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Proceedings of the National Academy of Sciences of the United States of America|October 25, 2013
IFI16 senses DNA forms of the lentiviral replication cycle and controls HIV-1 replicationMartin R Jakobsen, Rasmus O Bak, Annika Andersen, et al.Nature Communications|May 15, 2015
Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globinBeeke Wienert, Alister P W Funnell, Laura J Norton, et al.Molecular Therapy. Nucleic Acids|September 10, 2018
Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC PurificationSriram Vaidyanathan, Krist T Azizian, A K M Ashiqul Haque, et al.Nature Communications|August 12, 2025
Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous diseaseJonas Holst Wolff, Thomas Wisbech Skov, Didde Haslund, et al.American Journal of Human Genetics|July 24, 2026
Position effect at the SOX3 locus by an interchromosomal insertion causes hereditary spastic paraplegiaThorkild Terkelsen, Veronica Yumiceba, Joshua Kim, et al.Biorxiv : the Preprint Server for Biology|May 20, 2024
Dual α-globin and truncated EPO receptor knockin restores hemoglobin production in α-thalassemia-derived red blood cellsSimon N Chu, Eric Soupene, Beeke Wienert, et al.Biorxiv : the Preprint Server for Biology|March 18, 2024
Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic AntibodiesWilliam N Feist, Sofia E Luna, Kaya Ben-Efraim, et al.Cell Stem Cell|June 23, 2009
Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cellsJizhong Zou, Morgan L Maeder, Prashant Mali, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 31, 2025
Treatment of GATA2 deficiency by allele-specific CRISPR-Cas9-directed gene correction in hematopoietic stem cellsThomas Wisbech Skov, Jonas Holst Wolff, Didde Haslund, et al.Neuron|October 17, 2024
Human assembloids reveal the consequences of CACNA1G gene variants in the thalamocortical pathwayJi-Il Kim, Yuki Miura, Min-Yin Li, et al.Pageof 20