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.
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.
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.
Scientists at St. Jude Children's Research Hospital are tackling Mycobacterium abscessus (Mab) antibiotic resistance.
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.
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.
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.
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.
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.
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.
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.
Despite the astounding advances made in understanding the biologic underpinnings of cancer, many cancers are missing obvious genetic drivers.
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.
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.
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.
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.
CTCF is a critical protein known to play various roles in key biological processes such as transcription.
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.
To celebrate National DNA Day 2022, we spoke to science communicator Professor Matthew Cobb.
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.
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.