Health
New CRISPR-repressor system improves efficiency of gene therapies – The Science Board
Researchers have developed a CRISPR-based system that can simultaneously provide transcriptional control and gene editing on demand to improve the efficacy of gene therapies. The details were published in Nature Cell Biology on September 3.
Clinical gene therapies rely on viruses such as the adeno-associated virus (AAV) to deliver genes into a cell. CRISPR-based gene therapies act as molecular scissors to remove defective genes, add in missing sequences, or create a change in gene expression.
However, in response to AAVs, the host immune system can prevent the whole process by producing antibodies against the virus. This can result in rapid clearance of the virus and prevent them from delivering therapeutic payloads.
“Many clinic…
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