A partnership between St. Jude and Mustang Bio may change the challenges faced by those diagnosed with what is commonly referred to as “bubble boy disease.” A partnership between St. Jude and Mustang ...

The second edition of the only comprehensive textbook on inherited disorders of the immune system, co-edited by UCSF Professor of Pediatrics and Human Genetics Jennifer Puck, MD, will soon be ...

The cytokine receptor common gamma chain (γc) is shared among the receptors for interleukin (IL)-2, -4, -7, -9, -15, and plays a pivotal role in transmitting these cytokine signals. 1,2 Many of these ...

Several infants treated with autologous ex vivo gene therapy for newly diagnosed X-linked severe combined immunodeficiency (X-SCID) in a phase I/II trial have developed fully functioning immune ...

X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in T-cell, B-cell, and natural killer (NK) cell function, caused by mutations in ...

New animal studies conducted at the Salk Institute for Biological Studies show that the only human gene therapy treatment to date considered to be largely successful, is, in fact, riskier than ...

A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune ...

Severe Combined Immunodeficiency (SCID) is a rare, life-threatening immunological disorder characterized due to the lack of B-lymphocyte and T-lymphocyte functioning. Sporadic (occasional) ...

SCID is a genetic disorder impacting the immune system, making children highly susceptible to severe infections and illnesses, and without treatment, it can be fatal within the first 2 years of life.

La Jolla, CA – New animal studies conducted at the Salk Institute for Biological Studies show that the only human gene therapy treatment to date considered to be largely successful, is, in fact, ...