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Impaired STING Activation Due to a Variant in the E3 Ubiquitin Ligase AMFR in a Patient with Severe VZV Infection and Hemophagocytic Lymphohistiocytosis

Journal of clinical immunology, 2024-02, Vol.44 (2), p.56-56, Article 56

2024

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Restriction of C1-inhibitor activity in hereditary angioedema by dominant-negative effects of disease-associated SERPING1 gene variants

Journal of allergy and clinical immunology, 2023-11, Vol.152 (5), p.1218-1236.e9

2023

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CRISPR-screen identifies ZIP9 and dysregulated Zn2+ homeostasis as a cause of cancer-associated changes in glycosylation

Glycobiology (Oxford), 2023-10, Vol.33 (9), p.700-714

2023

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Resistance to vincristine in DLBCL by disruption of p53‐induced cell cycle arrest and apoptosis mediated by KIF18B and USP28

British journal of haematology, 2023-08, Vol.202 (4), p.825-839

2023

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Prime editing in hematopoietic stem cells-From ex vivo to in vivo CRISPR-based treatment of blood disorders

Frontiers in genome editing, 2023-03, Vol.5, p.1148650-1148650

2023

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Delivering genes with human immunodeficiency virus-derived vehicles: still state-of-the-art after 25 years

Journal of biomedical science, 2022-10, Vol.29 (1), p.1-79, Article 79

2022

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CRISPR-Cas9-directed gene tagging using a single integrase-defective lentiviral vector carrying a transposase-based Cas9 off switch

Molecular therapy. Nucleic acids, 2022-09, Vol.29, p.563-576

2022

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A genome-wide CRISPR-Cas9 knockout screen identifies novel PARP inhibitor resistance genes in prostate cancer

Oncogene, 2022-09, Vol.41 (37), p.4271-4281

2022

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A truncated reverse transcriptase enhances prime editing by split AAV vectors

Molecular therapy, 2022-09, Vol.30 (9), p.2942-2951

2022

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Simple Autofluorescence-Restrictive Sorting of eGFP+ RPE Cells Allows Reliable Assessment of Targeted Retinal Gene Therapy

Frontiers in Drug Delivery, 2022-05, Vol.2

2022

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Single-Cell Monitoring of Activated Innate Immune Signaling by a d2eGFP-Based Reporter Mimicking Time-Restricted Activation of IFNB1 Expression

Frontiers in cellular and infection microbiology, 2022-01, Vol.11, p.784762-784762

2022

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piggyPrime: High-Efficacy Prime Editing in Human Cells Using piggyBac-Based DNA Transposition

Frontiers in genome editing, 2021-11, Vol.3, p.786893-786893

2021

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Targeted Knockout of the Vegfa Gene in the Retina by Subretinal Injection of RNP Complexes Containing Cas9 Protein and Modified sgRNAs

Molecular therapy, 2021-01, Vol.29 (1), p.191-207

2021

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Identification of BLNK and BTK as mediators of rituximab‐induced programmed cell death by CRISPR screens in GCB‐subtype diffuse large B‐cell lymphoma

Molecular oncology, 2020-09, Vol.14 (9), p.1978-1997

2020

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Sustained transgene expression from sleeping beauty DNA transposons containing a core fragment of the HNRPA2B1-CBX3 ubiquitous chromatin opening element (UCOE)

BMC biotechnology, 2019-11, Vol.19 (1), p.75-75, Article 75

2019

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Suppression of Choroidal Neovascularization by AAV-Based Dual-Acting Antiangiogenic Gene Therapy

Molecular therapy. Nucleic acids, 2019-06, Vol.16, p.38-50

2019

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MicroRNA-155 controls vincristine sensitivity and predicts superior clinical outcome in diffuse large B-cell lymphoma

Blood advances, 2019-04, Vol.3 (7), p.1185-1196

2019

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Dominant-negative SERPING1 variants cause intracellular retention of C1 inhibitor in hereditary angioedema

The Journal of clinical investigation, 2019-01, Vol.129 (1), p.388-405

2019

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Toward In Vivo Gene Therapy Using CRISPR

Methods in molecular biology (Clifton, N.J.), 2019, Vol.1961, p.293

2019

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Production and Validation of Lentiviral Vectors for CRISPR/Cas9 Delivery

Methods in molecular biology (Clifton, N.J.), 2019, Vol.1961, p.93

2019

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Dominant-negative SERPING1 variants cause intracellular retention of C1 inhibitor in hereditary angioedema

The Journal of clinical investigation, 2019-01, Vol.129 (1), p.388

2019

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Enhanced Tailored MicroRNA Sponge Activity of RNA Pol II-Transcribed TuD Hairpins Relative to Ectopically Expressed ciRS7-Derived circRNAs

Molecular therapy. Nucleic acids, 2018-12, Vol.13, p.365-375

2018

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Viral delivery of genome-modifying proteins for cellular reprogramming

Current opinion in genetics & development, 2018-10, Vol.52, p.92-99

Mikkelsen, Jacob Giehm

2018

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Impaired STING Activation Due to a Variant in the E3 Ubiquitin Ligase AMFR in a Patient with Severe VZV Infection and Hemophagocytic Lymphohistiocytosis

Restriction of C1-inhibitor activity in hereditary angioedema by dominant-negative effects of disease-associated SERPING1 gene variants

CRISPR-screen identifies ZIP9 and dysregulated Zn2+ homeostasis as a cause of cancer-associated changes in glycosylation

Resistance to vincristine in DLBCL by disruption of p53‐induced cell cycle arrest and apoptosis mediated by KIF18B and USP28

Prime editing in hematopoietic stem cells-From ex vivo to in vivo CRISPR-based treatment of blood disorders

Delivering genes with human immunodeficiency virus-derived vehicles: still state-of-the-art after 25 years

CRISPR-Cas9-directed gene tagging using a single integrase-defective lentiviral vector carrying a transposase-based Cas9 off switch

A genome-wide CRISPR-Cas9 knockout screen identifies novel PARP inhibitor resistance genes in prostate cancer

A truncated reverse transcriptase enhances prime editing by split AAV vectors

Simple Autofluorescence-Restrictive Sorting of eGFP+ RPE Cells Allows Reliable Assessment of Targeted Retinal Gene Therapy

Single-Cell Monitoring of Activated Innate Immune Signaling by a d2eGFP-Based Reporter Mimicking Time-Restricted Activation of IFNB1 Expression

piggyPrime: High-Efficacy Prime Editing in Human Cells Using piggyBac-Based DNA Transposition

Essential role of autophagy in restricting poliovirus infection revealed by identification of an ATG7 defect in a poliomyelitis patient

Targeted Knockout of the Vegfa Gene in the Retina by Subretinal Injection of RNP Complexes Containing Cas9 Protein and Modified sgRNAs

Defects in LC3B2 and ATG4A underlie HSV2 meningitis and reveal a critical role for autophagy in antiviral defense in humans

Identification of BLNK and BTK as mediators of rituximab‐induced programmed cell death by CRISPR screens in GCB‐subtype diffuse large B‐cell lymphoma

Sustained transgene expression from sleeping beauty DNA transposons containing a core fragment of the HNRPA2B1-CBX3 ubiquitous chromatin opening element (UCOE)

Suppression of Choroidal Neovascularization by AAV-Based Dual-Acting Antiangiogenic Gene Therapy

MicroRNA-155 controls vincristine sensitivity and predicts superior clinical outcome in diffuse large B-cell lymphoma

Dominant-negative SERPING1 variants cause intracellular retention of C1 inhibitor in hereditary angioedema

Toward In Vivo Gene Therapy Using CRISPR

Production and Validation of Lentiviral Vectors for CRISPR/Cas9 Delivery

Dominant-negative SERPING1 variants cause intracellular retention of C1 inhibitor in hereditary angioedema

Enhanced Tailored MicroRNA Sponge Activity of RNA Pol II-Transcribed TuD Hairpins Relative to Ectopically Expressed ciRS7-Derived circRNAs

Viral delivery of genome-modifying proteins for cellular reprogramming

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