Publications

Gene-corrected regulatory T cell therapy for IL2RA deficiency

Alvin S Ha, Nechama Kalter, Michael Rosenberg, Luis A Acevedo, Brianna Liang, Weihong Liu, Sreenivasan Paruthiyil, Meenal Sinha, Alexander Vu, Vinh Nguyen, Zhongxia Qi, Netravathi Krishnappa, Jinna Shu, Jingwei Yu, Jason Catanzaro, Jeffrey A Bluestone, Qizhi Tang, Fyodor D Urnov, Alexander Marson, Ayal Hendel, Kevan C Herold, Brian R Shy, Jonathan H Esensten

Proceedings of the second annual meeting of GenE-HumDi (COST Action 21113)

María Ortiz-Bueno, Iris Ramos-Hernández, Luis Algeciras-Jiménez, Nechama Kalter, Juan Roberto Rodríguez-Madoz, Jose Bonafont, Rajeevkumar Raveendran Nair, Oliver Feeney, Laura Torella, Lluis Montoliu, Petros Patsali, Claudio Mussolino, Yonglun Luo, Merita Xhetani, Alessia Cavazza, Ayal Hendel, Karim Benabdellah, Carsten Werner Lederer, Francisco J Molina-Estévez

Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges

Nechama Kalter, Carla Fuster-García, Alfredo Silva, Víctor Ronco-Díaz, Stefano Roncelli, Giandomenico Turchiano, Jan Gorodkin, Toni Cathomen, Karim Benabdellah, Ciaran Lee, Ayal Hendel

Navigating disruption in the PID landscape: embracing opportunities and anticipating threats in the next ten years

Lúcia Mamede, Roser Cantenys Sabà, Samya Van Coillie, Johan Prévot, Silvia Sánchez-Ramón, Cecilia Poli, Anne Barasa, Björn W Schuller, Ayal Hendel, Nicolas Garcelon, Cornelis Boersma, Pamela Lee, Claire Booth, Luigi D Notarangelo, Jose Drabwell, Nicholas L Rider, Frank J T Staal, Martin Van Hagen, Martine Pergent, Jacques G Rivière, Nizar Mahlaoui

Precise measurement of CRISPR genome editing outcomes through single-cell DNA sequencing

Nechama Kalter, Saurabh Gulati, Michael Rosenberg, Qawer Ayaz, Joanne Nguyen, Shu Wang, Benjamin Schroeder, Chieh-Yuan Li, Ayal Hendel

Targeting Tumor-Associated Sialic Acids Using Chimeric Switch Receptors Based on Siglec-9 Enhances the Antitumor Efficacy of Engineered T Cells

Vasyl Eisenberg, Shiran Hoogi, Erel Katzman, Nimrod Ben Haim, Raphaelle Zur-Toledano, Maria Radman, Yishai Reboh, Oranit Zadok, Iris Kamer, Jair Bar, Irit Sagi, Ayal Hendel, Cyrille J Cohen

Quantifying allele-specific CRISPR editing activity with CRISPECTOR2.0

Guy Assa, Nechama Kalter, Michael Rosenberg, Avigail Beck, Oshry Markovich, Tanya Gontmakher, Ayal Hendel, Zohar Yakhini

Evolution of Gene Therapy for Inborn Errors of Immunity

Ido Somekh, Ayal Hendel, Raz Somech

Nicotinamide-Expanded Allogeneic Natural Killer Cells with CD38 Deletion, Expressing an Enhanced CD38 Chimeric Antigen Receptor, Target Multiple Myeloma Cells

Avishay Edri, Nimrod Ben-Haim, Astar Hailu, Nurit Brycman, Orit Berhani-Zipori, Julia Rifman, Sherri Cohen, Dima Yackoubov, Michael Rosenberg, Ronit Simantov, Hideshima Teru, Keiji Kurata, Kenneth Carl Anderson, Ayal Hendel, Aviad Pato, Yona Geffen

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

Daniel Allen, Orli Knop, Bryan Itkowitz, Nechama Kalter, Michael Rosenberg, Ortal Iancu, Katia Beider, Yu Nee Lee, Arnon Nagler, Raz Somech, Ayal Hendel

Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

Alessia Cavazza, Ayal Hendel, Rasmus O Bak, Paula Rio, Marc Güell, Duško Lainšček, Virginia Arechavala-Gomeza, Ling Peng, Fatma Zehra Hapil, Joshua Harvey, Francisco G Ortega, Coral Gonzalez-Martinez, Carsten W Lederer, Kasper Mikkelsen, Giedrius Gasiunas, Nechama Kalter, Manuel A F V Gonçalves, Julie Petersen, Alejandro Garanto, Lluis Montoliu, Marcello Maresca, Stefan E Seemann, Jan Gorodkin, Loubna Mazini, Rosario Sanchez, Juan R Rodriguez-Madoz, Noelia Maldonado-Pérez, Torella Laura, Michael Schmueck-Henneresse, Cristina Maccalli, Julian Grünewald, Gloria Carmona, Neli Kachamakova-Trojanowska, Annarita Miccio, Francisco Martin, Giandomenico Turchiano, Toni Cathomen, Yonglun Luo, Shengdar Q Tsai, Karim Benabdellah; COST Action CA21113

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

Daniel Allen, Nechama Kalter, Michael Rosenberg, Ayal Hendel

Editorial: CRISPR and beyond: Cutting-edge technologies for gene correction in therapeutic applications

Ayal Hendel, Rasmus O Bak

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

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

Lessons Learned From Five Years of Newborn Screening for Severe Combined Immunodeficiency in Israel

Atar Lev, Idan Sharir, Amos J Simon, Shiran Levy, Yu Nee Lee, Shirly Frizinsky, Suha Daas, Talia Saraf-Levy, Arnon Broides, Amit Nahum, Suhair Hanna, Polina Stepensky, Ori Toker, Ilan Dalal, Amos Etzioni, Jerry Stein, Etai Adam, Ayal Hendel, Nufar Marcus, Shlomo Almashanu, Raz Somech

Different hotspot p53 mutants exert distinct phenotypes and predict outcome of colorectal cancer patients

Ori Hassin, Nishanth Belugali Nataraj, Michal Shreberk-Shaked, Yael Aylon, Rona Yaeger, Giulia Fontemaggi, Saptaparna Mukherjee, Martino Maddalena, Adi Avioz, Ortal Iancu, Giuseppe Mallel, Anat Gershoni, Inna Grosheva, Ester Feldmesser, Shifra Ben-Dor, Ofra Golani, Ayal Hendel, Giovanni Blandino, David Kelsen, Yosef Yarden, Moshe Oren

Microfluidic tool for rapid functional characterization of CRISPR complexes

Dana Peleg-Chen, Guy Shuvali, Lev Brio, Amit Ifrach, Ortal Iancu, Efrat Barbiro-Michaely, Ayal Hendel, Doron Gerber

Stepwise-edited, human melanoma models reveal mutations' effect on tumor and microenvironment

Eran Hodis, Elena Torlai Triglia, John Y H Kwon, Tommaso Biancalani, Labib R Zakka, Saurabh Parkar, Jan-Christian Hütter, Lorenzo Buffoni, Toni M Delorey, Devan Phillips, Danielle Dionne, Lan T Nguyen, Denis Schapiro, Zoltan Maliga, Connor A Jacobson, Ayal Hendel, Orit Rozenblatt-Rosen, Martin C Mihm Jr, Levi A Garraway, Aviv Regev

High-Throughput Imaging of CRISPR- and Recombinant Adeno-Associated Virus-Induced DNA Damage Response in Human Hematopoietic Stem and Progenitor Cells

Daniel Allen, Lucien E Weiss, Alon Saguy, Michael Rosenberg, Ortal Iancu, Omri Matalon, Ciaran Lee, Katia Beider, Arnon Nagler, Yoav Shechtman, Ayal Hendel

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

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

Exploring genetic defects in adults who were clinically diagnosed as severe combined immune deficiency during infancy

Ido Somekh, Atar Lev, Ortal Barel, Yu Nee Lee, Ayal Hendel, Amos J Simon, Raz Somech

Inherited SLP76 deficiency in humans causes severe combined immunodeficiency, neutrophil and platelet defects

Atar Lev, Yu Nee Lee, Guangping Sun, Enas Hallumi, Amos J Simon, Keren S Zrihen, Shiran Levy, Tal Beit Halevi, Maria Papazian, Neta Shwartz, Ido Somekh, Sarina Levy-Mendelovich, Baruch Wolach, Ronit Gavrieli, Helly Vernitsky, Ortal Barel, Elisheva Javasky, Tali Stauber, Chi A Ma, Yuan Zhang, Ninette Amariglio, Gideon Rechavi, Ayal Hendel, Deborah Yablonski, Joshua D Milner, Raz Somech

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

Daniel Allen, Michael Rosenberg, Ayal Hendel

Chemical Modification of Guide RNAs for Improved CRISPR Activity in CD34+ Human Hematopoietic Stem and Progenitor Cells

Jenny Shapiro, Adi Tovin, Ortal Iancu, Daniel Allen, Ayal Hendel

The Cell Line A-to-I RNA Editing Catalogue

Amos A Schaffer, Eli Kopel, Ayal Hendel, Ernesto Picardi, Erez Y Levanon, Eli Eisenberg

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

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

CRISPR-based Gene Editing Enables FOXP3 Gene Repair in IPEX Patient Cells

M Goodwin, E Lee, U Lakshmanan, S Shipp, L Froessl, F Barzaghi, L Passerini, M Narula, A Sheikali, C M Lee, G Bao, C S Bauer, H K Miller, M Garcia-Lloret, M J Butte, A Bertaina, A Shah, M Pavel-Dinu, A Hendel, M Porteus, M G Roncarolo, R Bacchetta

Reduced function and diversity of T cell repertoire and distinct clinical course in patients with IL7RA mutation

Atar Lev, Amos J Simon, Ortal Barel, Eran Eyal, Efrat Glick-Saar, Omri Nayshool, Ohad Birk, Tali Stauber, Amit Hochberg, Arnon Broides, Shlomo Almashanu, Ayal Hendel, Yu Nee Lee, Raz Somech

Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells

M Kyle Cromer, Sriram Vaidyanathan, Daniel E Ryan, Bo Curry, Anne Bergstrom Lucas, Joab Camarena, Milan Kaushik, Sarah R Hay, Renata M Martin, Israel Steinfeld, Rasmus O Bak, Daniel P Dever, Ayal Hendel, Laurakay Bruhn, Matthew H Porteus

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification

Sriram Vaidyanathan, Krist T Azizian, A K M Ashiqul Haque, Jordana M Henderson, Ayal Hendel, Sabrina Shore, Justin S Antony, Richard I Hogrefe, Michael S D Kormann, Matthew H Porteus, Anton P McCaffrey

Therapeutic Genome Editing and its Potential Enhancement through CRISPR Guide RNA and Cas9 Modifications

Nurit Assia Batzir, Adi Tovin, Ayal Hendel

A Comprehensive TALEN-Based Knockout Library for Generating Human-Induced Pluripotent Stem Cell-Based Models for Cardiovascular Diseases

Ioannis Karakikes, Vittavat Termglinchan, Diana A Cepeda, Jaecheol Lee, Sebastian Diecke, Ayal Hendel, Ilanit Itzhaki, Mohamed Ameen, Rajani Shrestha, Haodi Wu, Ning Ma, Ning-Yi Shao, Timon Seeger, Nicole Woo, Kitchener D Wilson, Elena Matsa, Matthew H Porteus, Vittorio Sebastiano, Joseph C Wu

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

Daniel P Dever, Rasmus O Bak, Andreas Reinisch, Joab Camarena, Gabriel Washington, Carmencita E Nicolas, Mara Pavel-Dinu, Nivi Saxena, Alec B Wilkens, Sruthi Mantri, Nobuko Uchida, Ayal Hendel, Anupama Narla, Ravindra Majeti, Kenneth I Weinberg, Matthew H Porteus

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

Ayal Hendel, Rasmus O Bak, Joseph T Clark, Andrew B Kennedy, Daniel E Ryan, Subhadeep Roy, Israel Steinfeld, Benjamin D Lunstad, Robert J Kaiser, Alec B Wilkens, Rosa Bacchetta, Anya Tsalenko, Douglas Dellinger, Laurakay Bruhn, Matthew H Porteus

Quantifying on- and off-target genome editing

Ayal Hendel, Eli J Fine, Gang Bao, Matthew H Porteus

Quantifying Genome Editing Outcomes at Endogenous Loci using SMRT Sequencing

Ayal Hendel, Eric J Kildebeck, Eli J Fine, Niraj Punjya, Vittorio Sebastiano, Gang Bao, Matthew H Porteus

Nuclease-mediated gene editing by homologous recombination of the human globin locus

Richard A Voit, Ayal Hendel, Shondra M Pruett-Miller, Matthew H Porteus

DNA damage bypass operates in the S and G2 phases of the cell cycle and exhibits differential mutagenicity

Noam Diamant, Ayal Hendel, Ilan Vered, Thomas Carell, Thomas Reissner, Niels de Wind, Nicholas Geacinov, Zvi Livneh

Quantitative measurement of translesion DNA synthesis in mammalian cells

Omer Ziv, Noam Diamant, Sigal Shachar, Ayal Hendel, Zvi Livneh

PCNA Ubiquitination Is Important, But Not Essential for Translesion DNA Synthesis in Mammalian Cells

Ayal Hendel, Peter H L Krijger, Noam Diamant, Zohar Goren, Petra Langerak, Jungmin Kim, Thomas Reissner, Kyoo-young Lee, Nicholas E Geacintov, Thomas Carell, Kyungjae Myung, Satoshi Tateishi, Alan D'Andrea, Heinz Jacobs, Zvi Livneh

Repair of gaps opposite lesions by homologous recombination in mammalian cells

Sheera Adar, Lior Izhar, Ayal Hendel, Nicholas Geacintov, Zvi Livneh

Repair of gaps opposite lesions by homologous recombination in mammalian cells

Sheera Adar, Lior Izhar, Ayal Hendel, Nicholas Geacintov, Zvi Livneh

Separate domains of Rev1 mediate two modes of DNA damage bypass in mammalian cells

Jacob G Jansen, Anastasia Tsaalbi-Shtylik, Giel Hendriks, Himabindu Gali, Ayal Hendel, Fredrik Johansson, Klaus Erixon, Zvi Livneh, Leon H F Mullenders, Lajos Haracska, Niels de Wind

Reduced efficiency and increased mutagenicity of translesion DNA synthesis across a TT cyclobutane pyrimidine dimer, but not a TT 6-4 photoproduct, in human cells lacking DNA polymerase eta

Ayal Hendel, Omer Ziv, Quentin Gueranger, Nicholas Geacintov, Zvi Livneh

Single-stranded DNA-binding protein recruits DNA polymerase V to primer termini on RecA-coated DNA

Gali Arad, Ayal Hendel, Claus Urbanke, Ute Curth, Zvi Livneh

Mechanism of Translesion DNA Synthesis in Escherichia coli. DNA Damage Recognition

Zvi Livneh, Ayelet Maor-Shoshani, Moshe Goldsmith, Gali Arad, Ayal Hendel, Lior Izhar