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

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 ...

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 ...

These concerns are exacerbated by observations of cellular immune responses and other adverse events, including detrimental off-target transgene expression in ... of a vector based on a synthetic AAV ...

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

significantly shrinking the donor pool for organ transplantation. Performing CRISPR screens in the grafts will accelerate the discovery of resistance target genes to immune pressure. Additionally, ...

These cell types exhibit the functional characteristics of their respective modeled organs, which can be verified ... Both organoids were promising models for AAV screening for gene therapy ...