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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Book ChapterDOI
Epigenetik: Biologische, medizinische, soziale und ethische Herausforderungen
TL;DR: Theoretische Fragestellungen im Hinblick auf Biologie and Medizin betreffen ihren status im Nexus zwischen Nukleotidsequenz and Genregulation.
Journal ArticleDOI
Therapeutic Applications of the CRISPR-Cas System
TL;DR: Given its promising results, the successful implementation of the CRISPR-Cas system in clinical practice will require further investigation into its therapeutic applications, and the most recent treatment trends and limitations are discussed.
Patent
System comprising a nucleotide sequence encoding a volvox carteri light-activated ion channel protein (VCHR1)
TL;DR: In this paper, a light-activated ion channel molecule that responds to a light stimulus is implemented using a light activated ion channel that does not activate a ChR2 ion channel.
OtherDOI
Genome‐Editing Applications in Stem Cell Engineering and Regenerative Medicine
TL;DR: In this article , the authors discuss the use of CCR5 and CXCR4 as targets used in genome editing in designing therapies for HIV and discuss current progress in development of treatments for neurodegenerative diseases followed by cystic fibrosis, DMD, and certain interesting stem cell therapy strategies for ocular diseases.
References
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Journal ArticleDOI
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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
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.