news
Researchers achieve DNA methylation removal in mouse models
The study showed that DNA “de-methylation” activity can be targeted to anywhere in the DNA and may be a new therapeutic strategy.
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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
Gene therapy bolsters Parkinson’s drug benefits in pre-clinical study
The gene therapy restored the ability of neurons to convert levodopa to dopamine and may help develop therapies to slow disease progression.
news
Moderna and Metagenomi to develop in vivo gene therapies
Moderna and Metagenomi have announced a collaboration to jointly create next-generation in vivo gene editing therapeutics.
podcast
Episode 3 – Cell & gene therapy with Dr Sam Wadsworth, Ultragenyx Pharmaceutical & Dr Eric Ostertag, Poseida Therapeutics
Tune in to this podcast to learn about AAV vectors for gene therapy delivery and engineering CAR T cells against solid tumours.
news
Gene therapy shows promise for Angelman syndrome treatment
A new gene therapy restored motor skill-learning and usual behaviours in Angelman syndrome mouse models, suggesting a novel therapy for the condition.
news
Two-week-old human embryos reveal origin of Huntington’s disease
Scientists have discovered a signalling pathway alteration in embryos with Huntington’s disease, paving the way for ground-breaking treatments.
news
Experimental vaccine a major breakthrough for rheumatoid arthritis
A protein-based vaccine has proven a promising option to prevent rheumatoid arthritis, improving bone quality in animal models.
news
Gene therapy method could restore visual function after stroke
Scientists have revealed a way to use gene therapy to turn glial brain cells into neurons, restoring vision and potentially restoring motor function.
video
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.
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.
article
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.
news
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.
