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
Citations
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Transient loading of CD34+ hematopoietic progenitor cells with polystyrene nanoparticles
Sarah Deville,Wahyu Wijaya Hadiwikarta,Nick Smisdom,Bart Wathiong,Marcel Ameloot,Inge Nelissen,Jef Hooyberghs +6 more
TL;DR: It is demonstrated that a transient association of the NPs with HPCs is observed, reaching a maximum within 1 hour and declining afterwards, suggesting active loading and release of NPs by HPCe, and a simple theoretical approach modeling the kinetics of NPloading and release is presented.
Journal ArticleDOI
Improvement of DNA Vector Delivery of DOTAP Lipoplexes by Short-Chain Aminolipids.
Jonas Buck,Dennis Mueller,Ute Mettal,Ute Mettal,Miriam Ackermann,Hiu Man Grisch-Chan,Beat Thöny,Andreas Zumbuehl,Jörg Huwyler,Dominik Witzigmann,Dominik Witzigmann +10 more
TL;DR: A one-pot combinatorial method based on double-reductive amination for the synthesis of short-chain aminolipids showed twofold enhanced gene delivery and reduced toxicity compared to the native DOTAP:cholesterol lipoplexes and enabled enhanced transgene expression in vivo in the zebrafish embryo model.
Journal ArticleDOI
Single-Tailed Lipidoids Enhance the Transfection Activity of Their Double-Tailed Counterparts
TL;DR: This study uses combinatorial chemistry to synthesize 17 double-tail and 17 single-tail lipidoids using thiol-yne and thio-ene click chemistry and shows that combining single with double-tails lipidoids increases uptake of lipoplexes, as well as cellular transfection efficiency.
Journal ArticleDOI
Advanced nanotherapies to promote neuroregeneration in the injured newborn brain
TL;DR: Current efforts that promote neuroprotection and potential targets that are unique to the developing brain, which can be leveraged to facilitate neuroregeneration are described.
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Pushing the Right Buttons: Improving Efficacy of Therapeutic DNA Vectors
Ara Hacobian,David Hercher +1 more
TL;DR: This review summarizes different DNA vector modifications to enhance biological efficacy and efficiency of therapeutical vectors, aiming for low toxicity, high specificity, and biological efficacy—the cornerstones for successful translation of gene therapy into the clinic.
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.