Severe Combined Immunodeficiency News and Research

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Transplanted human cortical organoids filled most of a mouse cortex. Then they began to connect

Researchers created xenocortical mice by transplanting human cortical organoids into animals genetically depleted of much of their native cortex, allowing the human tissue to occupy most of the available cortical space and form anatomical connections throughout the host nervous system. The grafts developed diverse cortical cell types, including L5-ET and VEN-like neurons, generated organized neural activity, and produced measurable cellular and behavioral responses to hypoxic injury.

18 Sep 2026

Researchers urge caution over large-scale newborn genome screening

New research from large population studies provides invaluable evidence on genome screening of newborn babies to reduce risks from overdiagnosis.

15 Jun 2026

New SMArT platform improves safety of CRISPR gene editing

A team of researchers led by Luigi Naldini at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) has developed a new strategy to significantly improve the precision and safety of CRISPR-Cas9 gene editing in human blood stem cells, potentially overcoming one of the major barriers limiting broader clinical application of genome editing therapies.

1 Jun 2026

FDA approves first gene therapy for rare pediatric immune disorder

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years.

27 Mar 2026

Experimental gene therapy restores immune system function in children with genetic immune disorder

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained immune system function in 59 of 62 children born with ADA-SCID, a rare and deadly genetic immune disorder.

15 Oct 2025

Gene therapy shows long-term success in children with rare immune disorder

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by a UCL researcher.

15 Oct 2025

Investigation raises concerns over tooth stem cell banking claims

Parents are spending thousands of pounds to bank stem cells from their children's milk teeth – but the recipient companies' claims about their future medical value are unproven and potentially misleading, reveals an investigation by The BMJ, published today.

21 Aug 2025

Virtual memory T cells provide nonspecific immunity for infants early in life

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life.

31 Jul 2025

Study explores how a familiar spice tackles the toughest cancer cells

Researchers found that clinically achievable doses of curcumin, the active compound in turmeric, target and suppress colorectal cancer stem-like cells, especially in early adenomas. Curcumin shifts these cells toward differentiation, delays tumor growth in mice, and may offer a safe preventive strategy.

27 Jun 2025

Study identifies C5aR1 as a key marker of metastasis in skin cancer

Researchers have identified C5aR1 as a novel biomarker for metastasis risk and poor prognosis in patients with cutaneous squamous cell carcinoma (cSCC), the most common type of metastatic skin cancer.

29 May 2025

New study confirms CAR T cell therapy does not cause secondary cancers

Within a large group of more than 700 patients treated with CAR T cell therapy, researchers found no evidence that the therapy itself caused any type of secondary cancer in the modified T cells, according to new analysis reported today in Nature Medicine from the Perelman School of Medicine at the University of Pennsylvania and Penn Medicine's Abramson Cancer Center.

3 Feb 2025

DNA analysis outperforms standard newborn screening, study shows

Early results from a study of newborn screening methods show that DNA analysis picks up many more preventable or treatable serious health conditions than standard newborn screening and is favored by most parents who are offered the option.

24 Oct 2024

Perceived stress can directly trigger psoriasis relapse

Innovative research has provided compelling evidence that perceived stress can directly trigger the relapse of psoriatic skin lesions. The study, presented today at the European Academy of Dermatology and Venereology (EADV) Congress 2024, is the first to scientifically validate this connection in vivo.

26 Sep 2024

PDX engraftment predicts high recurrence and poor survival in triple-negative breast cancer

Researchers examine the association between patient-derived xenograft with recurrence and survival rates in triple negative breast cancer patients.

11 Sep 2024

Scientists identify genetic cause of rare, life-threatening immunodeficiency disorders in children

Scientists have pinpointed genetic changes that can leave children born with little to no immune defence against infection.

3 Jun 2024

NIH study uncovers vulnerable sites on Epstein-Barr virus 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.

12 Mar 2024

CRISPR cures on the horizon: Breakthrough center aims to heal rare, deadly genetic diseases

Children and adults with rare, deadly genetic diseases have fresh hope for curative therapies, thanks to a new collaboration between the Innovative Genomics Institute (IGI) and Danaher Corporation, a global life sciences and diagnostics innovator.

9 Jan 2024

Proof-of-concept study holds promise for safer gene therapy techniques for treating immunodeficiencies

Researchers described an approach to edit the recombination-activating gene 2 (RAG2) locus for therapeutic applications.

1 Nov 2023

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