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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Overexpression of Ad5 precursor terminal protein accelerates recombinant adenovirus packaging and amplification in HEK-293 packaging cells
Ningning Wu,Zhang H,Deng F,Li R,Zhang W,Xian Chen,Wen S,Wang N,Zhang J,Yin L,Liao Z,Zhen Zhang,Zhang Q,Yan Z,Liu W,Wu D,Ye J,Deng Y,Yang K,Hue H. Luu,Rex C. Haydon,Tong-Chuan He +21 more
TL;DR: It is suggested that exogenous pTP expression in 293 cells may accelerate the packaging and amplification of recombinant adenovirus production.
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Aptamer functionalized nanomaterials for biomedical applications: Recent advances and new horizons
Mansour Mahmoudpour,Shichao Ding,Zhaoyuan Lyu,Ghasem Ebrahimi,Dan Du,Jafar Ezzati Nazhad Dolatabadi,Mohammadali Torbati,Yuehe Lin +7 more
TL;DR: The versatile roles of nanomaterials in advanced AFMs are discussed, including optical indicators, conducting transducer, carriers, catalysts, and other functions, and some challenges and opportunities in this emerging field are presented.
Journal ArticleDOI
Oligopeptide-terminated poly(β-amino ester)s for highly efficient gene delivery and intracellular localization
TL;DR: New polycations for gene delivery based on poly(β-amino ester)s (pBAE) containing terminal oligopeptides that achieve better cellular viability and higher transfection efficacy than other end-modified pBAE and commercial transfections agents are described.
Journal ArticleDOI
In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges
TL;DR: The most recent progress of in vivo delivery of CRISPR-Cas9 systems is discussed, innovative viral and non-viral delivery technologies are highlighted, outstanding delivery challenges are emphasized, and the most updated perspectives are provided.
Reference EntryDOI
Gene Targeting by Homologous Recombination
Wade A. Reh,Karen M. Vasquez +1 more
TL;DR: Homology-directed gene targeting utilises homologous recombination to introduce defined modifications into sequences of interest in mammalian genomes, and most genes in a variety of mammalian species can now be manipulated by homology- directed gene targeting.
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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.