New model yields promising gene therapy for heart condition
Scientists in Japan have developed a credible heart cell model for arrhythmogenic cardiomyopathy and observed a positive response in the condition using PKP2 gene therapy.
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Scientists in Japan have developed a credible heart cell model for arrhythmogenic cardiomyopathy and observed a positive response in the condition using PKP2 gene therapy.
Researchers have developed a new drug delivery system using virus-like particles to successfully transport gene-editing proteins in pre-clinical studies.
Dr Greg Neely, University of Sydney, explains how he and his team used pooled whole genome CRISPR activation screening to identify LRRC15 as a SARS-CoV-2 spike-interacting protein.
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The capability to edit genes has been transformative in opening therapeutic avenues for hitherto untreatable diseases and aiding biological insight. Scientists have now discovered a way to enhance this process using retrons, making the process more efficient and effective.
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According to researchers, a new method to deliver CRISPR-Cas9 to vascular endothelial cells could be used to treat many diseases, including COVID-19.
Here, we round up some of the key takeaways from the expert panel discussions at the Cell & Gene Therapy Advancements Online Summit.
Dr Danilo Maddalo from Genentech outlines the main benefits of CRISPR within the clinical realm and highlights some of the safety concerns that must be considered before CRISPR products reach patients.
Scientists used a CRISPR-based screen to find that inhibiting MTHFD2 reduces disease severity in inflammatory disease models.
Rice University scientists have refined specific CRISPR-base editing strategies to avoid errors that occur during gene editing.
The funding will accelerate clinical development of cell therapies using breakthrough gene engineering technology opti-oxTM.
A protein-based vaccine has proven a promising option to prevent rheumatoid arthritis, improving bone quality in animal models.
Dubbed 'Zaki syndrome', the condition affects prenatal development of several organs and was identified using whole genome sequencing.