Journal ArticleDOI
Lentiviral vectors: basic to translational
TLDR
In the present high-throughput '-omics' era, the commercial availability of premade lentiviral vectors, which are engineered to express or silence genome-wide genes, accelerates the rapid expansion of this vector technology.Abstract:
More than two decades have passed since genetically modified HIV was used for gene delivery. Through continuous improvements these early marker gene-carrying HIVs have evolved into safer and more effective lentiviral vectors. Lentiviral vectors offer several attractive properties as gene-delivery vehicles, including: (i) sustained gene delivery through stable vector integration into host genome; (ii) the capability of infecting both dividing and non-dividing cells; (iii) broad tissue tropisms, including important gene- and cell-therapy-target cell types; (iv) no expression of viral proteins after vector transduction; (v) the ability to deliver complex genetic elements, such as polycistronic or intron-containing sequences; (vi) potentially safer integration site profile; and (vii) a relatively easy system for vector manipulation and production. Accordingly, lentivector technologies now have widespread use in basic biology and translational studies for stable transgene overexpression, persistent gene silencing, immunization, in vivo imaging, generating transgenic animals, induction of pluripotent cells, stem cell modification and lineage tracking, or site-directed gene editing. Moreover, in the present high-throughput '-omics' era, the commercial availability of premade lentiviral vectors, which are engineered to express or silence genome-wide genes, accelerates the rapid expansion of this vector technology. In the present review, we assess the advances in lentiviral vector technology, including basic lentivirology, vector designs for improved efficiency and biosafety, protocols for vector production and infection, targeted gene delivery, advanced lentiviral applications and issues associated with the vector system.read more
Citations
More filters
Journal ArticleDOI
Adenovirus-mediated gene delivery: Potential applications for gene and cell-based therapies in the new era of personalized medicine
Cody S. Lee,Elliot S. Bishop,Ruyi Zhang,Ruyi Zhang,Xinyi Yu,Xinyi Yu,Evan M. Farina,Shujuan Yan,Shujuan Yan,Chen Zhao,Chen Zhao,Zongyue Zeng,Zongyue Zeng,Yi Shu,Yi Shu,Xingye Wu,Xingye Wu,Jiayan Lei,Jiayan Lei,Yasha Li,Yasha Li,Wenwen Zhang,Wenwen Zhang,Chao Yang,Chao Yang,Ke Wu,Ke Wu,Ying Wu,Ying Wu,Sherwin S. W. Ho,Aravind Athiviraham,Michael J. Lee,Jennifer Moriatis Wolf,Russell R. Reid,Tong-Chuan He +34 more
TL;DR: It is expected that the continued improvements in adenoviral vectors should provide great opportunities for cell and gene therapies to live up to its enormous potential in personalized medicine.
Journal Article
Viral vectors: a look back and ahead on gene transfer technology
TL;DR: This review illustrates the strategies used to generate some of the most used viral vectors, and their advantages, limitations and principal applications.
Journal ArticleDOI
Molecular mechanisms of retroviral integration site selection
TL;DR: Approaches to alter integration site selection that could potentially improve the safety of retroviral vectors in the clinic are discussed.
Journal ArticleDOI
A time course analysis of the electrophysiological properties of neurons differentiated from human induced pluripotent stem cells (iPSCs).
Deborah Pre,Michael W. Nestor,Andrew A. Sproul,Samson T. Jacob,Peter Koppensteiner,Vorapin Chinchalongporn,Matthew Zimmer,Ai Yamamoto,Scott Noggle,Ottavio Arancio +9 more
TL;DR: The importance of properly evaluating the electrophysiological status of the newly generated neurons when using stem cell technology is demonstrated, as electrophYSiological properties of iPSC-derived neurons mature over time.
Journal ArticleDOI
Fluorescence optical imaging in anticancer drug delivery
TL;DR: The potential applications and limitations of non-invasive imaging techniques in the field of drug delivery, especially in anticancer therapy, are focused on and fluorescence imaging at both the cellular and systemic levels is discussed in detail.
References
More filters
Journal ArticleDOI
Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors
Kazutoshi Takahashi,Koji Tanabe,Mari Ohnuki,Megumi Narita,Tomoko Ichisaka,Kiichiro Tomoda,Shinya Yamanaka +6 more
TL;DR: It is demonstrated that iPS cells can be generated from adult human fibroblasts with the same four factors: Oct3/4, Sox2, Klf4, and c-Myc.
Journal ArticleDOI
Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
Andrew Fire,SiQun Xu,Mary K. Montgomery,Steven A. Kostas,Steven A. Kostas,Samuel E. Driver,Craig C. Mello +6 more
TL;DR: To their surprise, it was found that double-stranded RNA was substantially more effective at producing interference than was either strand individually, arguing against stochiometric interference with endogenous mRNA and suggesting that there could be a catalytic or amplification component in the interference process.
Journal ArticleDOI
Induced Pluripotent Stem Cell Lines Derived from Human Somatic Cells
Junying Yu,Maxim A. Vodyanik,Kim Smuga-Otto,Jessica Antosiewicz-Bourget,Jennifer L. Frane,Shulan Tian,Jeff Nie,Gudrun A. Jonsdottir,Victor Ruotti,Ron Stewart,Igor I. Slukvin,James A. Thomson +11 more
TL;DR: This article showed that OCT4, SOX2, NANOG, and LIN28 factors are sufficient to reprogram human somatic cells to pluripotent stem cells that exhibit the essential characteristics of embryonic stem (ES) cells.
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.
Related Papers (5)
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Nathalie Cartier,Salima Hacein-Bey-Abina,Cynthia C. Bartholomae,Gabor Istvan Veres,Manfred Schmidt,Ina Kutschera,Michel Vidaud,Ulrich Abel,Liliane Dal-Cortivo,Laure Caccavelli,Nizar Mahlaoui,Veronique Kiermer,Denice Mittelstaedt,Céline Bellesme,Najiba Lahlou,François Lefrère,Stéphane Blanche,Muriel Audit,Emmanuel Payen,Philippe Leboulch,Philippe Leboulch,Bruno l’Homme,Pierre Bougnères,Christof von Kalle,Alain Fischer,Marina Cavazzana-Calvo,Patrick Aubourg +26 more
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Marina Cavazzana-Calvo,Emmanuel Payen,Olivier Negre,Gary P. Wang,Kathleen M. Hehir,Floriane Fusil,Julian D. Down,Maria Denaro,Troy Brady,Karen A. Westerman,Resy Cavallesco,Beatrix Gillet-Legrand,Laure Caccavelli,Laure Caccavelli,Riccardo Sgarra,Leila Maouche-Chretien,Françoise Bernaudin,Robert Girot,Ronald Dorazio,Geert Jan Mulder,Axel Polack,Arthur Bank,Jean Soulier,Jérôme Larghero,Nabil Kabbara,Bruno Dalle,Bernard Gourmel,Gérard Socié,Stany Chrétien,Stany Chrétien,Nathalie Cartier,Patrick Aubourg,Alain Fischer,Alain Fischer,Kenneth Cornetta,Frédéric Galactéros,Yves Beuzard,Eliane Gluckman,Frederick D. Bushman,Salima Hacein-Bey-Abina,Salima Hacein-Bey-Abina,Philippe Leboulch +41 more