Human inborn errors of immunity: an expanding universe
Molecular, cellular, and clinical studies of human inborn errors of immunity have
revolutionized our understanding of their pathogenesis, considerably broadened their …
revolutionized our understanding of their pathogenesis, considerably broadened their …
Transcriptional and posttranslational regulation of Th17/Treg balance in health and disease
W Zhang, X Liu, Y Zhu, X Liu, Y Gu… - European Journal of …, 2021 - Wiley Online Library
Regulatory T (Treg) cells and T helper type 17 (Th17) cells play important roles in adaptive
immune responses, antagonizing each other in immune disorders. Th17/Treg balance is …
immune responses, antagonizing each other in immune disorders. Th17/Treg balance is …
High-yield genome engineering in primary cells using a hybrid ssDNA repair template and small-molecule cocktails
Enhancing CRISPR-mediated site-specific transgene insertion efficiency by homology-
directed repair (HDR) using high concentrations of double-stranded DNA (dsDNA) with …
directed repair (HDR) using high concentrations of double-stranded DNA (dsDNA) with …
Antigen-specific regulatory T cell therapy in autoimmune diseases and transplantation
C Selck, M Dominguez-Villar - Frontiers in immunology, 2021 - frontiersin.org
Regulatory T (Treg) cells are a heterogenous population of immunosuppressive T cells
whose therapeutic potential for the treatment of autoimmune diseases and graft rejection is …
whose therapeutic potential for the treatment of autoimmune diseases and graft rejection is …
Gene therapy for inborn errors of immunity: past, present and future
A Fischer - Nature Reviews Immunology, 2023 - nature.com
Inborn errors of immunity (IEI) are diseases caused by genetic mutations that affect the
immune system's ability to fight pathogens, cope with the microbiota or regulate …
immune system's ability to fight pathogens, cope with the microbiota or regulate …
Eras of designer Tregs: Harnessing synthetic biology for immune suppression
K Tuomela, K Salim, MK Levings - Immunological Reviews, 2023 - Wiley Online Library
Since their discovery, CD4+ CD25hiFOXP3hi regulatory T cells (Tregs) have been firmly
established as a critical cell type for regulating immune homeostasis through a plethora of …
established as a critical cell type for regulating immune homeostasis through a plethora of …
Therapeutic gene editing of T cells to correct CTLA-4 insufficiency
TA Fox, BC Houghton, L Petersone, E Waters… - Science Translational …, 2022 - science.org
Heterozygous mutations in CTLA-4 result in an inborn error of immunity with an autoimmune
and frequently severe clinical phenotype. Autologous T cell gene therapy may offer a cure …
and frequently severe clinical phenotype. Autologous T cell gene therapy may offer a cure …
[HTML][HTML] Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression
Background Chimeric antigen receptor (CAR) T cells have demonstrated high clinical
response rates against hematological malignancies (eg, CD19+ cancers) but have shown …
response rates against hematological malignancies (eg, CD19+ cancers) but have shown …
[HTML][HTML] The power and the promise of CRISPR/Cas9 genome editing for clinical application with gene therapy
N Guo, JB Liu, W Li, YS Ma, D Fu - Journal of Advanced Research, 2022 - Elsevier
Abstract Background Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)
is derived from the bacterial innate immune system and engineered as a robust gene-editing …
is derived from the bacterial innate immune system and engineered as a robust gene-editing …
Identification of unstable regulatory and autoreactive effector T cells that are expanded in patients with FOXP3 mutations
Studies of the monogenic autoimmune disease immunodysregulation polyendocrinopathy
enteropathy X-linked syndrome (IPEX) have elucidated the essential function of the …
enteropathy X-linked syndrome (IPEX) have elucidated the essential function of the …