We are testing a strategy to improve therapeutic CRISPR delivery with viruses by adding in a protein that helps viruses enter cells.
The recombinant adeno-associated virus (rAAV) is currently the most common delivery vehicle for gene therapies. The CRISPR genome editing system packaged in AAV capsids is potentially considered to hold great promise in transforming the future of gene therapy. One of the big hurdles in the field that limits its widespread use is the efficient delivery of genetic material in different cell types. A crucial factor that determines delivery of AAVs is the presence of AAV capsid receptors on cell membranes that facilitate binding and internalization of AAV particles. We chose to address this challenge by delivering a ‘pan AAV receptor’ to target tissues to improve the access of CRISPR-rAAV in different cell types. This approach holds promise to overcome a major delivery hurdle and achieve the level of CRISPR component expression required for optimal in vivo editing efficiency.
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