Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

St. Jude scientists develop novel spectinomycin derivatives to combat Mycobacterium abscessus

Scientists at St. Jude Children's Research Hospital are tackling Mycobacterium abscessus (Mab) antibiotic resistance.

5 Jan 2024

Study: Two-thirds of babies with sickle cell disease are born to mothers living in disadvantaged areas

As if starting life with a potentially disabling genetic blood disease wasn't enough, a study shows that almost two-thirds of babies born with sickle cell disease are born to mothers who live in disadvantaged areas.

4 Jan 2024

Existing beta blocker investigated as novel sickle cell therapy

A beta blocker typically used to treat heart problems, hemangioma, migraines and anxiety could be a new therapeutic for patients with sickle cell disease. Researchers led by Ankit A. Desai, MD, associate professor of medicine at the Krannert Cardiovascular Research Center (KCVRC) at Indiana University School of Medicine, have been awarded a $3 million grant by the U.S. Department of Defense to evaluate the efficacy of this drug.

3 Jan 2024

St. Jude researchers crack the code of the β2-adrenergic receptor

Through an international collaboration, scientists at St. Jude Children's Research Hospital leveraged data science, pharmacology and structural information to conduct an atomic-level investigation into how each amino acid in the receptor that binds adrenaline contributes to receptor activity in the presence of this natural ligand.

22 Dec 2023

St. Jude researchers reveal structure of Parkinson's-linked proteins

Scientists at St. Jude Children's Research Hospital revealed the complex structure of two Parkinson's disease-related proteins, both of which are implicated in late-onset cases.

21 Dec 2023

Scientists identify genetic and epigenetic culprits behind bilateral Wilms tumor

Children with bilateral Wilms tumor have a tumor in each of their kidneys -; a condition that strongly suggests an underlying genetic or epigenetic predisposition driving the disease.

18 Dec 2023

St. Jude study sheds light on prediabetes prevalence and diabetes risk in childhood cancer survivors

A study from St. Jude Children's Research Hospital sheds light on the incidence of prediabetes and diabetes in childhood cancer survivors for better prevention and treatment.

13 Dec 2023

Many patients may not benefit from gene-editing treatments for sickle cell disease

Federal approval of a breakthrough gene-editing technology that treats the pain and debilitating effects of sickle cell disease is cause for celebration among a community with few options for relief.

8 Dec 2023

New approach enhances CAR T-cell therapy for solid tumors

Immunotherapy using modified chimeric antigen receptor (CAR) T cells has greatly improved survival rates for pediatric patients with relapsed and recurrent leukemia.

30 Nov 2023

Scientists uncover new therapeutic targets for high-risk pediatric leukemia

Scientists at St. Jude Children's Research Hospital comprehensively identified genes directly regulated by a protein associated with high-risk pediatric leukemias.

28 Nov 2023

New CRISPR approach shows promise for treating glioblastoma

The gene-editing technology CRISPR shows early promise as a therapeutic strategy for the aggressive and difficult-to-treat brain cancer known as primary glioblastoma, according to findings of a new study from Gladstone Institutes.

27 Nov 2023

Sickle Cell Disease patients and the use of disease-modifying treatments

New disease-modifying treatments (DMTs), namely, crizanlizumab, L-glutamine, and voxelotor, have recently been approved.

23 Nov 2023

St. Jude scientists discover potential CAR T cell therapy target for brain and solid tumors

Findings from St. Jude Children's Research Hospital are moving the field of cancer immunotherapy one step closer to treating brain and solid tumors.

21 Nov 2023

Scientists uncover key to enhancing cancer-killing T cells

T cells are the key immune system component involved in killing cancer. Tumors produce signals that turn off these T cells, partially by forcing them to progressively differentiate (mature) into a hypofunctional state known as exhaustion.

21 Nov 2023

Long-term monitoring of gene therapy patients crucial for safety, study says

Medical research has shown promising results regarding the potential of gene therapy to cure genetic conditions such as sickle cell disease and the findings of this study, published in Nature Medicine, offer important new insights into processes happening in the body after treatment.

16 Nov 2023

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

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

St. Jude scientists develop novel spectinomycin derivatives to combat Mycobacterium abscessus

Study: Two-thirds of babies with sickle cell disease are born to mothers living in disadvantaged areas

Existing beta blocker investigated as novel sickle cell therapy

St. Jude researchers crack the code of the β2-adrenergic receptor

St. Jude researchers reveal structure of Parkinson's-linked proteins

Scientists identify genetic and epigenetic culprits behind bilateral Wilms tumor

St. Jude study sheds light on prediabetes prevalence and diabetes risk in childhood cancer survivors

Many patients may not benefit from gene-editing treatments for sickle cell disease

New approach enhances CAR T-cell therapy for solid tumors

Scientists uncover new therapeutic targets for high-risk pediatric leukemia

New CRISPR approach shows promise for treating glioblastoma

Sickle Cell Disease patients and the use of disease-modifying treatments

St. Jude scientists discover potential CAR T cell therapy target for brain and solid tumors

Scientists uncover key to enhancing cancer-killing T cells

Long-term monitoring of gene therapy patients crucial for safety, study says

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

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

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