Journal ArticleDOI
State-of-the-art gene-based therapies: the road ahead.
Reads0
Chats0
TLDR
Improvements in the gene transfer vectors used in therapeutic trials have led to substantial clinical successes in patients with serious genetic conditions, such as immunodeficiency syndromes, blindness and some cancer types.Abstract:
Improvements in the gene transfer vectors used in therapeutic trials have led to substantial clinical successes in patients with serious genetic conditions, such as immunodeficiency syndromes, blindness and some cancer types. Several barriers need to be overcome before this type of therapy becomes a widely accepted treatment for a broad group of medical diseases. However, recent progress in the field is finally realizing some of the promises made more than 20 years ago, providing optimism for additional successes in the near future.read more
Citations
More filters
Journal ArticleDOI
Modeling Inborn Errors of Hepatic Metabolism Using Induced Pluripotent Stem Cells.
TL;DR: A brief overview of the state-of-the-art in using pluripotent stem cells for hepatic metabolism studies and how such cell models could potentially provide a platform for the discovery of therapeutics is presented.
Journal ArticleDOI
Human Muscle Precursor Cells Overexpressing PGC-1α Enhance Early Skeletal Muscle Tissue Formation.
Deana Haralampieva,Souzan Salemi,Ivana Dinulovic,Tullio Sulser,Simon M. Ametamey,Christoph Handschin,Daniel Eberli +6 more
TL;DR: By genetically modifying hMPCs to overexpress PGC-1α, this research was able to promote early muscle fiber formation in vitro and in vivo, with an initial switch to slow-type myofibers.
Construction of stable packaging cell lines for clinical lentiviral vector
Sean Knight,Sam L. Stephen,Ranbir Bailey,David Escors,Jeremy Minshull,Giorgia Santilli,Adrian J. Thrasher,Mary Collins,Yasuhiro Takeuchi +8 more
TL;DR: A general method for construction of stable packaging cell lines that continuously produce lentiviral vectors using Cre recombinase-mediated cassette exchange to insert a codon-optimised HIV-1 Gag-Pol expression construct in a continuously expressed locus in 293FT cells is described.
Journal ArticleDOI
Synthesis at the interface of virology and genetic code expansion.
TL;DR: This review seeks to provide an account of the recent advances at the interface of synthetic biology and genetic code expansion technology, and holds much promise to advance their individual progress, while creating new paradigms for synthetic biology.
Journal ArticleDOI
Zinc ion coordination significantly improved the transfection efficiency of low molecular weight polyethylenimine.
Huapan Fang,Huapan Fang,Lin Lin,Jie Chen,Jie Chen,Jiayan Wu,Jiayan Wu,Huayu Tian,Huayu Tian,Xuesi Chen,Xuesi Chen +10 more
TL;DR: A novel and high-performance polycationic gene carrier was constructed by introducing a dipicolylamine compound along with zinc ion coordination onto polyethylenimine (PEI1.8k) via Schiff base bonds, which exhibited efficient transfection and negligible cytotoxicity.
References
More filters
Journal ArticleDOI
Gefitinib or Carboplatin–Paclitaxel in Pulmonary Adenocarcinoma
Tony Mok,Yi-Long Wu,Sumitra Thongprasert,Chih-Hsin Yang,Da Tong Chu,Nagahiro Saijo,Patrapim Sunpaweravong,Baohui Han,Benjamin Margono,Benjamin Margono,Yukito Ichinose,Yutaka Nishiwaki,Yuichiro Ohe,Jin Ji Yang,Busyamas Chewaskulyong,Haiyi Jiang,Emma Duffield,Claire Watkins,Alison Armour,Masahiro Fukuoka +19 more
TL;DR: Gefit inib is superior to carboplatin-paclitaxel as an initial treatment for pulmonary adenocarcinoma among nonsmokers or former light smokers in East Asia and the presence in the tumor of a mutation of the EGFR gene is a strong predictor of a better outcome with gefitinib.
Journal ArticleDOI
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Salima Hacein-Bey-Abina,C von Kalle,C von Kalle,Manfred Schmidt,Matthew P. McCormack,NM Wulffraat,Philippe Leboulch,Annick Lim,Cameron S. Osborne,R. Pawliuk,Estelle Morillon,R. Sorensen,A. Forster,Peter Fraser,Jeffrey I. Cohen,G de Saint Basile,Ian E. Alexander,Uwe Wintergerst,Thierry Frebourg,Alain Aurias,Dominique Stoppa-Lyonnet,Serge Romana,I. Radford-Weiss,Fabian Gross,Françoise Valensi,Eric Delabesse,Elizabeth Macintyre,F. Sigaux,Jean Soulier,L. E. Leiva,Manuela Wissler,Claudia Prinz,Terence H. Rabbitts,F. Le Deist,Alain Fischer,Marina Cavazzana-Calvo +35 more
TL;DR: Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.
Journal ArticleDOI
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
Marina Cavazzana-Calvo,Hacein-Bey S,Geneviève de Saint Basile,Fabian Gross,Eric Yvon,Patrick Nusbaum,Françoise Selz,Christophe Hue,Stéphanie Certain,Jean-Laurent Casanova,Philippe Bousso,Françoise Le Deist,Alain Fischer +12 more
TL;DR: A gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gammac Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells, which provided full correction of disease phenotype and clinical benefit.
Journal ArticleDOI
Progress and problems with the use of viral vectors for gene therapy
TL;DR: With the development of a leukaemia-like syndrome in two patients cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go.
Journal ArticleDOI
Genome editing with engineered zinc finger nucleases
TL;DR: A broad range of outcomes has resulted from the application of the same core technology: targeted genome cleavage by engineered, sequence-specific zinc finger nucleases followed by gene modification during subsequent repair.