R ecently 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 evalu-ate off-target effects and establish safety. In order to begin a dialogue on these issues, the National Institutes of Health (NIH) Office of Science Policy, in collaboration with several NIH-funded investigators and the NIH Recombinant DNA Advisory Committee, organized a workshop on 10 June 2014, in Bethesda, Maryland, to provide a forum to educate the scientific and oversight communities and the public on different genome editing technologies, clinical experiences to date, and the preclinical assays being developed to examine the precision of these tools and their suitability for clinical application.Targeted genome modification by designer nucleases is an emerging technology that can be used to investigate gene function and could also be used to treat genetic or acquired diseases. A wide range of genome alterations has been achieved by these nucleases, including localized mutagenesis, local and dispersed sequence replacements, large and small insertions and deletions, and even chromosomal translocations. The nuclease approach to targeted genome editing has been applied successfully to more than 50 different organisms, including crop plants, livestock, and humans. 1 Recently developed genome editing technologies such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), meganucleases, and clustered regularly interspaced short

T he rapidly expanding field of T-cell immunotherapy has experienced clinical successes along with some serious toxicities. "T Cell Immunotherapy: Optimizing Trial Design, " a workshop sponsored by the National Institutes of Health's (NIH's) Office of Biotechnology Activities (OBA), brought together researchers to discuss the scientific advances and share new data on key trial design issues, including the selection of new targets, optimizing the T-cell population, preconditioning regimens, strategies to promote persistence of cells, and analysis and management of acute reactions to T-cell infusions with the goal of identifying best practices and a research agenda that will facilitate further development and maximize the safety of this promising approach.

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Gene therapy has shown clinical efficacy for several rare diseases, using different approaches and vectors. The Gene Therapy for Rare Diseases workshop, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities and Office of Rare Diseases Research, brought together investigators from different disciplines to discuss 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.