Biobank 000000
- Generell biobank
- Biobank er plassert ved UNDEFINED
- Emma Haapaniemi er ansvarlig for biobank
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.
Disse dokumentene er kun synlige for prosjektleder, enhetens leder og forskningsadministrasjon.
Dokument ikke lastet opp
Norsk Senter for Molekylærmedisin