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Explore how we've utilized gene editing technologies in our research.
Chang, C. W. et al. Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting.
Cell reports 12, 1668-1677 (2015). https://doi.org/10.1016/j.celrep.2015.08.013
Gene Correction with CRISPR/Cas9
Modeled human Severe Combined Immunodeficiency (SCID) and demonstrated its correction using CRISPR/Cas9-enhanced gene targeting.
Li, C. et al. Novel HDAd/EBV Reprogramming Vector and Highly Efficient Ad/CRISPR-Cas Sickle Cell Disease Gene Correction.
Sci Rep 6, 30422 (2016).
https://doi.org/10.1038/srep30422
Gene Correction with CRISPR/Cas9
Use of CRISPR/Cas9 to efficiently correct the Sickle mutation in iPSCs derived from Sickle Cell Anemia patients
Mattapally, S. et al. Human Leukocyte Antigen Class I and II Knockout Human Induced Pluripotent Stem Cell-Derived Cells: Universal Donor for Cell Therapy.
J Am Heart Assoc 7, e010239 (2018). https://doi.org/10.1161/JAHA.118.010239
Gene Knock-out to Generate Universal Donors
Created human induced pluripotent stem cell-derived cells with knockout of Human Leukocyte Antigen (HLA) class I and II genes.
Li, J., et al. CRISPR-Cas9, Cre-lox, and Flp-FRT Cascade Strategy for the Precise and Efficient Integration of Exogenous DNA into Cellular Genomes.
CRISPR J 3, 470-486 (2020).
https://doi.org/10.1089/crispr.2020.0042
Exogenouse DNA Integration
Developed a cascade strategy combining CRISPR-Cas9, Cre-lox, and Flp-FRT systems for precise and efficient integration of exogenous DNA into cellular genomes.
Franks, T. M. et al. Engineering, Generation and Preliminary Characterization of a Humanized Porcine Sickle Cell Disease Animal Model.
bioRxiv, 2020.2009.2015.291864 (2020). https://doi.org/10.1101/2020.09.15.291864
​Humannized Porcine Model Generation
Created a humanized porcine model for sickle cell disease by introducing human sickle hemoglobin genes into pigs.
Townes, T. et al. An INTERACT meeting to discuss with FDA about the suitability of proposed animal models and the adequacy of the preclinical (proof of concept) data to support a Phase I/II clinical study to assess the safety of genetic editing of the sickle mutation ex vivo in autologous HSPCfollowed by transplantation to cure the disease.
U.S. Food & Durg Administration, Nov. 19, 2020.
Gene Correction Therapy for SCD
Autologous CRISPR/Cas edited CD34+ hematopoietic stem cells expressing betaglobin /Product Name: Modified CRISPR/Cas Complex
Zhang, Z. et al. RNF2 ablation reprograms the tumor-immune microenvironment and stimulates durable NK and CD4(+) T-cell-dependent antitumor immunity.
Nat Cancer 2, 1018-1038 (2021).
https://doi.org/10.1038/s43018-021-00263-z
Point Muation in Mouse Cells
Generation of point mutations in mouse cancer cell lines with CRISPR/Cas9 to study RNF2's role in tumor-immune microenvironments
Li, C. et al. Generation of chromosome 1p/19q co-deletion by CRISPR/Cas9-guided genomic editing.
Neurooncol Adv 4, vdac131 (2022).
https://doi.org/10.1093/noajnl/vdac131
Chromosome Manipulation
Generation of chromosome 1p/19q co-deletion by CRISPR/Cas9-guided genomic editing
Jin, L. et al. Epigenetic switch reshapes epithelial progenitor cell signatures and drives inflammatory pathogenesis in hidradenitis suppurativa.
Proc Natl Acad Sci U S A 120, e2315096120 (2023). https://doi.org/10.1073/pnas.2315096120
Large Fragemnt Gene Deletions
Generation of large fragment gene deletions with CRISPR/Cas9 in Hidradenitis suppurativa (HS) study
Li, C. et al. Prime editing-mediated correction of the CFTR W1282X mutation in iPSCs and derived airway epithelial cells.
PLoS One 18, e0295009 (2023). https://doi.org/10.1371/journal.pone.0295009
Gene Correction with Prime Editing
Prime editing-mediated correction of the CFTR W1282X mutation in iPSCs and derived airway epithelial cells
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