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Prime editing corrects the CFTR gene mutation
The approach precisely and durably corrects the CFTR mutation in human lung cells, which could lead to superior treatments.
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Broad Institute of MIT and Harvard
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Cancer dependencies missed by some CRISPR guides
Scientists discovered that, depending on germline variation, CRISPR-based experiments can result in false negatives.
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Potential for improved rheumatoid arthritis treatment
Six types of inflammation with various cell types and disease pathways identified may provide better treatment of rheumatoid arthritis.
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Researchers have engineered AAV vectors that cross the blood-brain barrier
In primate models, researchers have successfully shown that developed AAVs can cross the blood-brain barrier, which keeps many drugs from getting into the brain.
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Researchers gain insight into metabolic pathways hijacked by SARS-CoV-2
SARS-CoV-2 diverts building blocks from glucose production to the assembly of purine bases, researchers have found.
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Antisense oligonucleotides used to treat prion disease in animal models
Researchers have shown that using antisense oligonucleotides to reduce the levels of prion protein in lab animals with prion disease can extended their survival.
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First studies of human genetic variation released by the gnomAD Consortium
The Genome Aggregation Database (gnomAD) Consortium has released seven papers leveraging its database to study genetic variants and their potential for guiding discovery of safer drugs.
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Dozens of anti-cancer compounds revealed by screening existing drugs
Researchers have screened thousands of existing drug molecules against cancer cell lines to discover almost 50 compounds that combat the condition.
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CRISPR RNA-cutting enzyme programmed to kill viruses in human cells
Researchers have developed CRISPR-Cas13 enzyme-based technology that can be programmed to both detect and destroy RNA-based viruses in human cells.
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Scientists discover unique imaging technique to view synapse proteins
Researchers in the US have devised a new way to clearly image proteins located in synapses, which they hope will faciliate future treatment for diseases associated with blocked gene expression.
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Genetic model predicts risk for common deadly diseases
Using data collected from the UK, the data suggests that up to 25 million people in the US may be at more than triple the risk of coronary artery disease...
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Genomic analysis unravels complexities of the most common form of lymphoma
Genomic analysis has shown a better framework for understanding lymphoma’s many forms and will help to predict individual patient outcomes and guide personalised treatment.
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Scientists discover a small molecule that halts kidney disease progression
A team of researchers set out to discover the disease pathways of a rare genetic kidney disease and found a compound that stops the disease in its tracks.