Severe Combined Immunodeficiency News and Research

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NIH Researchers Map Vulnerable Sites on Epstein-Barr Virus gp42 Protein

Studies of interactions between two lab-generated monoclonal antibodies (mAbs) and an essential Epstein-Barr virus (EBV) protein have uncovered targets that could be exploited in designing treatments and vaccines for this extremely common virus.

13 Mar 2024

Effectiveness of SCID-X1 Gene Therapy Influenced by 3D Genome Architecture

Patients with X-linked severe combined immunodeficiency disorder (SCID-X1), often known as “bubble boy disease,” are born with a faulty gene that inhibits immune cells from being produced.

9 Oct 2023

New Proof-of-Concept Model for Delivering Gene Editing Tools to Treat Blood Disorders

In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

28 Jul 2023

Pioneering gene therapy has long-lasting effects on children with deadly immune disorder

Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects of the therapy appear to be long-lasting, with 90% of patients who received the treatment eight to 11 years ago still disease-free.

18 Oct 2021