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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Book ChapterDOI
Viral Vectors for Muscle Gene Therapy
TL;DR: This chapter summarizes how AAV vectors based on natural isolates contributed to the continuing success of human muscle gene therapy and introduces basic AAV biology that pertains to the vectorology of AAV for gene therapy.
Patent
Use of pon gene cluster in preparing medicament for treating atherosclerosis
TL;DR: In this paper, the use of PON gene cluster in preparing medicament for treating atherosclerosis in mammals, wherein the Pon gene cluster treat atheros sclerosis by promoting stability of atherosclerotic plaque.
Non-embryo-destructive Extraction of Pluripotent Embryonic Stem Cells: Implications for Regenerative Medicine and Reproductive Medicine Embryonenerhaltende Gewinnung pluripotenter Stammzellen: Implikationen für die regenerative Medizin und die Reproduktionsmedizin
R. Dittrich,M. W. Beckmann +1 more
TL;DR: A technique for the non-embryo-destructive extraction of pluripotent embryonic stem cells together with potential future applications for stem cells harvested in this manner is reported here on.
Patent
Delivery of a light-activated cation channel into the brain of a subject
Karl Deisseroth,Edward S. Boyden +1 more
TL;DR: In this paper, the authors provide compositions and methods for light-activated cation channel proteins and their uses within cell membranes and subcellular regions, including proteins, nucleic acids, vectors, and methods to genetically targeted expression of lightactivated Cation channels to specific cells or defined cell populations.
Patent
Composition for gene therapy of the central nervous system, process of production and use thereof
Roselena Silvestri Schuh,Ferreira Teixeira Helder,Guilherme Baldo,Ursula da Silveira Matte,Roberto Giugliani,Juliana Bidone +5 more
TL;DR: In this paper, a composition for gene therapy of the central nervous system comprising non-viral carriers of nanometric size (1.0 micrometer) complexed with at least one nucleic acid for purposes of gene therapy via nasal administration was described.
References
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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
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Journal ArticleDOI
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
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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
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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.