Rasmus O. Bak
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View article: CXCR2 deficiency with myelokathexis caused by a novel variant: correction via CRISPR/Cas9
CXCR2 deficiency with myelokathexis caused by a novel variant: correction via CRISPR/Cas9 Open
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View article: Orthogonal CRISPR systems for targeted integration and multiplex base editing enable nonviral engineering of allogeneic CAR-T cells
Orthogonal CRISPR systems for targeted integration and multiplex base editing enable nonviral engineering of allogeneic CAR-T cells Open
View article: Editing of <i>ADA2</i> Point Mutation in Human Hematopoietic Stem Cells
Editing of <i>ADA2</i> Point Mutation in Human Hematopoietic Stem Cells Open
Background The homozygous ADA2 : c.506G>A (p.Arg169Gln; p.R169Q) variant accounts for majority of Deficiency in Adenosine Deaminase 2 (DADA2). This monogenic disorder may be amenable to ex vivo gene therapy by correcting the pathogenic mut…
View article: Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous disease
Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous disease Open
Chronic granulomatous disease (CGD) is a severe inborn error of immunity caused by NADPH oxidase defects. Here, we develop CRISPR/Cas9-based gene editing strategies for correction of variants in the CYBA and CYBB genes causing CGD. For X-l…
View article: 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 nanoparticles
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 nanoparticles Open
Repair of double-strand DNA breaks generated by site-directed endonucleases, like Cas9, is the hallmark of gene editing based on homology-directed repair (HDR). HDR uses an exogenous DNA template to restore the cleaved DNA sequence and can…
View article: Development of a Gene Editing Platform to Enhance CAR-T Therapy Through Inducible IL-15 Expression at the PD-1 Locus
Development of a Gene Editing Platform to Enhance CAR-T Therapy Through Inducible IL-15 Expression at the PD-1 Locus Open
BackgroundAdoptive cell therapy (ACT) with genetically engineered T cells expressing chimeric antigen receptors (CARs) has emerged as a promising treatment option for patients with refractory leukaemia or lymphoma. Despite its success in t…
View article: Corrigendum: Characterization of TLR9 responsiveness in cell subsets derived from in vitro pDC differentiation of hematopoietic stem and progenitor cells
Corrigendum: Characterization of TLR9 responsiveness in cell subsets derived from in vitro pDC differentiation of hematopoietic stem and progenitor cells Open
[This corrects the article DOI: 10.3389/fimmu.2025.1550397.].
View article: CRISPR/Cas gene editing of haematopoietic stem cells for curing primary immunodeficiency
CRISPR/Cas gene editing of haematopoietic stem cells for curing primary immunodeficiency Open
Primary immunodeficiencies are rare monogenic inborn errors of immunity and can involve any combination of infection, autoimmunity, inflammation, and malignancy. While increased use of whole genome sequencing has vastly improved diagnosis,…
View article: Single-stranded HDR templates with truncated Cas12a-binding sequences improve knock-in efficiencies in primary human T cells
Single-stranded HDR templates with truncated Cas12a-binding sequences improve knock-in efficiencies in primary human T cells Open
View article: Characterization of TLR9 responsiveness in cell subsets derived from in vitro pDC differentiation of hematopoietic stem and progenitor cells
Characterization of TLR9 responsiveness in cell subsets derived from in vitro pDC differentiation of hematopoietic stem and progenitor cells Open
Plasmacytoid dendritic cells (pDCs) are multifunctional immune cells with roles in both the innate and adaptive immune system. Their hallmark function is production of large amounts of type I interferons in response to viral infections, bu…
View article: Distinctive CD8+ T cell activation by antigen-presenting plasmacytoid dendritic cells compared to conventional dendritic cells
Distinctive CD8+ T cell activation by antigen-presenting plasmacytoid dendritic cells compared to conventional dendritic cells Open
Plasmacytoid dendritic cells (pDCs) play a pivotal role in immune responses, particularly against viral infections. pDCs exhibit diverse functions, including interferon production, cytokine secretion, and antigen presentation. Here, we inv…
View article: Orthogonal transcriptional modulation and gene editing using multiple CRISPR-Cas systems
Orthogonal transcriptional modulation and gene editing using multiple CRISPR-Cas systems Open
View article: 417 Design of programmable immune reactive cells (PIRCs) with precision type I interferon and cytotoxicity response in solid tumors
417 Design of programmable immune reactive cells (PIRCs) with precision type I interferon and cytotoxicity response in solid tumors Open
View article: A simultaneous knockout knockin genome editing strategy in HSPCs potently inhibits CCR5- and CXCR4-tropic HIV-1 infection
A simultaneous knockout knockin genome editing strategy in HSPCs potently inhibits CCR5- and CXCR4-tropic HIV-1 infection Open
View article: CRISPR/Cas9 model of prostate cancer identifies Kmt2c deficiency as a metastatic driver by Odam/Cabs1 gene cluster expression
CRISPR/Cas9 model of prostate cancer identifies Kmt2c deficiency as a metastatic driver by Odam/Cabs1 gene cluster expression Open
Metastatic prostate cancer (PCa) poses a significant therapeutic challenge with high mortality rates. Utilizing CRISPR-Cas9 in vivo, we target five potential tumor suppressor genes (Pten, Trp53, Rb1, Stk11, and RnaseL) in the mouse prostat…
View article: SAM68 directs STING signaling to apoptosis in macrophages
SAM68 directs STING signaling to apoptosis in macrophages Open
View article: Single-cell transcriptome analysis of epithelial, immune, and stromal signatures and interactions in human ovarian cancer
Single-cell transcriptome analysis of epithelial, immune, and stromal signatures and interactions in human ovarian cancer Open
A comprehensive investigation of ovarian cancer (OC) progression at the single-cell level is crucial for enhancing our understanding of the disease, as well as for the development of better diagnoses and treatments. Here, over half a milli…
View article: CRISPR activation to characterize splice-altering variants in easily accessible cells
CRISPR activation to characterize splice-altering variants in easily accessible cells Open
View article: CRISPR/Cas9 model of prostate cancer identifies Kmt2c deficiency as a metastatic driver by Odam/Cabs1 gene cluster expression
CRISPR/Cas9 model of prostate cancer identifies Kmt2c deficiency as a metastatic driver by Odam/Cabs1 gene cluster expression Open
Prostate cancer (PCa) is the second leading cause of cancer-related deaths in men, and treatment options for metastatic disease remain limited. In this study, we employed Adeno-associated virus (AAV) delivery and CRISPR-Cas9 technology to …
View article: 442-E A novel targeted, type I interferon producing, innate lymphocyte therapy with potent anti-tumor activity
442-E A novel targeted, type I interferon producing, innate lymphocyte therapy with potent anti-tumor activity Open
Background Plasmacytoid Dendritic Cells (pDCs) - sometimes referred to as type I interferon producing innate lymphocyte cells - are known as a master regulator of the immune system. The pDCs are responsible for the body's Interferon (IFN) …
View article: Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi
Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi Open
View article: <scp>ER</scp> stress induces <scp>caspase‐2‐tBID‐GSDME</scp> ‐dependent cell death in neurons lytically infected with herpes simplex virus type 2
<span>ER</span> stress induces <span>caspase‐2‐tBID‐GSDME</span> ‐dependent cell death in neurons lytically infected with herpes simplex virus type 2 Open
View article: Dual intron-targeted CRISPR-Cas9-mediated disruption of the AML RUNX1-RUNX1T1 fusion gene effectively inhibits proliferation and decreases tumor volume in vitro and in vivo
Dual intron-targeted CRISPR-Cas9-mediated disruption of the AML RUNX1-RUNX1T1 fusion gene effectively inhibits proliferation and decreases tumor volume in vitro and in vivo Open
View article: Enrichment strategies to enhance genome editing
Enrichment strategies to enhance genome editing Open
View article: SAM68 directs STING signalling to apoptosis in macrophages
SAM68 directs STING signalling to apoptosis in macrophages Open
DNA is a danger signal and is sensed by cGAS to engage signaling through STING for activation of innate immune functions. The best-studied downstream responses to STING activation include expression of type I interferon and inflammatory ge…
View article: The p53 challenge of hematopoietic stem cell gene editing
The p53 challenge of hematopoietic stem cell gene editing Open
View article: Combined lineage tracing and scRNA-seq reveals unexpected first heart field predominance of human iPSC differentiation
Combined lineage tracing and scRNA-seq reveals unexpected first heart field predominance of human iPSC differentiation Open
During mammalian development, the left and right ventricles arise from early populations of cardiac progenitors known as the first and second heart fields, respectively. While these populations have been extensively studied in non-human mo…
View article: Editorial: CRISPR and beyond: Cutting-edge technologies for gene correction in therapeutic applications
Editorial: CRISPR and beyond: Cutting-edge technologies for gene correction in therapeutic applications Open
EDITORIAL article Front. Genome Ed., 20 April 2023Sec. Genome Editing in Human Health and Disease Volume 5 - 2023 | https://doi.org/10.3389/fgeed.2023.1203864
View article: Author response: Combined lineage tracing and scRNA-seq reveals unexpected first heart field predominance of human iPSC differentiation
Author response: Combined lineage tracing and scRNA-seq reveals unexpected first heart field predominance of human iPSC differentiation Open
View article: CRISPR-Cas-mediated transcriptional modulation: The therapeutic promises of CRISPRa and CRISPRi
CRISPR-Cas-mediated transcriptional modulation: The therapeutic promises of CRISPRa and CRISPRi Open