Journal ArticleDOI
Gene therapy clinical trials worldwide to 2017: An update
Samantha L. Ginn,Anais K. Amaya,Ian E. Alexander,Ian E. Alexander,Michael Edelstein,Mohammad R. Abedi +5 more
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TLDR
This review presents the analysis of clinical trials that, to the best of the knowledge, have been or are being performed worldwide, and discusses key trends since the previous review, namely the use of chimeric antigen receptor T cells for the treatment of cancer and advancements in genome editing technologies.Abstract:
To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical activity from trial databases, official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. As of our November 2017 update, we have entries on 2597 trials undertaken in 38 countries. We have analysed the geographical distribution of trials, the disease indications (or other reasons) for trials, the proportions to which different vector types are used, and the genes that have been transferred. Details of the analyses presented, and our searchable database are available via The Journal of Gene Medicine Gene Therapy Clinical Trials Worldwide website at: http://www.wiley.co.uk/genmed/clinical. We also provide an overview of the progress being made in gene therapy clinical trials around the world, and discuss key trends since the previous review, namely the use of chimeric antigen receptor T cells for the treatment of cancer and advancements in genome editing technologies, which have the potential to transform the field moving forward.read more
Citations
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Biopharmaceutical benchmarks 2018.
TL;DR: The market for monoclonal antibodies continues to reign supreme, although cellular and gene therapies are slowly starting to gather momentum, andgeoning growth in biosimilars may threaten future brand monopolies.
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Lipid Nanoparticles-From Liposomes to mRNA Vaccine Delivery, a Landscape of Research Diversity and Advancement.
TL;DR: Liposomes, an early version of LNPs, are a versatile nanomedicine delivery platform for treatment of a variety of diseases, such as antitumor and nucleic acid therapeutics and vaccine delivery systems as mentioned in this paper.
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Innovative approaches for cancer treatment: current perspectives and new challenges.
TL;DR: In this review, an in-depth analysis of the most innovative advances in basic and applied cancer research is provided.
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Correction of a pathogenic gene mutation in human embryos
Hong Ma,Nuria Marti-Gutierrez,Park Sw,Jun Wu,Young Hee Lee,Keiichiro Suzuki,Amy Koski,Ji D,Tomonari Hayama,Riffat Ahmed,Hayley Darby,Van Dyken C,Yang Li,Kang E,Park Ar,Kim D,Sangtae Kim,Gong J,Ying Gu,David Battaglia,Sacha A. Krieg,David M. Lee,Wu Dh,Don P. Wolf,Stephen B. Heitner,Belmonte Jci,Paula Amato,Jin-Soo Kim,Sanjiv Kaul,Shoukhrat Mitalipov +29 more
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Entering the Modern Era of Gene Therapy.
TL;DR: This review focuses on the clinical research that has forged the gene therapy field as it currently stands, and the strategies being translated from the lab to the clinic.
References
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Journal ArticleDOI
Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors.
TL;DR: Induction of pluripotent stem cells from mouse embryonic or adult fibroblasts by introducing four factors, Oct3/4, Sox2, c-Myc, and Klf4, under ES cell culture conditions is demonstrated and iPS cells, designated iPS, exhibit the morphology and growth properties of ES cells and express ES cell marker genes.
Journal ArticleDOI
Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
Andrew Fire,SiQun Xu,Mary K. Montgomery,Steven A. Kostas,Steven A. Kostas,Samuel E. Driver,Craig C. Mello +6 more
TL;DR: To their surprise, it was found that double-stranded RNA was substantially more effective at producing interference than was either strand individually, arguing against stochiometric interference with endogenous mRNA and suggesting that there could be a catalytic or amplification component in the interference process.
Journal ArticleDOI
A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.
Martin Jinek,Krzysztof Chylinski,Krzysztof Chylinski,Ines Fonfara,Michael H. Hauer,Jennifer A. Doudna,Emmanuelle Charpentier +6 more
TL;DR: This study reveals a family of endonucleases that use dual-RNAs for site-specific DNA cleavage and highlights the potential to exploit the system for RNA-programmable genome editing.
Journal ArticleDOI
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,Le Cong,F. Ann Ran,F. Ann Ran,David M. Cox,David M. Cox,Shuailiang Lin,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +15 more
TL;DR: The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage as discussed by the authors.
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,F. A. Ran,David Benjamin Turitz Cox,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +10 more
TL;DR: Two different type II CRISPR/Cas systems are engineered and it is demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.
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