Journal of Medicinal Chemistry, 2021, ISSN 0022-2623
Publisher: American Chemical Society
DOI: 10.1021/acs.jmedchem.1c00603

Preclinical Development of the Na-K-2Cl Co-transporter-1 (NKCC1) Inhibitor ARN23746 for the Treatment of Neurodevelopmental Disorders

Author(s): Annalisa Savardi; Andrea Patricelli Malizia; Marco De Vivo; Laura Cancedda; Marco Borgogno
Published in: Crossref, Issue 1, 2023, ISSN 0002-7863
Publisher: American Chemical Society
DOI: 10.1021/acsptsci.2c00197

Discovery of a Small Molecule Drug Candidate for Selective NKCC1 Inhibition in Brain Disorders

Author(s): Annalisa Savardi, Marco Borgogno, Roberto Narducci, Giuseppina La Sala, Jose Antonio Ortega, Maria Summa, Andrea Armirotti, Rosalia Bertorelli, Andrea Contestabile, Marco De Vivo, Laura Cancedda
Published in: Chem, Issue 6/8, 2020, Page(s) 2073-2096, ISSN 2451-9294
Publisher: Chem, Cell press
DOI: 10.1016/j.chempr.2020.06.017

Restoring neuronal chloride homeostasis with anti-NKCC1 gene therapy rescues cognitive deficits in a mouse model of Down syndrome

Author(s): Martina Parrini; Shovan Naskar; Micol Alberti; Ilaria Colombi; Giovanni Morelli; Anna Rocchi; Marina Nanni; Federica Piccardi; Severine Charles; Giuseppe Ronzitti; Federico Mingozzi; Andrea Contestabile; Laura Cancedda
Published in: Molecular Therapy, Issue 1, 2021, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2021.05.023

The development of synaptic transmission is time-locked to early social behaviors in rats

Author(s): Shovan Naskar, Roberto Narducci, Edoardo Balzani, Andrzej W. Cwetsch, Valter Tucci, Laura Cancedda
Published in: Nature Communications, Issue 10/1, 2019, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-019-09156-3

NEGR1 and FGFR2 cooperatively regulate cortical development and core behaviours related to autism disorders in mice

Author(s): Joanna Szczurkowska, Francesca Pischedda, Bruno Pinto, Francesca Managò, Carola A Haas, Maria Summa, Rosalia Bertorelli, Francesco Papaleo, Michael K Schäfer, Giovanni Piccoli, Laura Cancedda
Published in: Brain, 2018, ISSN 0006-8950
Publisher: Oxford University Press
DOI: 10.1093/brain/awy190

In vivo methods for acute modulation of gene expression in the central nervous system

Author(s): Andrzej W. Cwetsch, Bruno Pinto, Annalisa Savardi, Laura Cancedda
Published in: Progress in Neurobiology, Issue 168, 2018, Page(s) 69-85, ISSN 0301-0082
Publisher: Pergamon Press Ltd.
DOI: 10.1016/j.pneurobio.2018.04.008

Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration

Author(s): Paola Vagni, Laura E. Perlini, Naïg A. L. Chenais, Tommaso Marchetti, Martina Parrini, Andrea Contestabile, Laura Cancedda, Diego Ghezzi
Published in: Frontiers in Neuroscience, Issue 13, 2019, ISSN 1662-453X
Publisher: Frontiers in Neuroscience
DOI: 10.3389/fnins.2019.00945

Pharmacological tools to target NKCC1 in brain disorders.

Author(s): Annalisa Savardi; Marco Borgogno; Marco De Vivo; Laura Cancedda
Published in: Trends in Pharmacological Sciences, Issue 1, 2021, ISSN 0165-6147
Publisher: Elsevier BV
DOI: 10.1016/j.tips.2021.09.005

Intellectual Property Rights

Patent (1)

A VECTOR AND PHARMACEUTICAL COMPOSITION FOR REDUCING THE EXPRESSION OF NKCC1 IN A SUBJECT IN NEED THEREOF, AS WELL AS THE RELATED THERAPEUTIC TREATMENT METHOD

Application/Publication number: 20 18059255
Date: 2018-04-11
Applicant(s): FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA