Marina O'Reilly

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 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: 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.

TL;DR: A transparent oversight system that extends to human gene transfer protocols that are either conducted with NIH funding or conducted at institutions that receive NIH funding for recombinant DNA research has fostered the development of retroviral, lentiviral, and adeno-associated viral vector mediated gene delivery.

TL;DR: The challenges and opportunities for advancing the field including means for enhancing data sharing for preclinical and clinical studies, development and utilization of available NIH resources, and interactions with the U.S. Food and Drug Administration are discussed.