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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Journal ArticleDOI
Influence of DNA sequence on the structure of minicircles under torsional stress.
Qian Wang,Rossitza N. Irobalieva,Wah Chiu,Michael F. Schmid,Jonathan M. Fogg,Lynn Zechiedrich,B. Montgomery Pettitt,B. Montgomery Pettitt +7 more
TL;DR: It is found that DNA minicircle topoisomers can have multiple bend locations under high torsional stress and that the positions of these sharp bends are determined by the sequence, and by a positive mechanical correlation along the sequence.
Journal ArticleDOI
DNA Ministrings: Highly Safe and Effective Gene Delivery Vectors
Nafiseh Nafissi,Samih Alqawlaq,Eric A. Lee,Marianna Foldvari,Paul A. Spagnuolo,Roderick A. Slavcev +5 more
TL;DR: Ministring DNA that integrated into the genome of human cells caused chromosomal disruption and apoptotic death of possibly oncogenic vector integrants; thus, they may be safer than plasmid and circular DNA vectors.
Journal ArticleDOI
Gene therapy for neurological disorders: challenges and recent advancements.
Stefanie A. Pena,Rahul Iyengar,Rebecca S. Eshraghi,Nicole Bencie,Jeenu Mittal,Abdulrahman Aljohani,Rahul Mittal,Adrien A. Eshraghi +7 more
TL;DR: An overview of the latest advances in targeted gene delivery is presented and the challenges and future direction of gene therapy in the treatment of neurological disorders are discussed.
Journal ArticleDOI
Gene therapy for haemophilia: Prospects and challenges to prevent or reverse inhibitor formation
David W. Scott,Jay N. Lozier +1 more
TL;DR: This review will describe the laboratory and clinical progress, and the challenges met thus far, in achieving the goal of gene therapy efficacy, with a focus on thegoal of tolerance induction.
Journal ArticleDOI
Postprandial dyslipidaemia and diabetes: mechanistic and therapeutic aspects.
TL;DR: Regulating the plasma concentrations of triglyceride-rich lipoproteins may decrease the cardiovascular complications of diabetes and the mechanisms of action of incretin-based treatments on dyslipidaemia and endothelial dysfunction need further investigation.
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.