CRISPR gene editing is a promising therapy for treating rare genetic diseases. Delivering this therapy directly to patients is challenging because the CRISPR cargo needs protection from degradation. For this reason, most successful CRISPR clinical trials use patient-derived cells that are corrected in vitro. New approaches in nanoparticle and virus delivery methods make it possible to edit disease-causing genes directly in the body. In this webinar, researchers will discuss in vivo gene editing advancements that open the door for treating a wide range of genetic disorders.

Topics to be covered

• CRISPR-Cas9 delivery vehicles for in vivo editing
• How to minimize off-target CRISPR-induced mutations
• Ameliorating behavior deficits in Fragile X syndrome

Speaker Information: