Abstract：
:Transcriptional targeting using a mammalian cellular promoter to restrict transgene expression to target cells is often desirable for gene therapy. This strategy is, however, hindered by relatively weak activity of some cellular promoters, which may lead to low levels of gene expression, thus declining therapeutic efficacy. Here we outline the advances accomplished in the area of transcriptional targeting to brain cells, with a particular focus on engineering gene cassettes to augment cell type-specific expression. Among the effective approaches that improve gene expression while retaining promoter specificity are promoter engineering to change authentic sequences of a cellular promoter and the combined use of a native cellular promoter and other cis-acting elements. Success in achieving high level and sustained transgene expression only in the cell types of interest would be of importance in allowing gene therapy to have its impact on patient treatment.
journal_name
Adv Drug Deliv Revjournal_title
Advanced drug delivery reviewsauthors
Boulaire J,Balani P,Wang Sdoi
10.1016/j.addr.2009.02.007subject
Has Abstractpub_date
2009-07-02 00:00:00pages
589-602issue
7-8eissn
0169-409Xissn
1872-8294pii
S0169-409X(09)00134-3journal_volume
61pub_type
杂志文章,评审abstract：:Adult neural stem and progenitor cells (NSPCs) offer a unique opportunity for neural regeneration and niche modification in physiopathological conditions, harnessing the capability to modify from neuronal circuits to glial scar. Findings exposing the vast plasticity and potential of NSPCs have accumulated over the pas...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2017.07.016
更新日期：2017-10-01 00:00:00
abstract：:Safe nasal vaccines capable of promoting both mucosal and systemic immunity are needed for effective protection against bacterial and viral pathogens. While parenteral cytokine treatment could lead to unwanted toxicity, the nasal delivery route results in low but biologically active serum cytokine levels. Interleukin ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(01)00170-3
更新日期：2001-09-23 00:00:00
abstract：:The extreme shortage of human donor organs for treatment of patients with end-stage organ failures is well known. Xenotransplantation, which might provide unlimited organ supply, is a most promising strategy to solve this problem. Domestic pigs are regarded as ideal organ-source animals owing to similarity in anatomy,...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.04.001
更新日期：2021-01-01 00:00:00
abstract：:The development of lipid-based delivery vehicles for therapeutic molecules has become a topic of intense research. Recently, much of this effort has been directed towards mimicking the characteristics of viruses that give them an advantage for the delivery of genetic medicines. One of the most desirable properties of ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(99)00032-0
更新日期：1999-08-20 00:00:00
abstract：:Among non-viral vectors, chitosan and chitosan derivatives have been developed in vitro and in vivo for DNA and siRNA delivery systems because of their cationic charge, biodegradability and biocompatibility, as well as their mucoadhesive and permeability-enhancing properties. However, the transfection efficiency of ch...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2009.08.004
更新日期：2010-01-31 00:00:00
abstract：:Hydrogels are synthetic or natural polymer networks that closely mimic native extracellular matrices. As hydrogel-based vehicles are being increasingly employed in therapeutic cell delivery, two inherent traits of most common hydrogels, namely low cell affinity and high cell constraint, have significantly drawn the at...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2010.02.001
更新日期：2010-06-15 00:00:00
abstract：:Amphotericin B colloidal dispersion (ABCD) is a near 1:1 discoidal complex of amphotericin B (AMB) and sodium cholesteryl sulfate (SCS) arranged as a bilayer of SCS interspersed with AMB via noncovalent interactions. The complex is stable in blood and plasma with minimal dissociation. In vitro and in vivo studies show...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(01)00104-1
更新日期：2001-04-25 00:00:00
abstract：:Human genome sequencing revealed that only ~1.5% of the DNA sequence coded for proteins. More and more evidence has uncovered that a substantial part of the 98.5% so-called "junk" DNAs actually code for noncoding RNAs. Two milestones, chemical drugs and protein drugs, have already appeared in the history of drug devel...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2013.11.006
更新日期：2014-02-01 00:00:00
abstract：:Cancer treatment regimens often include multiple anticancer agents targeting different cellular mechanisms in delicate balance with associated toxicity. Drug delivery systems offer a unique tool in the treatment of cancer, and applications in the local treatment of cancer have demonstrated utility in providing sustain...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2009.04.010
更新日期：2009-08-10 00:00:00
abstract：:The great interest in mucosal vaccine delivery arises from the fact that mucosal surfaces represent the major site of entry for many pathogens. Among other mucosal sites, nasal delivery is especially attractive for immunization, as the nasal epithelium is characterized by relatively high permeability, low enzymatic ac...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2008.09.005
更新日期：2009-02-27 00:00:00
abstract：:The role of metal ions in the body and particularly in the formation, regulation and maintenance of bone is only just starting to be unravelled. The role of some ions, such as zinc, is more clearly understood due to its central importance in proteins. However, a whole spectrum of other ions is known to affect bone for...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.05.015
更新日期：2013-04-01 00:00:00
abstract：:The description of irregular particle morphology and powder properties may offer unique insights into the performance of materials intended for use in dry powder inhalers. A number of mathematically similar approaches have been used to study these phenomena, all based on evaluating data in terms of irregular oscillati...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(97)00508-5
更新日期：1997-06-09 00:00:00
abstract：:The cellular membrane constitutes an effective barrier that offers protection for the complex, yet highly ordered, intracellular environment that defines the cell. Passage of molecules across this barrier is highly regulated and highly restricted, with molecular size being the most significant criteria. Over the last ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2007.03.004
更新日期：2007-03-30 00:00:00
abstract：:DNA plasmid-based gene expression systems are being widely investigated for the potential treatment of genetic and acquired disease and for DNA-based vaccination. A number of human clinical trials are in progress using plasmid-based drugs. The regulatory framework that has been applied to biologicals such as recombina...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(97)00105-1
更新日期：1998-03-02 00:00:00
abstract：:Injury to the podocytes is the initiating cause of many renal diseases, leading to proteinuria with possible progression to end-stage renal disease. Podocytes are highly specialized cells, with an important role in maintaining the glomerular filtration barrier and producing growth factors for both mesangial cells and ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2010.08.012
更新日期：2010-11-30 00:00:00
abstract：:While a vast array of liposomes, peptides, and molecular conjugates have been evaluated for nonviral gene transfer, the entity containing the actual gene itself is almost always a plasmid. The layout of most plasmid DNA (pDNA) vectors is usually quite simple, consisting of a promoter, transgene, polyadenylation signal...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2004.12.009
更新日期：2005-04-05 00:00:00
abstract：:Inflammation, a common feature of many diseases, is an essential immune response that enables survival and maintains tissue homeostasis. However, in some conditions, the inflammatory process becomes detrimental, contributing to the pathogenesis of a disease. Targeting inflammation by using nanomedicines (i.e. nanopart...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.07.010
更新日期：2020-01-01 00:00:00
abstract：:Orally administered anti-HIV drugs must be adequately and consistently absorbed for therapy to be successful. This review discusses the barriers to achieving oral bioavailability for the currently available anti-HIV drugs. Most reverse transcriptase inhibitors have good oral bioavailabilities. Didanosine bioavailabili...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(99)00022-8
更新日期：1999-10-18 00:00:00
abstract：:Silicon is one of the most abundant chemical elements found on the Earth. Due to its unique chemical and physical properties, silicon based materials and their oxides (e.g. silica) have been used in several industries such as building and construction, electronics, food industry, consumer products and biomedical engin...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.05.008
更新日期：2012-12-01 00:00:00
abstract：:In its 33 years, ADDR has published regularly on the po5tential of oral delivery of biologics especially peptides and proteins. In the intervening period, analysis of the preclinical and clinical trial failures of many purported platform technologies has led to reflection on the true status of the field and reigning i...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.05.007
更新日期：2020-01-01 00:00:00
abstract：:Nanomedicine, which is an application of nanotechnologies in healthcare is developed to improve the treatments and lives of patients suffering from a range of disorders and to increase the successes of drug candidates. Within the nanotechnology universe, the remarkable unique and tunable properties of dendrimers have ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2017.11.007
更新日期：2018-01-01 00:00:00
abstract：:miRNA-based technologies provide attractive tools for several liver tissue engineering approaches. Herein, we review the current state of miRNA applications in liver tissue engineering. Several miRNAs have been implicated in hepatic disease and proper hepatocyte function. However, the clinical translation of these fin...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2015.06.005
更新日期：2015-07-01 00:00:00
abstract：:Islet transplantation has the potential to cure type 1 diabetes. Despite recent therapeutic success, it is still not common because a large number of transplanted islets get damaged by multiple challenges including instant blood mediated inflammatory reaction, hypoxia/reperfusion injury, inflammatory cytokines, and im...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2010.11.003
更新日期：2011-01-01 00:00:00
abstract：:Several synthetic and natural materials are used in soft tissue engineering and regenerative medicine with varying degrees of success. Among them, silkworm silk protein fibroin, a naturally occurring protein-based biomaterial, exhibits many promising characteristics such as biocompatibility, controllable biodegradabil...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2019.09.003
更新日期：2020-01-01 00:00:00
abstract：:Cellular processes depend on the fidelity of intracellular membrane traffic. Lipids, proteins, receptor ligands and solute molecules are trafficked to distinct compartments within the cell through both the biosynthetic and endocytic pathways. An appreciation of these pathways is vital for a complete understanding of i...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2004.05.003
更新日期：2005-01-02 00:00:00
abstract：:Hydrogels prepared via self-assembly offer scalable and tunable platforms for drug delivery applications. Molecular-scale self-assembly leverages an interplay of attractive and repulsive forces; drugs and other active molecules can be incorporated into such materials by partitioning in hydrophobic domains, affinity-me...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2021.01.006
更新日期：2021-01-12 00:00:00
abstract：:The coiled-coil is a ubiquitous protein folding and assembly motif made of alpha-helices wrapping around each other forming a supercoil. The sequences of coiled-coils are made of seven-residue repeats, called heptads, and thus are polymer-like. Due to its simplicity and regularity, the coiled-coil is the most extensiv...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(02)00058-3
更新日期：2002-10-18 00:00:00
abstract：:The mitochondrial trifunctional protein (MTP) is a heterotrimeric protein that consists of four alpha-subunits and four beta-subunits and catalyzes three of the four chain-shortening reactions in the mitochondrial beta-oxidation of long-chain fatty acids. Families with recessively inherited MTP defects display a spect...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2008.04.014
更新日期：2008-10-01 00:00:00
abstract：:Theranostic systems support diagnostic and therapeutic functions in a single integrated entity and enable precise spatiotemporal control of the generation of therapeutic molecules according to the individual patient's disease state, thereby maximizing the therapeutic outcome and minimizing side effects. These systems ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2016.05.006
更新日期：2016-10-01 00:00:00
abstract：:Lanthanide-doped upconverting luminescent nanoparticles (UCNPs) are promising materials for optical imaging-guided drug delivery and therapy due to their unique optical and chemical properties. UCNPs absorb low energy near-infrared (NIR) light and emit high-energy shorter wavelength photons. Their special features all...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.05.007
更新日期：2013-05-01 00:00:00
