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.

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

Research offers alternative cell option for organs-on-chips

Organ-on-a-chip technology has provided a push to discover new drugs for a variety of rare and ignored diseases for which current models either don't exist or lack precision. In particular, these platforms can include the cells of a patient, thus resulting in patient-specific discovery.

13 Dec 2021