Journal ArticleDOI
State-of-the-art gene-based therapies: the road ahead.
Reads0
Chats0
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
More filters
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. [5YIF: 27.14; Citations: 298]
Fulvio Mavilio,Graziella Pellegrini,Sergio Ferrari,Francesca Di Nunzio,E Di Iorio,Alessandra Recchia,Giulietta Maruggi,Giuliana Ferrari,Elena Provasi,Chiara Bonini,Sergio Capurro,Andrea Conti,Cristina Magnoni,Alberto Giannetti,Michele De Luca +14 more
TL;DR: In this paper, a retroviral vector expressing LAMB3 cDNA (encoding LAM5-β3) was used to prepare genetically corrected cultured epidermal grafts, which were transplanted onto surgically prepared regions of the patient's legs.
Journal ArticleDOI
CRISPR technologies for stem cell engineering and regenerative medicine.
TL;DR: In vitro stem cell fate manipulation and in vivo applications such as prevention of retinal and muscular degeneration, neural regeneration, bone regeneration, cartilage tissue engineering, and treatment of diseases in blood, skin and liver are focused on.
Journal ArticleDOI
Synthetic riboswitches for external regulation of genes transferred by replication-deficient and oncolytic adenoviruses
TL;DR: This study demonstrates the fidelity of aptazymes in viral vectors and oncolytic viruses and highlights the potency of riboswitches for medical applications.
Journal ArticleDOI
Targeted siRNA Delivery Using a Lipo-Oligoaminoamide Nanocore with an Influenza Peptide and Transferrin Shell.
Wei Zhang,Katharina Müller,Eva Kessel,Eva Kessel,Sören Reinhard,Dongsheng He,Dongsheng He,Philipp M. Klein,Miriam Höhn,Wolfgang Rödl,Susanne Kempter,Ernst Wagner,Ernst Wagner +12 more
TL;DR: In vivo distribution studies not only demonstrate an enhanced tumor residence of siRNA in N2a tumor‐bearing mice with the Tf&INF7 as compared to the 454 polyplex group but also a reduced siRNA nanoparticle stability limiting the in vivo performance.
Journal ArticleDOI
Interleukin-13 immune gene therapy prevents CNS inflammation and demyelination via alternative activation of microglia and macrophages
Caroline Guglielmetti,Debbie Le Blon,Eva Santermans,Angélica Salas-Perdomo,Jasmijn Daans,Nathalie De Vocht,Disha Shah,Chloé Hoornaert,Jelle Praet,Jurgen Peerlings,Firat Kara,Christian Bigot,Zhenhua Mai,Zhenhua Mai,Herman Goossens,Niel Hens,Niel Hens,Sven Hendrix,Marleen Verhoye,Anna M. Planas,Zwi N. Berneman,Annemie Van der Linden,Peter Ponsaerts +22 more
TL;DR: Evidence is provided that lentiviral vector‐mediated expression of the immune‐modulating cytokine interleukin 13 induces an alternative activation program in both microglia and macrophages conferring protection against severe oligodendrocyte loss and demyelination in the cuprizone mouse model for multiple sclerosis (MS).
References
More filters
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.