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


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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

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