EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity
AC Lankester, MH Albert, C Booth… - Bone Marrow …, 2021 - nature.com
Inborn errors of immunity (IEI) are a group of rare heterogeneous diseases. Currently, more
than 400 monogenetic IEI have been identified and increasingly a genetic diagnosis can be …
than 400 monogenetic IEI have been identified and increasingly a genetic diagnosis can be …
Type 1 diabetes
T Quattrin, LD Mastrandrea, LSK Walker - The Lancet, 2023 - thelancet.com
Type 1 diabetes is a chronic disease caused by autoimmune destruction of pancreatic β
cells. Individuals with type 1 diabetes are reliant on insulin for survival. Despite enhanced …
cells. Individuals with type 1 diabetes are reliant on insulin for survival. Despite enhanced …
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 …
Human inborn errors of immunity: 2019 update on the classification from the International Union of Immunological Societies Expert Committee
We report the updated classification of Inborn Errors of Immunity/Primary
Immunodeficiencies, compiled by the International Union of Immunological Societies Expert …
Immunodeficiencies, compiled by the International Union of Immunological Societies Expert …
A genetic mouse model recapitulates immune checkpoint inhibitor–associated myocarditis and supports a mechanism-based therapeutic intervention
Immune checkpoint inhibitors (ICI) targeting CTLA4 or PD-1/PD-L1 have transformed cancer
therapy but are associated with immune-related adverse events, including myocarditis …
therapy but are associated with immune-related adverse events, including myocarditis …
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 …
Moving towards personalized treatments of immune-related adverse events
K Esfahani, A Elkrief, C Calabrese, R Lapointe… - Nature reviews Clinical …, 2020 - nature.com
The enhancement of immune responses upon treatment with immune checkpoint inhibitors
can have the desired outcome of reinvigorating antitumour immune surveillance, but often at …
can have the desired outcome of reinvigorating antitumour immune surveillance, but often at …
A unified metric of human immune health
R Sparks, N Rachmaninoff, WW Lau, DC Hirsch… - Nature Medicine, 2024 - nature.com
Immunological health has been challenging to characterize but could be defined as the
absence of immune pathology. While shared features of some immune diseases and the …
absence of immune pathology. While shared features of some immune diseases and the …
Regulatory T cells: the many faces of Foxp3
Regulatory T (Treg) cells expressing the transcription factor forkhead box P3 (Foxp3) play a
requisite role in the maintenance of immunological homeostasis and prevention of …
requisite role in the maintenance of immunological homeostasis and prevention of …
Hematopoietic stem cell transplantation in primary immunodeficiency diseases: current status and future perspectives
Primary immunodeficiencies (PID) are disorders that for the most part result from mutations
in genes involved in immune host defense and immunoregulation. These conditions are …
in genes involved in immune host defense and immunoregulation. These conditions are …