Publications by authors named "J Tolar"
Background: Almost all acquired severe aplastic anaemia is immune mediated and characterised by hypocellular bone marrow and ≥2 affected haematopoietic lineages. The optimal preparartive regimen for unrelated donor transplantation remains to be established. We aimed to study long-term outcomes after unrelated donor transplantation for severe aplastic anaemia with de-escalation of cyclophosphamide (Cy) dose in steps of 50 mg/kg (150, 100, 50, 0 mg/kg) in combination with total body irradiation (TBI) 2 Gy, anti-thymocyte globulin (ATG) and fludarabine.
View Article and Find Full Text PDFBackground: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genetic skin-blistering disorder that often progresses to metastatic cutaneous squamous cell carcinoma (cSCC) at chronic wound sites. Chondroitin sulfate proteoglycan 4 (CSPG4) is a cell-surface proteoglycan that is an oncoantigen in multiple malignancies, where it modulates oncogenic signalling, drives epithelial-to-mesenchymal transition (EMT) and enables cell motility.
Objectives: To evaluate CSPG4 expression and function in RDEB cSCC.
Article Synopsis
- Dyskeratosis congenita/telomere biology disorders (DC/TBD) often lead to bone marrow failure, and although allogeneic hematopoietic cell transplant (alloHCT) can help, traditional conditioning methods can cause significant toxicity.
- A clinical trial compared the outcomes of reduced intensity conditioning (RIC) alloHCT to a prior trial that included total body irradiation (TBI) in patients with DC/TBD, focusing on less toxic methods to ensure successful engraftment.
- While both groups demonstrated comparable outcomes in acute and chronic graft-versus-host disease and no primary graft failures, the non-TBI cohort showed slower donor chimerism achievement, indicating a balance between conditioning intensity and patient safety.
Methods for the targeted integration of genes in mammalian genomes suffer from low programmability, low efficiencies or low specificities. Here we show that phage-assisted continuous evolution enhances prime-editing-assisted site-specific integrase gene editing (PASSIGE), which couples the programmability of prime editing with the ability of recombinases to precisely integrate large DNA cargoes exceeding 10 kilobases. Evolved and engineered Bxb1 recombinase variants (evoBxb1 and eeBxb1) mediated up to 60% donor integration (3.
View Article and Find Full Text PDF