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Discussing next-gen AAV vectors with Dr Arun Srivastava, University of Florida
An interview with Dr Arun Srivastava about his research into safe and effective AAV vectors that do not prompt a reaction from the immune system.
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Gene Therapy
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
news
CRISPR-based droplet method speeds up gene therapy research
The new CRISPR-based technology called MIC-Drop rapidly identified several genes for heart development and function in zebrafish.
article
CRISPR-Cas9-based gene dropout screens: a powerful platform for drug discovery
CRISPR holds great promise in advancing pharmacological research and has fuelled the rapid expansion of using gene-edited cells for drug discovery processes. CRISPR-Cas9 dropout screens have emerged as a useful tool for high-throughput large-scale loss-of-function screens, which seek to identify the relationship between genotype and phenotype. Dr Pushpanathan Muthuirulan, Research…
whitepaper
Cell & Gene Therapy Advancements Whitepaper
Download this whitepaper to discover the latest research and developments in cell and gene therapies via exclusive articles and interviews.
news
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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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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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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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!
news
New skin cell mechanism discovery could treat muscle-related diseases
Scientists have identified the mechanism behind the conversion of skin cells into immature muscle cells, which could lead to therapies preventing muscle degeneration.
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TREM2 regulation to prevent neurodegeneration is possible, study shows
Scientists used a synthetic thyroid hormone in mice to regulate the TREM2 gene implicated in diseases such as Alzheimer’s and Parkinson’s.
news
Prime editing method corrects cystic fibrosis in stem cells
A team of scientists used a CRISPR-Cas9 technique known as prime editing to correct cystic fibrosis in cultured human stem cells.
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CRISPR-Cas9 used to prevent Fuchs’ corneal dystrophy in mice
Scientists have shown that start codon disruption with CRISPR-Cas9 gene editing can prevent Fuchs’ corneal dystrophy in mouse models.
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Mitochondria repair in mice may provide answers on Parkinson’s disease
Researchers used an experimental small molecule that helped restore the removal of damaged mitochondria from brain cells in a mouse model of Parkinson's.
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Genetic treatment reverses age-related memory loss in mice
Scientists have shown that manipulating the perineuronal nets (PNNs) in the brains of mice led to the reversal of age-related memory loss.