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Current Progress in Therapeutic Gene Editing for Monogenic Diseases.
Mol Ther. 2016 Mar;24(3):465-74. doi: 10.1038/mt.2016.5. Epub 2016 Jan 14.
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Recent Progress in Genome Editing Approaches for Inherited Cardiovascular Diseases.
Curr Cardiol Rep. 2018 Jun 2;20(7):58. doi: 10.1007/s11886-018-0998-3.
3
Genome editing in large animal models.
Mol Ther. 2021 Nov 3;29(11):3140-3152. doi: 10.1016/j.ymthe.2021.09.026. Epub 2021 Oct 1.
4
Genome-editing Technologies for Gene and Cell Therapy.
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5
Therapeutic genome editing with engineered nucleases.
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6
Gene therapy comes of age.
Science. 2018 Jan 12;359(6372). doi: 10.1126/science.aan4672.
7
The delivery challenge: fulfilling the promise of therapeutic genome editing.
Nat Biotechnol. 2020 Jul;38(7):845-855. doi: 10.1038/s41587-020-0565-5. Epub 2020 Jun 29.
8
Genome Editing Techniques and Their Therapeutic Applications.
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9
Point: Treating Human Genetic Disease One Base Pair at a Time: The Benefits of Gene Editing.
Clin Chem. 2018 Mar;64(3):486-488. doi: 10.1373/clinchem.2017.278309.
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Acceptance and Access to Gene Editing: Science and Our Obligations to Mankind.
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The Interface of Gene Editing with Regenerative Medicine.
Engineering (Beijing). 2025 Mar;46:73-100. doi: 10.1016/j.eng.2024.10.019. Epub 2024 Nov 30.
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Male gamete copies to characterize genome inheritance and generate progenies.
Sci Rep. 2025 May 4;15(1):15600. doi: 10.1038/s41598-025-99188-1.
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Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives.
Pharmaceutics. 2025 Jan 14;17(1):104. doi: 10.3390/pharmaceutics17010104.
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In utero delivery of targeted ionizable lipid nanoparticles facilitates in vivo gene editing of hematopoietic stem cells.
Proc Natl Acad Sci U S A. 2024 Aug 6;121(32):e2400783121. doi: 10.1073/pnas.2400783121. Epub 2024 Jul 30.
7
Rapid Whole-Genome Sequencing and Clinical Management in the PICU: A Multicenter Cohort, 2016-2023.
Pediatr Crit Care Med. 2024 Aug 1;25(8):699-709. doi: 10.1097/PCC.0000000000003522. Epub 2024 Apr 26.
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Developing a genetic testing panel for evaluation of morbidities in kidney transplant recipients.
Kidney Int. 2024 Jul;106(1):115-125. doi: 10.1016/j.kint.2024.02.021. Epub 2024 Mar 21.
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Engineered mischarged transfer RNAs for correcting pathogenic missense mutations.
Mol Ther. 2024 Feb 7;32(2):352-371. doi: 10.1016/j.ymthe.2023.12.014. Epub 2023 Dec 16.
10
Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec.
Mol Ther Methods Clin Dev. 2023 Aug 9;30:474-483. doi: 10.1016/j.omtm.2023.08.002. eCollection 2023 Sep 14.

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A mechanism for the suppression of homologous recombination in G1 cells.
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Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system.
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BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis.
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Functional footprinting of regulatory DNA.
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Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs.
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In vivo genome editing of the albumin locus as a platform for protein replacement therapy.
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Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9.
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Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model.
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