Organoids were cultured from healthy donor iPSC sources and differentiated using the human iPSC-derived Cerebral Organoid ...

The team then optimizes the dose for IV delivery of the AAV vectors to target the cells that are critical to treating the disease of interest. “We believe that optimizing each of the capsid and ...

are attracted to off target tissues leading to unintended consequences. Further, traditional approaches of AAV capsid shuffling and rational design have not resulted in the predicted advances ...

Cardiac editing reached 15%, and off-target editing in the liver remained below 2%. NanoCas represents the first single-AAV CRISPR system to demonstrate efficient in vivo muscle editing in non ...

To flip the ratio and bias an AAV away from toxic hotspots and towards a target organ or cell, researchers like Deverman, Gradinaru, and Davidson have developed capsid engineering strategies to put ...

Due to these barriers in getting the genetic payload to the correct target cells ... vivo delivery via a single adeno-associated viral (AAV) vector. Now, new preclinical research presents ...

The most efficient delivery systems today are those based on a harmless virus named adeno-associated virus (AAV) that has a natural ... would affect a major organ, the liver—something that ...

Existing methods of engineering one of the most commonly used gene-delivery vehicles, adeno-associated viruses (AAV), are often slow ... cargo to a specific organ but not others or to work in ...

The company will discontinue development of its original ALS gene therapy candidate VY9323 amid toxic effects in nonhuman ...