A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells
Translation of the CRISPR–Cas9 system to human therapeutics holds high promise. However,
specificity remains a concern especially when modifying stem cell populations. We show …
specificity remains a concern especially when modifying stem cell populations. We show …
Tuning the antigen density requirement for CAR T-cell activity
…, RM Richards, S Heitzeneder, SM Nguyen, V Wiebking… - Cancer discovery, 2020 - AACR
Optimal CAR T-cell activity is dependent on antigen density, which is variable in many
cancers, including lymphoma and solid tumors. CD28ζ-CARs outperform 4-1BBζ-CARs when …
cancers, including lymphoma and solid tumors. CD28ζ-CARs outperform 4-1BBζ-CARs when …
[HTML][HTML] Gene correction for SCID-X1 in long-term hematopoietic stem cells
M Pavel-Dinu, V Wiebking, BT Dejene, W Srifa… - Nature …, 2019 - nature.com
Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective
therapy for monogenic diseases of the blood and immune system. Here we describe an …
therapy for monogenic diseases of the blood and immune system. Here we describe an …
[PDF][PDF] Highly efficient and marker-free genome editing of human pluripotent stem cells by CRISPR-Cas9 RNP and AAV6 donor-mediated homologous recombination
…, J Camarena, M Pavel-Dinu, C Sindhu, V Wiebking… - Cell stem cell, 2019 - cell.com
Genome editing of human pluripotent stem cells (hPSCs) provides powerful opportunities
for in vitro disease modeling, drug discovery, and personalized stem cell-based therapeutics. …
for in vitro disease modeling, drug discovery, and personalized stem cell-based therapeutics. …
[HTML][HTML] Genome editing of donor-derived T cells to generate allogeneic chimeric antigen receptor-modified T cells: optimizing αβ T-cell-depleted haploidentical …
Allogeneic hematopoietic stem cell transplantation is an effective therapy for high-risk leukemias.
In children, graft manipulation based on the selective removal of αβT cells and B cells …
In children, graft manipulation based on the selective removal of αβT cells and B cells …
Metabolic engineering generates a transgene-free safety switch for cell therapy
V Wiebking, JO Patterson, R Martin, MK Chanda… - Nature …, 2020 - nature.com
Safeguard mechanisms can ameliorate the potential risks associated with cell therapies but
currently rely on the introduction of transgenes. This limits their application owing to …
currently rely on the introduction of transgenes. This limits their application owing to …
Impairment of endothelial progenitor cell function and vascularization capacity by aldosterone in mice and humans
…, K Schmitter, F Fleissner, V Wiebking… - European heart …, 2011 - academic.oup.com
Aims Hyperaldosteronism is associated with vascular injury and increased cardiovascular
events. Bone marrow-derived endothelial progenitor cells (EPCs) play an important role in …
events. Bone marrow-derived endothelial progenitor cells (EPCs) play an important role in …
[HTML][HTML] T-cell replete haploidentical bone marrow transplantation and post-transplant cyclophosphamide for patients with inborn errors
M Kurzay, F Hauck, I Schmid, V Wiebking… - …, 2019 - ncbi.nlm.nih.gov
Matched donor hematopoietic stem cell transplantation (HSCT) cures most patients with
inborn hematopoietic disorders, but haploidentical HSCT was historically associated with graft …
inborn hematopoietic disorders, but haploidentical HSCT was historically associated with graft …
Subcutaneous mosunetuzumab is active with a manageable safety profile in patients (pts) with relapsed/refractory (R/R) B-cell non-Hodgkin lymphomas (B-NHLs) …
…, M Dreyling, DH Yoon, G Hess, J Radford, V Wiebking… - Blood, 2022 - ashpublications.org
Background: Mosunetuzumab (Mosun), a CD20xCD3 T-cell engaging bispecific monoclonal
antibody (Bi-mAb) that redirects T cells to eliminate malignant B cells, is the first Bi-mAb …
antibody (Bi-mAb) that redirects T cells to eliminate malignant B cells, is the first Bi-mAb …
Reduced toxicity, myeloablative HLA-haploidentical hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for sickle cell disease
V Wiebking, S Hütker, I Schmid, S Immler… - Annals of …, 2017 - Springer
Allogeneic hematopoietic stem cell transplantation (HSCT) offers the possibility of cure for
sickle cell disease (SCD) patients. Unfortunately, the probability of finding an HLA-matched …
sickle cell disease (SCD) patients. Unfortunately, the probability of finding an HLA-matched …