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Whitepaper: How to monitor and minimise off-target events
Whitepaper discusses strategies to optimize various factors that cause off-target events with Invitrogen™ TrueCut™ HiFi Cas9 Protein.
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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
Promising new gene therapy approach for LGMD2B
Researchers at Children’s National Hospital in the US have developed a promising new gene therapy for limb-girdle muscular dystrophy (LGMD) 2B.
article
Under the microscope: Keying into UK biosciences – GenScript Biotech operations lands in Oxford
With operations in over 100 countries worldwide, GenScript Biotech, an industry leader in biotechnology reagent services, has established a new UK office in the Oxford area to better connect with the UK biosciences sector.
news
Researchers investigate gene network to identify “cancer driver genes”
The new study looked at the network of gene-gene interactions associated with cancer onset and progression to identify therapeutic targets.
article
Highlights from Drug Target Review’s Cell & Gene Therapy Advancements Online Summit
Here, we round up some of the key takeaways from the expert panel discussions at the Cell & Gene Therapy Advancements Online Summit.
news
Positive pre-clinical results for CRISPR-based HIV therapy
The CRISPR-based therapy called EBT-101 excised HIV proviral DNA from the genomes of different cells and tissues in human cells and mice.
news
Researchers discover new link between CRISPR, p53 and cancer
The new findings suggest that inhibiting p53 can reduce CRISPR-associated cancer risks and may lead to developments in precision medicine.
news
Researchers achieve DNA methylation removal in mouse models
The study used CRISPR to show that DNA “de-methylation” activity can be targeted to anywhere in the DNA and may be a new therapeutic strategy.
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.
podcasts
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.