Applied StemCell Inc. (ASC) is a fast growing biotechnology company headquartered in Milpitas, California. Our goal is to advance gene-editing and stem cell innovation for biomedical research and the biotechnology industry. Using our TARGATT™ technology, a gene-of-interest can be specifically inserted at a well-characterized, transcriptionally active locus in the genome with guaranteed transgene expression. This site-specific Knock-in technology has been first established in mouse models, and we have successfully applied it to human cell lines. We are moving forward to various projects that will combine the CRISPR/Cas9 technology and TARGATT™ for highly specified transgene expression. are characterized by high genetic penetrance. Our therapeutic pipeline will have a strong focus on monogenic blood disorders that are characterized by high genetic penetrance Such diseases are a strong point for the company, which specializes in the genetic engineering of stem cell lineages.
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