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

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