Update on advances in gene therapy From National Center for Advancing Translational Sciences

(Mary Ann Liebert, Inc./Genetic Engineering News) New initiatives by the National Center for Advancing Translational Sciences (NCATS) to use gene therapy approaches to treat rare diseases and especially promising aspects of gene transfer and gene editing technology, such as adeno-associated viral (AAV) vectors and CRISPR-Cas9 are highlighted in an editorial published in Human Gene Therapy.