Optimization and Functional Effects of Stable Short Hairpin RNA Expression in Primary Human Lymphocytes via Lentiviral Vectors
Dong Sung An,F. Xiao-Feng Qin,Vincent C. Auyeung,Si Hua Mao,Sam K. P. Kung,David Baltimore,Irvin S. Y. Chen +6 more
TLDR
The results suggest that functional shRNA screens should include tests for both potency and adverse metabolic effects upon primary cells, and lentiviral vectors bearing the H1 promoter are more suitable for stable transduction and expression of shRNA in primary human T lymphocytes.About:
This article is published in Molecular Therapy.The article was published on 2006-10-01 and is currently open access. It has received 157 citations till now. The article focuses on the topics: Small hairpin RNA & RNA interference.read more
Citations
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Strategies for silencing human disease using RNA interference.
Daniel H. Kim,John J. Rossi +1 more
TL;DR: This work has highlighted both promise and challenges in using RNAi for therapeutic applications and suggests design and delivery strategies for RNAi effector molecules must be carefully considered to address safety concerns and to ensure effective, successful treatment of human diseases.
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Breaking down the barriers: siRNA delivery and endosome escape
TL;DR: This Commentary focuses on novel strategies designed to enhance endosomal escape and thereby increase the efficacy of siRNA-mediated gene silencing.
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RNA-Based Gene Therapy for HIV with Lentiviral Vector–Modified CD34+ Cells in Patients Undergoing Transplantation for AIDS-Related Lymphoma
David DiGiusto,Amrita Krishnan,Lijing Li,Haitang Li,Shirley Li,Anitha Rao,Shu Mi,Priscilla Yam,Sherri Stinson,Michael Kalos,Joseph C. Alvarnas,Simon F. Lacey,Jiing Kuan Yee,Mingjie Li,Larry A. Couture,David Hsu,Stephen J. Forman,John J. Rossi,John A. Zaia +18 more
TL;DR: A step toward workable gene therapy is reported in the form of stable expression of a lentiviral vector encoding anti-HIV RNAs in blood stem cells transplanted into AIDS patients, and cells that survived for long periods of time in patients, although too scarce to cure or even improve their HIV infections.
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Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo
TL;DR: It is found that shRNAs cause Purkinje cell neurotoxicity, and the safety of these vectors in mouse cerebella is compared, and these findings suggest that miRNA-based platforms are better suited for therapeutic silencing in the mammalian brain.
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Genetic therapies against HIV
TL;DR: Gene therapy offers the promise of preventing progressive HIV infection by sustained interference with viral replication in the absence of chronic chemotherapy, andGene-targeting strategies are being developed with RNA-based agents, and protein-basedagents, such as the mutant HIV Rev protein M10, fusion inhibitors and zinc-finger nucleases.
References
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MicroRNAs: Genomics, Biogenesis, Mechanism, and Function
TL;DR: Although they escaped notice until relatively recently, miRNAs comprise one of the more abundant classes of gene regulatory molecules in multicellular organisms and likely influence the output of many protein-coding genes.
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Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
TL;DR: 21-nucleotide siRNA duplexes provide a new tool for studying gene function in mammalian cells and may eventually be used as gene-specific therapeutics.
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The functions of animal microRNAs
TL;DR: Evidence is mounting that animal miRNAs are more numerous, and their regulatory impact more pervasive, than was previously suspected.
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Role for a bidentate ribonuclease in the initiation step of RNA interference
TL;DR: Dicer is a member of the RNase III family of nucleases that specifically cleave double-stranded RNAs, and is evolutionarily conserved in worms, flies, plants, fungi and mammals, and has a distinctive structure, which includes a helicase domain and dualRNase III motifs.
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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.