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Nature Biomedical Engineering|June 17, 2024
Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cellsSofia E Luna, Joab Camarena, Jessica P Hampton, et al.
Molecular and Cellular Biology|May 19, 2010
MOF and histone H4 acetylation at lysine 16 are critical for DNA damage response and double-strand break repairGirdhar G Sharma, Sairei So, Arun Gupta, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 16, 2023
Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34+ hematopoietic stem and progenitor cellsM Kyle Cromer, Kiran R Majeti, Garrett R Rettig, et al.
Blood Advances|October 5, 2019
High-efficiency CRISPR induction of t(9;11) chromosomal translocations and acute leukemias in human blood stem cellsJohan Jeong, Astraea Jager, Pablo Domizi, et al.
Nature Protocols|April 5, 2007
Standardized reagents and protocols for engineering zinc finger nucleases by modular assemblyDavid A Wright, Stacey Thibodeau-Beganny, Jeffry D Sander, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 15, 2018
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic RearrangementsAnnalisa Lattanzi, Vasco Meneghini, Giulia Pavani, et al.
Molecular Therapy. Methods & Clinical Development|February 25, 2025
Human striatal progenitor cells that contain inducible safeguards and overexpress BDNF rescue Huntington's disease phenotypesDanielle A Simmons, Sridhar Selvaraj, Tingshuo Chen, et al.
Blood Advances|November 7, 2025
Novel humanized loss-of-function NF1 mouse model of juvenile myelomonocytic leukemiaRoshani Sinha, Rachana V Patil, Rosa Romano, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 20, 2021
Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editingJose Bonafont, Angeles Mencía, Esteban Chacón-Solano, et al.
Nucleic Acids Research|August 14, 2025
Gene editing in hematopoietic stem cells by co-delivery of Cas9/sgRNA ribonucleoprotein and templates for homology-directed repair in 'all-in-one' lentivirus-derived nanoparticlesSofie Andersen, Jonas Holst Wolff, Thomas Wisbech Skov, et al.
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