Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

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

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

New generation of CRISPR shows safer path to treating genetic diseases

A new generation of CRISPR technology developed at UNSW Sydney offers a safer path to treating genetic diseases like Sickle Cell, while also proving beyond doubt that chemical tags on DNA - often thought to be little more than genetic cobwebs - actively silence genes.

17 Aug 2025

Immigrant kids detained in ‘unsafe and unsanitary’ sites as Trump team seeks to end protections

A child developed a rash after he was prevented from changing his underwear for four days. A little boy, bored and overcome with despair, began hitting himself in the head.

30 Jul 2025

Timepoint at which B cells transform into leukemic cells impacts treatment outcomes

New findings show that the stage of normal cell development at which B cells transform into leukemic cells impacts treatment outcomes for pediatric patients with B-cell acute lymphoblastic leukemia (B-ALL).

2 Jul 2025

HHS eliminates CDC staff who made sure birth control is safe for women at risk

For Brianna Henderson, birth control isn’t just about preventing pregnancy.

1 Jul 2025

New tool reveals hidden water patterns in protein structures

Every protein in the body is encased in a water shell that directs protein structure, provides vital stability and steers function.

30 Jun 2025

Gene therapy shows promise for reducing stroke risk in patients with sickle cell disease

Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital.

30 Jun 2025

Scientists identify novel combination therapy approach to treat pediatric acute myeloid leukemia

Scientists at St. Jude Children's Research Hospital and Dana-Farber Cancer Institute today report the identification of a novel combination therapy approach to treat pediatric acute myeloid leukemia (AML).

20 Jun 2025

Researchers find unique genetic profile in childhood cancer survivors with cardiomyopathy

Scientists at St. Jude Children’s Research Hospital studied how common and rare genetic variants influence late-onset cardiomyopathy risk, highlighting the unique genetic complexity of childhood cancer survivors.

20 Jun 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

Global guideline released by WHO on managing sickle cell disease in pregnancy

The World Health Organization (WHO) today released its first-ever global guideline on the management of sickle cell disease (SCD) during pregnancy, addressing a critical and growing health challenge that can have life-threatening consequences for both women and babies.

19 Jun 2025

Gene therapy shows long-term success in hemophilia B patients

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years of follow-up data.

12 Jun 2025

MUSC creates protocol to improve emergency care for pediatric sickle cell patients

The Pediatric Emergency Medicine and Pediatric Hematology/Oncology divisions at the Medical University of South Carolina (MUSC) joined forces to create and test an algorithm for treating children with sickle cell disease who present to the Emergency Department (ED) with a fever.

28 May 2025

Stress granules play a protective role against neurodegenerative disease

Scientists from St. Jude Children's Research Hospital and Washington University in St. Louis report mechanistic insights into the role of biomolecular condensation in the development of neurodegenerative disease.

28 May 2025

Study reveals dual role of TEAD proteins in neural development

Scientists at St. Jude Children’s Research Hospital found that, in neural development, the same set of proteins involved in cellular self-renewal can also facilitate differentiation with the right binding partner.

20 May 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

St. Jude Children's scientists use brain organoids to study rare encephalopathy

While extremely rare, encephalopathy (a condition affecting brain function) triggered by mutations in the UBA5 gene has devastating impacts, with affected individuals reaching few developmental milestones and experiencing frequent and early-onset seizures.

8 May 2025

Scientists unlock secrets of how cells navigate the body

Scientists from St. Jude Children's Research Hospital and the Medical College of Wisconsin have created a data science framework to better understand how cells travel through the body.

23 Apr 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

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

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

Investigation raises concerns over tooth stem cell banking claims

New generation of CRISPR shows safer path to treating genetic diseases

Immigrant kids detained in ‘unsafe and unsanitary’ sites as Trump team seeks to end protections

Timepoint at which B cells transform into leukemic cells impacts treatment outcomes

HHS eliminates CDC staff who made sure birth control is safe for women at risk

New tool reveals hidden water patterns in protein structures

Gene therapy shows promise for reducing stroke risk in patients with sickle cell disease

Scientists identify novel combination therapy approach to treat pediatric acute myeloid leukemia

Researchers find unique genetic profile in childhood cancer survivors with cardiomyopathy

New gene therapy strategy reactivates dormant genes to treat blood disorders

Global guideline released by WHO on managing sickle cell disease in pregnancy

Gene therapy shows long-term success in hemophilia B patients

MUSC creates protocol to improve emergency care for pediatric sickle cell patients

Stress granules play a protective role against neurodegenerative disease

Study reveals dual role of TEAD proteins in neural development

Customized CRISPR gene editing therapy successfully treats infant with rare disease

St. Jude Children's scientists use brain organoids to study rare encephalopathy

Scientists unlock secrets of how cells navigate the body

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

Innovation in neuroscience drug discovery - eBook eBook

Unveiling Hidden Potential: Organoids for Disease Modeling in Neuroscience Research

How GLP‑1 Agonists Are Shaping the Future of Obesity Treatment With Dr Bryony Henderson

Flow chemistry, AI, and NMR: A next-gen approach to scalable API production

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Innovation in neuroscience drug discovery - eBook eBook

Unveiling Hidden Potential: Organoids for Disease Modeling in Neuroscience Research

How GLP‑1 Agonists Are Shaping the Future of Obesity Treatment With Dr Bryony Henderson

Flow chemistry, AI, and NMR: A next-gen approach to scalable API production

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