Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.

12 Nov 2023

Children need to be screened for vision problems as often as adults with sickle cell disease, study suggests

Sickle cell disease is a rare, inherited disorder in which the red blood cells become hard, sticky, and change shape, resembling the farm tool.

6 Nov 2023

Scientists determine structure of key protein in neuronal communication

Neurons talk to each other using chemical signals called neurotransmitters. Scientists at St. Jude Children's Research Hospital have drawn on structural biology expertise to determine structures of vesicular monoamine transporter 2 (VMAT2), a key component of neuronal communication.

2 Nov 2023

Major new campaign launched in the UK to encourage flu and COVID-19 vaccinations

The UK Health Security Agency (UKHSA), Department of Health and Social Care and NHS England are urging those eligible who've not yet come forward for the flu and COVID-19 vaccines to 'get winter strong' and join the millions of others in taking up the offer ahead of the festive and new year season when flu and COVID-19 are expected to peak.

1 Nov 2023

Sickle cell disease patients willing to accept high risks for gene therapy cure

Individuals living with severe sickle cell disease (SCD) are highly interested in new, potentially curative gene therapy treatments and are willing to accept associated risks for a chance at a cure, according to a study published today in Blood Advances.

31 Oct 2023

For people with sickle cell disease, ERs can mean life-threatening waits

Heather Avant always dresses up when she goes to the emergency room.

17 Oct 2023

Mount Sinai researchers receive NIH grant to compare new treatment options for sickle cell disease

The Mount Sinai Health System has received a $12,180,625 grant from the National Heart, Lung and Blood Institute to compare new treatment options for sickle cell disease and determine which work best for specific patients.

12 Oct 2023

St. Jude Children's researchers better define hyperdiploidy in childhood B-ALL

Hyperdiploidy is a genetic condition observed in cancer cells, where the cells contain more chromosomes than usual.

20 Sep 2023

Stuart Orkin receives the third Elaine Redding Brinster Prize for development of sickle cell disease therapy

For his work discovering the basis for hemoglobin gene switching and applying those insights to develop a therapy for sickle cell disease and other blood diseases, the Institute for Regenerative Medicine at the University of Pennsylvania awarded Stuart Orkin, MD the third Elaine Redding Brinster Prize in Science or Medicine.

15 Sep 2023

A subset of CD8+ T cells can play a protective role against Alzheimer’s disease pathogenesis

Scientists at St. Jude Children’s Research Hospital found that a subset of CD8+ T cells in the brain lessens the activation of microglia and limits disease pathology in a model of Alzheimer’s disease.

8 Sep 2023

Stem cell gene therapy may offer a promising, curative treatment for sickle cell disease

New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD).

30 Aug 2023

Immune cells present in people months before influenza infection, finds study

St. Jude Children's Research Hospital scientists, in collaboration with the Institute of Environmental Science and Research (ESR) Limited, found that immune cells present in people months before influenza (flu) infection could more accurately predict if an individual would develop symptoms than current methods which primarily rely on antibody levels.

18 Aug 2023

Mutational accessibility: A key driver of viral evolution

The influenza (flu) virus is constantly undergoing a process of evolution and adaptation through acquiring new mutations.

29 Jul 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

Rice bioengineers receive NIH grant for sickle cell disease research

Rice University bioengineer Gang Bao and his team have won a 4-year, $2.6 million grant from the National Institutes of Health to address critical questions surrounding the safety and efficacy of using gene editing to treat sickle cell disease.

18 Jul 2023

Study describes the scope and integration of music therapy program within UH medical centers

A new study from University Hospitals (UH) Connor Whole Health describes the scope and integration of their music therapy program within10 UH medical centers.

14 Jul 2023

Sickle cell disease: A global call to action

Reducing the burden of Sickle Cell Disease (SCD) requires substantial financial and political commitment to improving data-collection, diagnosis, treatment and training - doing so will positively impact the lives of millions of patients and families worldwide - says a new Commission published in The Lancet Haematology journal.

12 Jul 2023

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Children need to be screened for vision problems as often as adults with sickle cell disease, study suggests

Scientists determine structure of key protein in neuronal communication

Major new campaign launched in the UK to encourage flu and COVID-19 vaccinations

Sickle cell disease patients willing to accept high risks for gene therapy cure

For people with sickle cell disease, ERs can mean life-threatening waits

Mount Sinai researchers receive NIH grant to compare new treatment options for sickle cell disease

St. Jude Children's researchers better define hyperdiploidy in childhood B-ALL

Stuart Orkin receives the third Elaine Redding Brinster Prize for development of sickle cell disease therapy

A subset of CD8+ T cells can play a protective role against Alzheimer’s disease pathogenesis

Stem cell gene therapy may offer a promising, curative treatment for sickle cell disease

Immune cells present in people months before influenza infection, finds study

Mutational accessibility: A key driver of viral evolution

New gene editing approach could revolutionize the treatment of blood disorders

Rice bioengineers receive NIH grant for sickle cell disease research

Study describes the scope and integration of music therapy program within UH medical centers

Sickle cell disease: A global call to action

Study shows how glutamine can help or hinder cancer-killing activity

Base editing shows promise for treating sickle cell disease and beta thalassemia

New study warns of dangers of gene editing in human embryos

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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