CRISPR-Cas9 gene therapy for monogenic blood disorders
AIMS: We aim to develop a CRISPR-Cas-based gene therapy platform for monogenic blood disorders.
BACKGROUND: The novel CRISPR-Cas system can be used to cure various genetic blood diseases by correcting the causative mutation in harvested patient stem cells, that are then infused back to the patient.
WORKPLAN: We have developed a modified CRISPR system that utilizes an engineered Cas9 that is more efficient in inducing HR (homologous recombination) -mediated precision genome editing than wild-type Cas9. As HR-mediated mutation correction happens exclusively in the synthesis (S) phase of the cell cycle, the platform also uses an inhibitor that allows Cas9 only to be active in the S phase, thus increasing safety of the technology. In addition, we have bound the repair template to Cas9, and will transiently inhibit p53 during genome editing.
SIGNIFICANCE: If successful, the method can treat a wide variety of diseases and mutations. It can also be used for cancer immunotherapy and other applications that benefit from clinical immune cell engineering.
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