Severe Combined Immunodeficiency News and Research

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New gene editing approach could revolutionize the treatment of 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.

27 Jul 2023

Newborn screening for "bubble-baby" disease boosts survival of children

Screening newborns for severe combined immunodeficiency disease (SCID) significantly increases the survival of children after bone marrow transplantation, a new North American study finds.

21 Jun 2023

Newborn screening facilitates early identification of infants with severe combined immunodeficiency

Introducing widespread screening of newborns for a deadly disease called severe combined immunodeficiency, or SCID, followed by early treatment boosted the five-year survival rate of children with the disorder from 73% before the advent of screening to 87% since, researchers report.

20 Jun 2023

Avanced genome editing technology could be used as a one-time treatment for CD3 delta SCID

A new UCLA-led study suggests that advanced genome editing technology could be used as a one-time treatment for the rare and deadly genetic disease CD3 delta severe combined immunodeficiency.

21 Mar 2023

Study shows vaccine-derived poliovirus in infant with severe immunodeficiency

Researchers identified type 3 vaccine-derived poliovirus (VDPV) in an infant.

13 Sep 2022

The QCOVID4 algorithm developed using data from the Omicron wave identifies those at highest risk of severe COVID-19 outcomes

A recent article illustrated the derivation and validation of the QCOVID4 risk determination algorithm for predicting COVID-19 hospitalization and death risk in England.

18 Aug 2022

A polymer-nanoparticle hydrogel to subcutaneously deliver broadly neutralizing antibodies against SARS-CoV-2

Researchers compared the polymer-nanoparticle hydrogel system and standard routes of delivery of broadly neutralizing antibodies against SARS-CoV-2 in a mouse model.

27 May 2022

Exploring the potential utility of calcium channel inhibitors in SARS-CoV-2 infection

In a new study, researchers discuss the role of calcium signaling pathways as targets of inhibition in potential new antiviral therapeutic pathways for SARS-CoV-2.

20 Jan 2022

Researchers report long-lasting effects of gene therapy in 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.

18 Oct 2021

Researchers identify novel disorder resulting from a mutation in AIOLOS protein

Primary immunodeficiencies, such as severe combined immunodeficiency disease (SCID), occur when the immune system does not work properly, leading to increased susceptibility to various infections, autoimmunity, and cancers.

16 Jul 2021

UCSF launches new initiative to propel the development of living therapeutics

UC San Francisco is launching a new initiative to propel the development of living therapeutics - a category of treatments broadly defined as human and microbial living cells that are selected, modified, or engineered to treat or cure disease - and bring them quickly to patients.

14 Jun 2021

Investigational gene therapy can restore immune function in children with inherited immunodeficiency disorder

An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National Institutes of Health.

12 May 2021

Experimental gene therapy successfully treats children born without an immune system

An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves them without an immune system.

11 May 2021

Scientists launch clinical trial of CRISPR gene correction therapy in patients with sickle cell disease

Scientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patient's own blood-forming stem cells.

30 Mar 2021

New test will help better manage patients with severe combined immunodeficiency

A new test developed at CHU Sainte-Justine in Montreal will enable better management of patients with severe combined immunodeficiency.

17 Jun 2020

Research team generates comprehensive developmental map of human T-cells

Chintan Parekh, MD, of The Saban Research Institute of Children's Hospital Los Angeles, has led a team of investigators that generated a comprehensive roadmap for how T-cells develop in the human thymus.

16 Jun 2020

COVID-19 and immunodeficiency

COVID-19 disease, caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), is a highly infectious disease. As of today, over 170,000 people worldwide have succumbed to the infection. Reports have shown that some comorbidities and illnesses raise the risk of getting infected and also influence the severity of the disease. Having a compromised immune system can also drastically raise the risk of getting COVID-19.

21 Apr 2020