Jacqueline Corrigan-Curay

TL;DR: Recently developed genomic editing technologies have the potential to be powerful tools for gene therapy because of their ability to inactivate genes, correct mutated sequences, or insert intact genes While the genomic editing field is advancing at an exceptionally rapid pace, there remain key issues regarding development of appropriate preclinical assays to evaluate off-target effects and establish safety.

TL;DR: The Recombinant DNA Advisory Committee held a symposium to first review the reported treatment-associated toxicities and, second, to discuss methods for improving safety and efficacy, with a specific focus on protocols infusing CAR T cells.

TL;DR: The T Cell Immunotherapy: Optimizing T-cell trial design workshop as discussed by the authors was a workshop sponsored by the National Institutes of Health's Office of Biotechnology Activities (OBA), which brought together researchers to discuss the scientific advances and share new data on key trial design issues.

TL;DR: A framework of points for investigators to consider when designing trials that involve direct delivery of a therapeutic agent to the CNS, and the need to minimise bias and confounds is developed.

TL;DR: Recent reports of insertional mutagenesis leading to myelodysplastic syndrome in a trial for chronic granulomatous disease and a case of leukemia in a Trial for Wiskott-Aldrich syndrome, both of which used g-retroviral vectors, underscored that this type of toxicity can also apply to other disease settings.