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Landmark gene therapy study shows safety for children

A landmark study published in the New England Journal of Medicine reports the long-term safety and efficacy of gene therapy for children with ADA-SCID, a rare immune disorder. The treatment resulted in a 100% survival rate and over 95% cure rate, with patients able to respond to routine childhood vaccinations.

SourceUniversity College London·JournalNew England Journal of Medicine·TypeObservational study·DateOct 15, 2025

IL2RG gene therapy for X-SCID

Researchers have developed a new gene therapy protocol using the SIN-EFS-IL2RG.co vector, which demonstrates safety and efficacy in preclinical studies. The treatment restores immune functions and lacks oncogenicity, paving the way for further clinical trials in X-SCID patients.

SourceCompuscript Ltd·JournalGenes & Diseases·DateMar 13, 2025

International Alliance for Primary Immunodeficiency Societies selects Rockefeller University Press to publish new Journal of Human Immunity

The International Alliance for Primary Immunodeficiency Societies has partnered with Rockefeller University Press to launch the Journal of Human Immunity, an open-access journal focused on human immunity and inborn errors of immunity. The journal aims to provide a platform for groundbreaking research and attract top-tier submissions.

Better diagnosis and treatment of cryptococcosis

The new guideline aims to improve diagnosis and treatment of cryptococcosis, a fatal fungal infection that affects mainly the lungs and brain. It provides practical guidance for medical staff to recognize and manage invasive fungal infections, with the goal of improving patient survival rates.

SourceUniversity of Cologne·JournalThe Lancet Infectious Diseases·TypeObservational study·DateFeb 13, 2024

A long-acting biologic with transmucosal transport properties that arrest SARS-CoV-2 virus variants

A long-acting biologic with transmucosal transport properties has been developed to block cellular infection of all SARS-CoV-2 variants. The biologic utilizes a soluble recombinant human ACE2 protein fused to an engineered human albumin variant, offering improved pharmacokinetic properties and delivery across selective barriers.

Journal of Clinical Immunology publishes Octapharma USA research on cutaquig® evaluating PI treatment options

A recent study published in the Journal of Clinical Immunology suggests that cutaquig infusions at higher infusion rates and volumes are well-tolerated and effective, with reduced infusion time, fewer injection sites, and improved compliance. The study results will be presented at the Clinical Immunology Society 2023 Annual Meeting.

SourceYankee Public Relations·JournalJournal of Clinical Immunology·TypeObservational study·DateMay 18, 2023

UCLA-led study uses base editing to correct mutation that causes rare immune deficiency

Researchers at UCLA successfully used base editing to correct a mutation causing rare immune deficiency CD3 delta SCID. The treatment corrected an average of 71% of patient stem cells and allowed them to produce fully functional T cells, suggesting long-term persistence of corrected blood stem cells.

SourceUniversity of California - Los Angeles Health Sciences·JournalCell·TypeExperimental study·DateMar 20, 2023

Cancer patients treated with immunotherapy can safely receive mRNA COVID-19 vaccines, according to JNCCN study

Researchers analyzed 408 patients receiving immune checkpoint inhibitor therapy and found no increased risk of side effects from receiving both immunotherapy and the vaccine. The study supports NCCN's recommendations for COVID-19 vaccination in people with cancer, citing strong protection against severe COVID-19 for all variants.

SourceNational Comprehensive Cancer Network·JournalJournal of the National Comprehensive Cancer Network·DateOct 17, 2022

Genetic test can diagnose certain immune system disorders

Researchers developed a genetic test that diagnoses primary immunodeficiency disorders (PID), revealing inherited genetic defects in nearly half of patients. The test uses next-generation sequencing technology to identify specific gene variants associated with PID, enabling targeted treatment and earlier intervention for family members.

SourceElsevier·JournalJournal of Molecular Diagnostics·TypeExperimental study·DateMay 23, 2022

UTMB study shows common antibody therapy has anti-SARS-CoV-2 Antibodies

Researchers at UTMB confirmed the presence of neutralizing antibodies to SARS-CoV-2 in a commonly used immunoglobulin product, providing protective benefits to immunocompromised patients. The study's findings have value for future immunoglobulin-based modalities and reduce anxiety about infection or exposure to the virus.

SourceUniversity of Texas Medical Branch at Galveston·JournalJournal of Allergy and Clinical Immunology·TypeExperimental study·DateApr 25, 2022

Researchers identify sustainable source of immunodeficiency virus-resistant immune cells

Researchers have successfully generated large numbers of virus-resistant immune cells from monkeys using CRISPR/Cas9 gene editing. This breakthrough could lead to the development of a new treatment for HIV/AIDS by providing an alternative to current therapies that require lifelong medication and can cause side effects.

SourceInternational Society for Stem Cell Research·JournalStem Cell Reports·DateMar 31, 2022

From the god of sun to immunodeficiency: international research team identifies new rare disease affecting hematopoiesis and immunity

An international research team discovered a new rare disease caused by a disrupted Helios-dependent epigenetic regulation mechanism, leading to T and B cell defects. The study highlights the importance of Helios in immune homeostasis and suggests potential therapeutic targets for immunodeficiency and malignancy.

SourceSt. Anna Children's Cancer Research Institute·JournalScience Immunology·TypeExperimental study·DateNov 29, 2021

New test for rare immunodeficiency

Researchers at the University of Basel developed a rapid test to diagnose Sp110 protein deficiency, a severe immune defect. The test uses flow cytometry to detect the presence of Sp110 protein in patient blood cells, enabling quick diagnosis in hours.

SourceUniversity of Basel·JournalJournal of Clinical Immunology·DateAug 22, 2017

Cause of viral infection of the brain mapped out

A study published in the Journal of Experimental Medicine has identified a genetic defect in the immune system that can lead to fatal brain inflammation in some people infected with the herpes virus. The discovery may also shed light on other types of viral infections, such as meningitis and influenza.

SourceAarhus University·JournalJournal of Experimental Medicine·DateOct 20, 2015

Identified the epigenetic basis of CVID through the study of identic twins

Researchers identified epigenetic alterations in CVID patients by comparing monozygotic twins. They found higher DNA methylation levels and impaired DNA demethylation in immunodeficient B cells, leading to reduced antibody production and altered cell maturation. These findings provide new insights into the diagnosis and treatment of CVID.

SourceIDIBELL-Bellvitge Biomedical Research Institute·JournalNature Communications·DateJun 17, 2015

JCI table of contents: May 10, 2007

Researchers found that a gene deficiency in CalDAG-GEFI may explain the loss of platelet function leading to recurrent bleeding in patients with leukocyte adhesion deficiency III. Additionally, IL-33 and ST2 signaling was identified as a critical mechanism for protecting the heart from stress, suggesting potential new therapeutic targets.

SourceJCI Journals·JournalJournal of Clinical Investigation·DateMay 10, 2007

Infusion nurses examine need for standards of practice in immunoglobulin therapy

The need for consensus on IVIG dosing and frequency arises as its use expands, with infusion nurses playing a crucial role in achieving best results from therapy. The article highlights the importance of developing guidelines that maximize patient benefit and minimize risk, given the unique response to different IVIG products.

SourceEdelman Public Relations, New York·JournalJournal of Infusion Nursing·DateAug 12, 2005

Artemis: a little bit is not enough

Individuals with reduced functional Artemis protein are prone to mild immunodeficiency and increased risk of developing lymphomas. This finding suggests that mutations in Artemis or other DNA repair genes may be responsible for immune deficiency and/or lymphoma cases.

SourceJCI Journals·JournalJournal of Clinical Investigation·DateFeb 4, 2003