Journal ArticleDOI
State-of-the-art gene-based therapies: the road ahead.
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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
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Evaluation of BMP-2 Minicircle DNA for Enhanced Bone Engineering and Regeneration
Ara Hacobian,David Hercher,Benedikt Regner,Amelie Frischer,Simon Sperger,Heinz Redl,Alice Zimmermann +6 more
TL;DR: A highly bioactive BMP-2 minicircle plasmid with the potential to fulfill requirements for clinical translation in the field of bone regeneration is presented and shows suitability for stem cell approaches by showing significantly higher levels of ALP activity and mineralization when introduced into human bone marrow stem cells.
Patent
Optogenetic method for generating an inhibitory current in a mammalian neuron
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Transferencia génica hepática hidrodinámica en modelos de cerdo in vivo y segmento humano ex vivo
TL;DR: In this paper, a vehiculo utilizado for entregar el gen is described, and three models of transferencia genica hepatica are presented.
Journal ArticleDOI
EDTA‐Capped Iron Oxide Core‐Corona System as Vehicle for Gene Delivery to Transform E.coli: Mimicking the Lipid Bilayer Environment
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La biología sintética y el imperativo de mejoramiento
TL;DR: The authors discusses the main arguments offered in this debate, and points out some of the most problematic assumptions in recent proposals concerning human bioenhancement, and discusses the problem of human bio-enhancements.
References
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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.