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.

Disrupting a Gene Boosts T Cell Therapy, St. Jude Study Shows

Immunotherapy, using a patient's own immune system to treat disease, has shown promise in some patients with cancer but has not worked in most.

15 Oct 2024

New Gene-Editing Tool Offers More Precision and Flexibility

Researchers at the University of Sydney have created a gene-editing instrument that is more precise and adaptable than the industry standard CRISPR, which has transformed genetic engineering in biotechnology, agriculture, and medicine.

24 Jun 2024

St. Jude Researchers Discover NLRC5's Role in Inflammatory Cell Death

The innate immune system is responsible for protecting the human body from threats that could cause disease or infection.

17 Jun 2024

New Clinical Trial Offers Path to Sickle Cell Cure

The most recent results from a clinical trial were given by researchers in an effort to find a cure for sickle cell disease, a painful genetic blood illness that has few available treatments.

17 Jun 2024

Revolutionizing Cell Subpopulation Studies: The ConVERGD Approach

As gene sequencing technologies become more powerful, our understanding of cellular diversity has grown in parallel.

29 May 2024

Cryo-EM Reveals Structural Insights Into ADAM17/iRhom2 Complex

Efficient communication between cells is vital for many biological processes, such as recruiting immune cells to combat disease.

24 May 2024

St. Jude Scientists Reveal Role of Non-Coding DNA Variants in Childhood Leukemia

St. Jude Children's Research Hospital scientists have identified specific DNA variants in the non-coding regions of the genome contributing to chemotherapy resistance in acute lymphoblastic leukemia (ALL).

2 May 2024

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