Journal ArticleDOI
The Basic Science of Gene Therapy
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TLDR
A large number of key technical issues need to be resolved before gene therapy can be safely and effectively applied in the clinic, and future technological developments will be critical for the successful practice of gene therapy.Abstract:
The development over the past decade of methods for delivering genes to mammalian cells has stimulated great interest in the possibility of treating human disease by gene-based therapies. However, despite substantial progress, a number of key technical issues need to be resolved before gene therapy can be safely and effectively applied in the clinic. Future technological developments, particularly in the areas of gene delivery and cell transplantation, will be critical for the successful practice of gene therapy.read more
Citations
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Journal ArticleDOI
In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
Luigi Naldini,Ulrike Blömer,Philippe Gallay,Daniel S. Ory,Richard C. Mulligan,Fred H. Gage,Inder M. Verma,Didier Trono +7 more
TL;DR: The ability of HIV-based viral vectors to deliver genes in vivo into nondividing cells could increase the applicability of retroviral vectors in human gene therapy.
PatentDOI
Self-assembly and mineralization of peptide-amphiphile nanofibers
TL;DR: In this paper, pH-induced self-assembly of a peptide-amphiphile was used to make a nanostructured fibrous scaffold reminiscent of extracellular matrix.
Journal ArticleDOI
Gene therapy -- promises, problems and prospects
Inder M. Verma,Nikunj V. Somia +1 more
TL;DR: The prospects are good — by the year 2010, gene therapy may be as routine a practice as heart transplants are today.
PatentDOI
Production of high titer helper-free retroviruses by transient transfection
TL;DR: In this article, a method for producing high-titer, helper-free infectious retroviruses is disclosed which employs a novel strategy that uses transient transfection of new retroviral producer cell lines, ecotropic line BOSC 23 and amphotropic line CAK 8.
Journal ArticleDOI
Design and development of polymers for gene delivery
TL;DR: With the growing understanding of polymer gene-delivery mechanisms and continued efforts of creative polymer chemists, it is likely that polymer-based gene-Delivery systems will become an important tool for human gene therapy.
References
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