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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 Fraser Owen 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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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.
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On-demand webinar: An AI-based approach to phenotypic characterisation of neuronal cells
Evaluation of neurotoxicity effects is an active area of investigation in drug discovery and disease modeling.
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Cartilage growth gene TRPV4 shows promise for joint repair therapy
The discovery that the TRPV4 gene regulates cartilage growth could lead to treatments for osteoarthritis and other cartilage diseases.
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Researchers identify potential drug to treat GIST tumours
A potential drug to treat a subset of gastrointestinal stroma tumours (GIST) have been found to tackle intestinal tract cancer.
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Drug Target Review update: exclusive report, online summit and more!
Hear about the latest Drug Target Review updates from Deputy Editor Victoria Rees who discusses our new report on AI & Informatics, the Cell & Gene Therapy Advancements online summit and much more!
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Multi-omics approach identifies genes associated with alcohol use disorder
First-of-its-kind study uses a multi-omics approach to provide a list of causal candidate genes associated with alcohol use disorder (AUD).
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Researchers identify novel genomic marker of childhood leukaemia
A team have found that two DNA changes appear to predict acute lymphoblastic leukaemia, illuminating genetic understanding of the disease.