Bone Marrow Transplant News and Research

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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

Innovative screening tool launched to identify individuals at risk for hereditary cancers

Ochsner Health announces the launch of its Genetic Wellness Assessment, an innovative screening tool to help identify individuals at risk for hereditary cancers.

9 Oct 2025

Newborn genomic screening may detect hundreds of additional childhood conditions

Adding genomic sequencing to newborn blood screening would detect hundreds of additional childhood conditions, providing much earlier diagnosis and treatment, according to a new study. A baby's genome, which stays with them for life, could also be re-examined if a health issue arises during their lifetime.

9 Oct 2025

Targeted microglia replacement shows promise against fatal neurodegenerative conditions

Tay-Sachs and Sandhoff diseases, genetic disorders affecting the brain, have no effective treatment and are typically fatal within the first years of life.

6 Aug 2025

Many patients with high-risk myelodysplastic syndromes do not receive guideline-recommended treatment

Most patients with high-risk myelodysplastic syndromes (MDS) do not receive guideline-recommended treatment with hypomethylating agents (HMAs), according to results published in Blood Neoplasia.

6 Aug 2025

UCLA study shows stem cells can be reprogrammed to fight cancer

In a first-of-its-kind clinical trial, UCLA scientists have shown it's possible to reprogram a patient's blood-forming stem cells to generate a continuous supply of functional T cells, the immune system's most powerful cancer-killing agents.

4 Aug 2025

New approach makes mismatched stem cell transplants safer for blood cancer patients

Advances in blood stem cell transplants now make it possible for people with blood cancers to get safe and effective "mismatched" transplants that will potentially cure their disease, new UVA Cancer Center research reveals. The advances will allow far more people to receive the lifesaving treatment.

15 Jul 2025

New immunotherapy targets leukemia at its root

Researchers at MUSC Hollings Cancer Center have identified a signaling loop involved in the growth and persistence of leukemia cells – and developed a novel immunotherapy that can disrupt that loop to boost immune function and improve survival.

14 Jul 2025

New app can help improve quality of life for caregivers of bone marrow transplant patients

A new study by investigators from Mass General Brigham showed that a new app they created can help improve the quality of life for caregivers of patients undergoing bone marrow transplant (BMT).

30 May 2025

Anal cancer rates increasing among older white and Hispanic women in the U.S.

Anal cancer has been steadily increasing in the United States, with the biggest jumps among older women, especially white and Hispanic women - a shift that challenges assumptions about high-risk groups and who should be screened, according to a study to be presented today at Digestive Disease Week 2025.

5 May 2025

Hydroxyurea shows long-term benefits for children with sickle cell disease

Hydroxyurea remains effective long-term in reducing emergency department visits and hospital days for children living with sickle cell disease (SCD), according to new research published in Blood Advances.

17 Apr 2025

Pioneering the world’s first CRISPR medicine for sickle cell disease

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters was with a 24-year-old patient whose sickle cell disease left them with almost weekly pain episodes.

3 Mar 2025

UCSF study reveals lungs as source of hematopoietic stem cells

Red blood cells carry oxygen from the lungs to every other organ, and blood-forming stem cells must make about 200 billion new red blood cells each day to keep the oxygen flowing.

28 Feb 2025

Huntsman Cancer Institute joins innovative clinical trials program for blood cancers

Huntsman Cancer Institute at the University of Utah (the U) has joined other institutions in an innovative clinical trials program designed to match patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) with a clinical trial specifically designed for the genetic signature of their disease.

17 Dec 2024

First U.S. trial uses non-viral CRISPR to correct sickle cell mutation

UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease.

26 Nov 2024

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

Genetic insights transform treatment for children with developmental disorders

Thousands of children with severe developmental disorders have benefited from more targeted treatments and support with genetic insights from the large-scale Deciphering Developmental Disorders (DDD) study, finds a new study.

15 Oct 2024

New cell therapy offers effective treatment for patients with T-cell acute lymphoblastic leukemia

A new cell therapy, targeting CD7 on leukaemia cells, gives a potentially effective treatment for patients with T-cell acute lymphoblastic leukaemia (T-ALL) who have exhausted all standard treatment options.

7 Oct 2024

Frailty linked to diminished quality of life in cancer and transplant survivors

A new research paper was published on the in Aging (listed by MEDLINE/PubMed as "Aging (Albany NY)" and "Aging-US" by Web of Science), Volume 16, Issue 18 on September 26, 2024, entitled, "Frailty and pre-frailty associated with long-term diminished physical performance and quality of life in breast cancer and hematopoietic cell transplant survivors."

2 Oct 2024