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Gene therapy for Morquio A disease successful in rat model
A novel gene therapy has fully corrected whole-body alterations in a rat model, paving the way for Morquio A therapies.
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Genomics
Genomics is the branch of molecular biology concerned with the structure, function, evolution, and mapping of genomes.
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Immune cells found co-operating to capture and “eat” bacteria
Researchers have identified a spider-like antibacterial mechanism by immune cells that could inspire Staphylococcus aureus treatments.
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Scientists create cellular blueprint of multiple sclerosis lesions
An NIH team have built a cellular map of chronic multiple sclerosis (MS) lesions to identify cells that drive inflammation and potential therapies.
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New Gut Cell Atlas reveals genetic origins of Crohn’s disease
The Gut Cell Atlas comprises 428,000 cells in the gut and sheds light on the origin of Crohn’s disease and other intestinal diseases.
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Genomic study uncovers causes of lung cancer in non-smokers
An NIH study used whole genome sequencing to describe three molecular subtypes of lung cancer in non-smokers, possibly improving treatments.
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Researchers engineer “mini” CRISPR genome editing system
Stanford researchers have developed a multi-purpose “mini” CRISPR system, called CasMINI, that may be easier to deliver into human cells.
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Genetic defect in IBD patients linked to gut leakiness
A genetic defect in patients with inflammatory bowel disease (IBD) was found to affect how intestinal epithelial cells maintain a barrier.
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Immune cell changes detected in Waldenstrom macroglobulinemia (WM)
A new study has found mutations originating in blood progenitor cells, possibly leading to Waldenstrom macroglobulinemia (WM) therapies.
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DNA mechanism discovery may open up Huntington’s disease therapies
Scientists unveiled how a DNA repair protein may prevent Huntington’s disease, presenting a new target in future therapies.
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Innovative algorithm could help advance single-cell genomics
Researchers have developed a novel algorithm, “scArches”, that can compare data on single-cell genomics to better understand diseases.
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NPSR1 gene discovered as a key cause of endometriosis
NPSR1 has been identified as a genetic cause of endometriosis, revealing a potential drug target that may lead to better therapies.
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Delivering gene therapies to the brain to treat Alzheimer’s
Dr Ronald G Crystal, Professor and Chairman of the Department of Genetic Medicine, Weill Cornell Medical College, spoke to Drug Target Review’s Victoria Rees about his research into Alzheimer’s disease and why gene therapies represent a promising area of research for neurodegenerative conditions.
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Brain organoids mimic autism-related head size changes
Scientists have developed brain organoids that recapitulate the head size of autism patients to study the condition as well as possible therapies.
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Detergent-like APOL3 protein shown to kill bacteria in human cells
Researchers have revealed that the protein APOL3 acts as a detergent in human cells, potentially leading to new antibacterial treatments.
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Spike gene mutants could be used as novel COVID-19 vaccine
SARS-CoV-2 Spike gene mutants may be developed into immunogens for new vaccines against COVID-19, a study in hamsters has shown.
