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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Synthetic Nucleic Acid Analogues in Gene Therapy: An Update for Peptide–Oligonucleotide Conjugates
TL;DR: The main objective of this work is to provide an update on synthetic nucleic acid analogues and nanoassemblies as tools in gene therapy, focusing on peptide–oligonucleotide conjugates (POCs) and compares their efficiency.
Journal ArticleDOI
Specific targeting of point mutations in EGFR L858R-positive lung cancer by CRISPR/Cas9
Alvin Ho-Kwan Cheung,Chit Chow,Jinglin Zhang,Yuhang Zhou,Tingting Huang,Kayla Ching-Kei Ng,Terry Cho-Tsun Or,Yoyo Yao Yao,Yujuan Dong,Jackie Mei-Wah Fung,Lei Xiong,Aden Ka-Yin Chan,Wai-Ming Raymond Lung,Wei Kang,Ka Fai To +14 more
TL;DR: It is found that a therapeutic oligonucleotide-based strategy, which utilized PAM to differentiate cancer mutation from normal, afforded high specificity to the extent of a single nucleotide substitution, and may serve as a useful approach to target cancer-defining mutations with specificity.
Journal ArticleDOI
Bacteriophage-derived vectors for targeted cancer gene therapy.
Pranjol Mz,Amin Hajitou +1 more
TL;DR: The current progress and challenges in the targeted gene therapy of cancer are summarized and the recent developments of bacteriophage-derived vectors and their contributions in targeting cancer with therapeutic genes following systemic administration are highlighted.
Journal ArticleDOI
The Cleaved N-Terminus of pVI Binds Peripentonal Hexons in Mature Adenovirus
Joost Snijder,Marco Benevento,Crystal L. Moyer,Vijay S. Reddy,Glen R. Nemerow,Albert J. R. Heck +5 more
TL;DR: A combination of proteomics-based peptide identification, native mass spectrometry and hydrogen-deuterium exchange mass Spectrometry is used to show that pVIn is associated with mature human adenovirus, where it binds at the base of peripentonal hexons in a pH-dependent manner.
Journal ArticleDOI
Bait-and-Switch Supramolecular Strategy To Generate Noncationic RNA–Polymer Complexes for RNA Delivery
Ziwen Jiang,Wei Cui,Priyaa Prasad,Mollie A. Touve,Mollie A. Touve,Nathan C. Gianneschi,Nathan C. Gianneschi,Jesse Mager,Sankaran Thayumanavan +8 more
TL;DR: An in situ electrostatic complexation using a methylated pyridinium group is used to generate noncationic RNA-polymer complexes for RNA delivery with low cytotoxicity, demonstrating successful knockdown in preimplantation mammalian embryos, thus providing a new approach for nucleic acid delivery.
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.