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Open AccessNon-osteopenic Bone Pathology After Allo-hematopoietic Stem Cell Transplantation in Patients with Inborn Errors of Immunity
There is a lack of data on post-HSCT non-osteopenic bone pathology specifically for children with inborn errors of immunity (IEI). We collected data on non-osteopenic bone pathology in children with IEI post-H...
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Off-the-Shelf Allogeneic CAR-T Cells or Other Immune Effector Cells
“Off-the-shelf” allogeneic CAR TCRab T cells and other immune effector cells, such as natural killer (NK) or gamma delta (gd) T cells, can be premanufactured from healthy donors and may offer alternatives to a...
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Off-the-Shelf CAR-T
The FDA has approved CAR-T cell therapy for the treatment of B cell malignancies. Despite the clinical success of CD19 targeted CAR-T, several barriers limit the wider adoption of CAR-based therapies as viable...
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Article
Open AccessBase-edited CAR T cells for combinational therapy against T cell malignancies
Targeting T cell malignancies using chimeric antigen receptor (CAR) T cells is hindered by ‘T v T’ fratricide against shared antigens such as CD3 and CD7. Base editing offers the possibility of seamless disrup...
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Article
Open AccessMetformin hydrochloride entrapment in sorbitan monostearate for intestinal permeability enhancement and pharmacodynamics
Penetration enhancement of metformin hydrochloride via its molecular dispersion in sorbitan monostearate microparticles is reported. This represents basic philosophy to maximize its entrapment for maximum pene...
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Outcome of Non-hematological Autoimmunity After Hematopoietic Cell Transplantation in Children with Primary Immunodeficiency
Knowledge of post-hematopoietic cell transplantation (HCT) non-hematological autoimmune disease (AD) is far from satisfactory.
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Article
‘Mini’ U6 Pol III promoter exhibits nucleosome redundancy and supports multiplexed coupling of CRISPR/Cas9 effects
RNA polymerase III (Pol III) promoters express short non-coding RNAs and have been adopted for expression of microRNA, interference RNA, and CRISPR single guide RNA (sgRNA). Vectors incorporating H1 and U6 Pol...
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Article
Open AccessCD70 expression determines the therapeutic efficacy of expanded human regulatory T cells
Regulatory T cells (Tregs) are critical mediators of immune homeostasis. The co-stimulatory molecule CD27 is a marker of highly suppressive Tregs, although the role of the CD27-CD70 receptor-ligand interaction...
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Hematopoietic stem cell transplantation for cytidine triphosphate synthase 1 (CTPS1) deficiency
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Open AccessGenome-Edited T Cell Therapies
Alternative approaches to conventional drug-based cancer treatments have seen T cell therapies deployed more widely over the last decade. This is largely due to their ability to target and kill specific cell t...
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Article
Open AccessDeep sequencing reveals persistence of cell-associated mumps vaccine virus in chronic encephalitis
Routine childhood vaccination against measles, mumps and rubella has virtually abolished virus-related morbidity and mortality. Notwithstanding this, we describe here devastating neurological complications ass...
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Article
Variable Phenotype of Severe Immunodeficiencies Associated with RMRP Gene Mutations
Mutations in RMRP primarily give rise to Cartilage Hair Hypoplasia (CHH), a highly diverse skeletal disorder which can be associated with severe immunodeficiency. Increased availability of RMRP mutation screening...
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Article
Open AccessNovel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases
TAL-effector nucleases (TALENs) are attractive tools for sequence-specific genome modifications, but their delivery still remains problematic. It is well known that the presence of multiple sequence repeats in...
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Article
Gene Therapy for Primary Immunodeficiencies: Current Status and Future Prospects
Gene therapy using autologous haematopoietic stem cells offers a valuable treatment option for patients with primary immunodeficiencies who do not have access to an HLA-matched donor, although such treatments ...
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Christopher Baum: Genetic modification of haematopoietic stem cells: methods and protocols
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RISC control for gene therapy
A new approach to reducing the immunological side effects of gene therapy exploits microRNA to knock down transgene expression in immune cells.
