CRISPR Gene Correction for Severe Combined Immunodeficiency Caused by Mutations in Recombination-activating gene 1 and 2 (RAG1 and RAG2)

Project Information

CRISS

Grant agreement ID: 755758

DOI 10.3030/755758

Project closed

EC signature date 12 September 2017

Start date 1 October 2017

End date 30 September 2023

Funded under

• EXCELLENT SCIENCE - European Research Council (ERC)

Total cost € 1 372 839,00

EU contribution € 1 372 839,00

Coordinated by

BAR ILAN UNIVERSITY
Israel

Deliverables

Open Research Data Pilot (1)

Data Management Plan

Make my research project results open to the public as much as possible

Publications

Peer reviewed articles (7)

Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood Disorders

Author(s): Daniel Allen; Nechama Kalter; Michael Rosenberg; Ayal Hendel
Published in: Pharmaceutics, 2023, ISSN 2078-2489
Publisher: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/pharmaceutics15051329

High-Throughput Imaging of CRISPR- and RecombinantAdeno-Associated Virus–Induced DNA Damage Responsein Human Hematopoietic Stem and Progenitor Cells

Author(s): Daniel Allen, Lucien E Weiss, Alon Saguy, Michael Rosenberg, Ortal Iancu, Omri Matalon, Ciaran Lee, Katia Beider, Arnon Nagler, Yoav Shechtman, Ayal Hendel
Published in: The CRISPR Journal, 2022, ISSN 2573-1602
Publisher: Mary Ann Liebert, Inc.
DOI: 10.1089/crispr.2021.0128

Using Synthetically Engineered Guide RNAs to Enhance CRISPR Genome Editing Systems in Mammalian Cells.

Author(s): Daniel Allen, Michael Rosenberg, Ayal Hendel
Published in: Frontiers in Genome Editing, 2021, ISSN 2673-3439
Publisher: Frontiers Media
DOI: 10.3389/fgeed.2020.617910

Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs

Author(s): Ortal Iancu , Daniel Allen, Orli Knop, Yonathan Zehavi, Dor Breier, Adaya Arbiv, Atar Lev, Yu Nee Lee, Katia Beider, Arnon Nagler, Raz Somech, Ayal Hendel
Published in: Molecular Therapy Nucleic Acids, 2022, ISSN 2162-2531
Publisher: Nature Publishing Group
DOI: 10.1016/j.omtn.2022.12.006

CRISPECTOR provides accurate estimation of genome editing translocation and off-target activity from comparative NGS data

Author(s): Ido Amit, Ortal Iancu, Alona Levy-Jurgenson, Gavin Kurgan, Matthew S McNeill, Garrett R Rettig, Daniel Allen, Dor Breier, Nimrod Ben Haim, Yu Wang, Leon Anavy, Ayal Hendel, Zohar Yakhini
Published in: Nature Communication, 2021, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-021-22417-4

Increasing CRISPR Efficiency and Measuring Its Specificity in HSPCs Using a Clinically Relevant System

Author(s): Jenny Shapiro, Ortal Iancu, Ashley M. Jacobi, Matthew S. McNeill, Rolf Turk, Garrett R. Rettig, Ido Amit, Adi Tovin-Recht, Zohar Yakhini, Mark A. Behlke, Ayal Hendel
Published in: Molecular Therapy - Methods & Clinical Development, Issue 17, 2020, Page(s) 1097-1107, ISSN 2329-0501
Publisher: Elsevier B.V.
DOI: 10.1016/j.omtm.2020.04.027

CRISPR-Cas9 engineering of the RAG2 locus via complete coding sequence replacement for therapeutic applications

Author(s): Daniel Allen, Orli Knop, Bryan Itkowitz, Nechama Kalter, Michael Rosenberg, Ortal Iancu, Katia Beider, Yu Nee Lee, Arnon Nagler, Raz Somech, Ayal Hendel
Published in: Nature Communications, 2023, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-023-42036-5

Intellectual Property Rights

Other (1)

Patent title: Methods and systems for determining effects of nucleic acid editing

Application/Publication number: PCT/IL2021/050539 WO2021229569
Date: 2020-11-05
Applicant(s): BAR ILAN UNIVERSITY