Recombinant Adeno-Associated Virus
Universal Cells uses recombinant Adeno-Associated Virus (rAAV)-mediated gene editing to engineer chromosomal genes without the use of genotoxic nucleases. Serotype 3B rAAV vectors efficiently infect pluripotent stem cells and their single-stranded DNA genomes pair with homologous chromosomal sequences during replication to precisely alter human genomic sequences.
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Unlike nuclease-based genome editing, our technolo...
Unlike nuclease-based genome editing, our technology does not require a DNA strand break, does not produce off-target alterations to the genome, does not create unwanted mutations at the target site, and does not introduce foreign nuclease genes. rAAV vectors pose fewer safety concerns than most viral vectors, and have been used in numerous clinical trials, making this technology well-suited for clinical use.
Universal Cells conducts clinical-grade gene editing of human stem cells in our Seattle facilities. Our technology allows us to produce customized stem cells that contain deletions, insertions, or point mutations at any genomic position. Editing is coupled with genomic analysis to ensure that the editing was precise and that unwanted mutations did not occur during the editing process.
NO OFF-TARGET CUTTING
NO OFF-TARGET CUTTING
rAAV vectors do not create DNA strand breaks in the genome.
NO ON TARGET MISTAKES
Homologous pairing edits genes with single nucleotide precision.
NON-TOXIC DELIVERY
No need for electroporation or transfection.
NO NUCLEASE PROTEINS
No foreign nucleases that could lead to immune rejection.
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