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Researchers design genetic therapy to prevent brain deterioration

Drug Discovery World

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. The researchers have shown that problems with calcium underlie these progressive diseases.

Therapies 147
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Exploring liver disease therapies  

Drug Target Review

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Our team of chemists then uses this information to develop novel therapies.

Therapies 102
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Researchers identify potential new genetic target for cancer drugs

Drug Discovery World

UK researchers have mapped the exact variants in a gene that dramatically increase a person’s risk of developing cancer, paving the way for targeted treatment across diverse populations. The research team tested all 18,108 possible DNA changes in the BAP1 gene using ‘saturation genome editing’.

Research 130
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Research points to potential new medical therapy for Lyme disease

SCIENMAG: Medicine & Health

Pearson, co-author of the paper, is now a NEWVEC post-doctoral researcher at UMass Amherst. Lynch, lead author, is now a research fellow in the Department of Veterinary and Animal Sciences. The discovery began with an “aha” moment by then-Ph.D. Credit: UMass Amherst […]

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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

Therapies 162
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With retinal images and genetic data, researchers predict cardiovascular, metabolic, and other disease risks

Broad Institute

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

Disease 124
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Personalising whole genome sequencing doubles diagnosis of rare diseases 

Drug Discovery World

A new study led by Medical Research Council-funded researchers from UCL has found that tailoring the analysis of whole genome sequencing to individual patients could double the diagnostic rates of rare diseases. . It also detected potential disease-causing variants in a further 3.9% Context . The study .

Disease 130