Thomas Wisbech Skov
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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: Engineered lentivirus-derived nanoparticles (LVNPs) for delivery of CRISPR/Cas ribonucleoprotein complexes supporting base editing, prime editing and <i>in vivo</i> gene modification
Engineered lentivirus-derived nanoparticles (LVNPs) for delivery of CRISPR/Cas ribonucleoprotein complexes supporting base editing, prime editing and <i>in vivo</i> gene modification Open
Implementation of therapeutic in vivo gene editing using CRISPR/Cas relies on potent delivery of gene editing tools. Administration of ribonucleoprotein (RNP) complexes consisting of Cas protein and single guide RNA (sgRNA) offers short-li…
View article: Resistance to vincristine in <scp>DLBCL</scp> by disruption of p53‐induced cell cycle arrest and apoptosis mediated by <scp>KIF18B</scp> and <scp>USP28</scp>
Resistance to vincristine in <span>DLBCL</span> by disruption of p53‐induced cell cycle arrest and apoptosis mediated by <span>KIF18B</span> and <span>USP28</span> Open
Summary The frontline therapy R‐CHOP for patients with diffuse large B‐cell lymphoma (DLBCL) has remained unchanged for two decades despite numerous Phase III clinical trials investigating new alternatives. Multiple large studies have unco…
View article: Resistance to vincristine in cancerous B-cells by disruption of p53-dependent mitotic surveillance
Resistance to vincristine in cancerous B-cells by disruption of p53-dependent mitotic surveillance Open
The frontline therapy R-CHOP for patients with diffuse large B-cell lymphoma (DLBCL) has remained unchanged for two decades despite numerous phase III clinical trials investigating new alternatives. Multiple large studies have uncovered ge…
View article: CRISPR-Cas9-directed gene tagging using a single integrase-defective lentiviral vector carrying a transposase-based Cas9 off switch
CRISPR-Cas9-directed gene tagging using a single integrase-defective lentiviral vector carrying a transposase-based Cas9 off switch Open