Viral vectors provide an efficient means for modification of eukaryotic cells, and their use is now commonplace in academic laboratories and industry for both research and clinical gene therapy applications. Lentiviral vectors, derived from the human immunodeficiency virus, have been extensively investigated and optimized over the past two decades. Third-generation, self-inactivating lentiviral vectors have recently been used in multiple clinical trials to introduce genes into hematopoietic stem cells to correct primary immunodeficiencies and hemoglobinopathies. These vectors have also been used to introduce genes into mature T cells to generate immunity to cancer through the delivery of chimeric antigen receptors (CARs) or cloned T-cell receptors. CAR T-cell therapies engineered using lentiviral vectors have demonstrated noteworthy clinical success in patients with B-cell malignancies leading to regulatory approval of the first genetically engineered cellular therapy using lentiviral vectors. In this review, we discuss several aspects of lentiviral vectors that will be of interest to clinicians, including an overview of lentiviral vector development, the current uses of viral vectors as therapy for primary immunodeficiencies and cancers, large-scale manufacturing of lentiviral vectors, and long-term follow-up of patients treated with gene therapy products.
Response to 'Evolving M-protein pattern in patients with smoldering multiple myeloma: impact on early progression'
Response to 'Evolving M-protein pattern in patients with smoldering multiple myeloma: impact on early progression', Published online: 25 May 2018; doi:10.1038/s41375-018-0155- ... more
Coppery inks paint an underwater rainbow
Coppery inks paint an underwater rainbow, Published online: 25 May 2018; doi:10.1038/d41586-018-05282-y
Inexpensive version of luminescent pigment shrugs off water. more
Previous studies suggested that MeCP2 competes with linker histone H1, but this hypothesis has never been tested in vivo. Here, we performed chromatin immunoprecipitation followed by sequencing (ChIP-seq) of Flag-tagged-H1.0 in mouse forebrain excitatory neurons. Unexpectedly, Flag-H1.0 and ... more
Olympus Microscopy has partnered with the Nature Publishing Group to present the Neurotechniques Collection. This compilation of articles will draw together some of the groundbreaking research that has recently been published in Nature Reviews Neuroscience and Nature Methods. Sponsored by O ... more