Beta Thalassemia News and Research

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Beta-thalassemia (ß-thalassemia) is a form of thalassemia due to mutations in the HBB gene on chromosome 11 , inherited in an autosomal recessive fashion.

New base editing techniques accurately modify genes in human embryos

A study by researchers at Columbia University Vagelos College of Physicians and Surgeons has found that new cutting-edge techniques can accurately edit genes in human embryos-giving scientists indispensable tools for understanding normal human development-but has also uncovered important risks that currently preclude the use of the techniques in the clinic.

9 Sep 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

Diatron introduces advanced RUO indices on its Aquila, Aquarius 3, Abacus 5 analyzers

Diatron, a STRATEC brand specializing in the development, manufacturing, and marketing of hematology and clinical chemistry analyzers and related reagents for human and veterinary use, expands the capabilities of its Aquila, Aquarius 3, and Abacus 5 hematology analyzers with six advanced hematological indices.

From Diatron 29 Apr 2026

Disrupted iron levels after SARS-CoV-2 infection may trigger long COVID

Problems with iron levels in the blood and the body's ability to regulate this important nutrient as a result of SARS-CoV-2 infection could be a key trigger for long COVID, new research has discovered.

6 Mar 2026

Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia

The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers prepare to meet growing demand for gene therapies in the coming years.

7 Dec 2025

Exa-cel shows complete success in early pediatric trials for sickle cell disease and beta-thalassemia

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger than 12.

6 Dec 2025

Gene editing therapy exa-cel improves quality of life in sickle cell disease and beta thalassemia

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia, according to two studies published in Blood Advances.

27 Aug 2025

New gene therapy strategy reactivates dormant genes to treat blood disorders

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers.

19 Jun 2025

Customized CRISPR gene editing therapy successfully treats infant with rare disease

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine.

15 May 2025

CRISPR tool unveils genetic drivers of blood cell maturation

Collaborative research led by investigators at Dana-Farber/Boston Children's Cancer and Blood Disorders Center defines a novel approach to understanding how certain proteins called transcription factors determine which genetic programs will drive cell growth and maturation.

4 Apr 2025

In the Thymus We Trust: ProTcell Therapy & the Future of Allogeneic Immunotherapy

In this interview, News Medical speaks with Olivier Negre, Chief Scientific Officer at Smart Immune, about how immunotherapy is being revolutionized.

From Hanson Wade Group - 7th Allogeneic Cell Therapies Summit 18 Mar 2025

Scientists discover key molecular switch for blood stem cell regeneration

A single molecular switch is essential for blood stem cells to enter an activated, regenerative state in which they produce new blood cells, according to a preclinical study led by Weill Cornell Medicine investigators.

10 Mar 2025

Iron dysregulation linked to long COVID development

5 Mar 2025

JRC releases a new CRM to aid the fight against beta-thalassemia

Our blood is a window to our health. Doctors examine biomarkers in our blood to determine our health status.

12 Oct 2024

Comprehensive cost and utilization of commercial CAR-T therapy in pediatric B-ALL patients

A new editorial paper was published in Oncoscience (Volume 11) on April 25, 2024, entitled, "The price of hope: CAR-T therapy in pediatric leukemia."

17 May 2024

Breakthrough in CRISPR delivery promises safer gene editing

Recent research in PNAS highlights advancements in non-viral, cell-specific delivery methods for CRISPR-Cas systems, emphasizing their potential to enhance research and gene therapy applications by minimizing risks associated with off-target effects and improving targeted delivery.

7 Mar 2024

Beta Thalassemia News and Research

Further Reading

New base editing techniques accurately modify genes in human embryos

New SMArT platform improves safety of CRISPR gene editing

Diatron introduces advanced RUO indices on its Aquila, Aquarius 3, Abacus 5 analyzers

Disrupted iron levels after SARS-CoV-2 infection may trigger long COVID

Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia

Exa-cel shows complete success in early pediatric trials for sickle cell disease and beta-thalassemia

Gene editing therapy exa-cel improves quality of life in sickle cell disease and beta thalassemia

New gene therapy strategy reactivates dormant genes to treat blood disorders

Customized CRISPR gene editing therapy successfully treats infant with rare disease

CRISPR tool unveils genetic drivers of blood cell maturation

In the Thymus We Trust: ProTcell Therapy & the Future of Allogeneic Immunotherapy

Scientists discover key molecular switch for blood stem cell regeneration

Iron dysregulation linked to long COVID development

JRC releases a new CRM to aid the fight against beta-thalassemia

Comprehensive cost and utilization of commercial CAR-T therapy in pediatric B-ALL patients

Breakthrough in CRISPR delivery promises safer gene editing

Iron dysregulation identified as potential trigger for long COVID

Breakthrough gene-editing therapy transforms lives of patients with a hereditary disorder

Fetal hormone GDF15 linked to nausea and vomiting in pregnancy

New CRISPR approach shows promise for treating glioblastoma

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

How Omics Technologies Are Transforming Biological Research and Drug Discovery

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