Genome Editing for CNS Disorders

Beta testing: preclinical genome editing in β-globin disorders

The β-globin disorders β-thalassemia and sickle cell disease have been at the forefront of gene therapy development from its very inception. Owing to their frequency, severity and exceptionally well characterized molecular pathology, and to the availability of hematopoietic stem and progenitor cells as substrate for therapies, these disorders promise both fast insights into new methodologies an...

Genome Editing



Ginseng Use in Medicine: Perspectives on CNS Disorders

Ginseng, the root of Panax species, is a well-known folk medicine. It has been used as traditional herbal medicine in China, Korea and Japan for thousands of years and today is a popular and worldwide used natural medicine. The active ingredients of ginseng are ginsenosides which are also called ginseng saponins. Recently, there is increasing evidence in the literature on the pharmacological an...

Advancing Chimeric Antigen Receptor-Engineered T-Cell Immunotherapy Using Genome Editing Technologies: Challenges and Future Prospects

Chimeric antigen receptor engineered-T (CAR-T) cells also named as living drugs, have been recently known as a breakthrough technology and were applied as an adoptive immunotherapy against different types of cancer. They also attracted widespread interest because of the success of B-cell malignancy therapy achieved by anti-CD19 CAR-T cells. Current genetic toolbox enabled the synthesis of CARs ...

Monomeric site-specific nucleases for genome editing.

Targeted manipulation of complex genomes often requires the introduction of a double-strand break at defined locations by site-specific DNA endonucleases. Here, we describe a monomeric nuclease domain derived from GIY-YIG homing endonucleases for genome-editing applications. Fusion of the GIY-YIG nuclease domain to three-member zinc-finger DNA binding domains generated chimeric GIY-zinc finger ...