Eugene Rosenthal

TL;DR: Observations suggest the feasibility of in vivo CFTR gene transfer as therapy for the pulmonary manifestations of CF.

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: First-generation adenovirus vectors that had been rendered replication defective by removal of the E1 region of the viral genome or lacking the Ad E3 region in addition to E1 sequences induced G2 cell cycle arrest and inhibited traverse across G1/S in primary and immortalized human bronchial epithelial 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: 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.