Sickle Cell Disease News and Research

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Sickle-cell disease, or sickle-cell anaemia (or drepanocytosis), is a life-long blood disorder characterized by red blood cells that assume an abnormal, rigid, sickle shape. Sickling decreases the cells' flexibility and results in a risk of various complications.

Precision Combination Therapy Better Controls High-Risk Subtypes of B-ALL in Mouse Models

Despite having an overall survival rate of 94%, B-cell acute lymphoblastic leukemia (B-ALL), the most common childhood cancer, can prove challenging to treat, with survival among relapsed or resistant cases falling between 30-50%.

9 Apr 2024

Retrotransposon-Based PRINT Technique Holds Potential for Precise Gene Insertion

The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease.

22 Feb 2024

Phage Proteins Tame CRISPR-Cas3, Paving Way for Precise Gene Therapy

Through the use of CRISPR, an immune system that bacteria use to defend themselves against viruses, researchers have been able to alter genetic information within cells.

22 Jan 2024

Scientists Reinvent Spectinomycin as a Powerhouse Weapon for Drug-Resistant Superbug

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

10 Jan 2024

Scientists Unravel the Mystery of Parkinson's Disease With 3D Protein Structures

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.

26 Dec 2023

The Adrenaline Puzzle Solved: Scientists Untangle Key Cell Receptor's Inner Workings

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.

26 Dec 2023

Scientists Unlock the Structure of Vesicular Monoamine Transporter 2

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

Study Finds Diverse Set of Immune Cells Linked to Flu Protection

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

New Proof-of-Concept Model for Delivering Gene Editing Tools to Treat 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.

28 Jul 2023

Self-Collected STI Tests Perform as Well as Provider-Collected Tests

Self-collected tests performed similarly to provider-collected tests for detecting common sexually transmitted infections (STIs), according to research presented today at the 2023 AACC Annual Scientific Meeting & Clinical Lab Expo. These findings could enable discreet new testing options that expand access to screening.

26 Jul 2023

Nutrient Tug-of-War Between Tumor and Immune Cells Could Be Targeted for Cancer Therapy

Scientists at St. Jude Children's Research Hospital found immune and tumor cells compete over glutamine, a major nutrient in their local environment, with significant implications for anti-cancer activity.

6 Jul 2023

Researchers Create a Computational Tool to Gain New Insights From Multi-Omics Data

Despite the astounding advances made in understanding the biologic underpinnings of cancer, many cancers are missing obvious genetic drivers.

5 May 2023

STING-seq Discovers Causal Variants and Genetic Mechanisms for Blood Cell Traits

A major challenge in human genetics is understanding which parts of the genome drive specific traits or contribute to disease risk. This challenge is even greater for genetic variants found in the 98% of the genome that does not encode proteins.

5 May 2023

Sickle Cell Disease Cure? Prime Editing Offers Hope for Blood Disorder

At St Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard, researchers have shown how prime editing could help correct the mutation that results in sickle cell disease in a possibly curative method.

18 Apr 2023

Researchers provide an entirely new perspective on tissue oxygenation

Cardiovascular medicine, hematology and pulmonary medicine may soon have the first-ever therapies to correct poor tissue oxygenation, a key driver of disease in millions, including peripheral artery disease, sickle cell disease, heart failure, stroke, emphysema and many others.

2 Mar 2023

Novel framework provides solutions to evade detoxification networks

Scientists at St. Jude Children's Research Hospital have demonstrated how drug makers can avoid two key problems: toxicity and resistance. The researchers made slight changes to a small molecule to reduce its metabolism and elimination by the cellular detoxification network regulated by the pregnane X receptor (PXR). This research provides a framework to develop solutions to the long-standing issue of how to evade detoxification networks using medicinal chemistry. The findings were published today in Proceedings of the National Academy of Sciences.

1 Mar 2023

Novel protein degradation technology provides functional insights into how CTCF regulates transcription

CTCF is a critical protein known to play various roles in key biological processes such as transcription.

30 Jan 2023

Study describes the importance of the autoimmune response in bacteria

CRISPR has made headlines in recent years for its potential to help patients with conditions as diverse as blindness and sickle cell disease. However, bacteria were already using CRISPR as an immune system to combat viruses long before humans adopted it to combat genetic disorders.

3 Jun 2022

National DNA Day 2022: an interview with Professor Matthew Cobb

To celebrate National DNA Day 2022, we spoke to science communicator Professor Matthew Cobb.

From The University of Manchester 25 Apr 2022

Researchers use CRISPR gene editing tool to analyze globin gene switching

CRISPR gene editing—a form of “molecular scissors”—was used by UNSW researchers to investigate how deletions in one section of the genome might impact the expression of surrounding genes.

From The University of New South Wales (UNSW) 19 Apr 2022