Abstract:
:The future of non-viral gene therapy depends on a detailed understanding of the barriers to delivery of polynucleotides. These include physicomechanical barriers, which limit the design of delivery devices, physicochemical barriers that influence self-assembly of colloidal particulate formulations, and biological barriers that compromise delivery of the DNA to its target site. It is important that realistic delivery strategies are adopted for early clinical trials in non-viral gene therapy. In the longer term, it should be possible to improve the efficiency of gene delivery by learning from the attributes which viruses have evolved; attributes that enable translocation of viral components across biological membranes. Assembly of stable, organized virus-like particles will require a higher level of control than current practice. Here, we summarize present knowledge of the biodistribution and cellular interactions of gene delivery systems and consider how improvements in gene delivery will be accomplished in the future.
journal_name
Adv Drug Deliv Revjournal_title
Advanced drug delivery reviewsauthors
Pouton CW,Seymour LWdoi
10.1016/s0169-409x(00)00133-2subject
Has Abstractpub_date
2001-03-01 00:00:00pages
187-203issue
1-3eissn
0169-409Xissn
1872-8294pii
S0169-409X(00)00133-2journal_volume
46pub_type
杂志文章,评审abstract::In recent years, nanotechnology has offered attractive opportunities to overcome the (bio)pharmaceutical drawbacks of most drugs such as low aqueous solubility and bioavailability. Among the numerous methodologies that have been applied to improve drug performance, a special emphasis has been made on those that increa...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2018.07.012
更新日期:2018-06-01 00:00:00
abstract::Nuclear import of macromolecules or particles is a strictly regulated phenomenon that is now understood in some depth at the molecular level. Transport across nuclear membranes is controlled by nuclear pores, with the involvement of cytoplasmic receptors and accessory molecules. Each nuclear pore complex is an assembl...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章
doi:10.1016/s0169-409x(98)00050-7
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abstract::Antibodies can have exquisite specificity of target recognition and thus generate highly selective outcomes following their systemic administration. While antibodies can have high specificity, the doses required to treat patients, particularly for a chronic condition, are typically large. Fortunately, advances in prod...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2006.03.011
更新日期:2006-08-07 00:00:00
abstract::Molecular imaging is an emerging technology that allows the visualization of interactions between molecular probes and biological targets. Molecules that either direct or are subject to homeostatic controls in biological systems could be labeled with the appropriate radioisotopes for the quantitative measurement of se...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2010.09.007
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abstract::Oral lipid-based drug delivery systems may include a broad range of oils, surfactants, and cosolvents. This diversity makes comparison of lipid-based formulations difficult. Although the relationship between formulation and drug absorption is understood at a conceptual level, performance in vivo cannot be predicted wi...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2007.10.010
更新日期:2008-03-17 00:00:00
abstract::To design an effective particulate drug delivery system having mucoadhesive function, several mucoadhesion tests for polymers and the resultant particulate systems were developed. Mucin particle method is a simple mucoadhesion test for polymers, in which the commercial mucin particles are used. By measuring the change...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2005.07.008
更新日期:2005-11-03 00:00:00
abstract::Low back pain is generally attributed to intervertebral disc (IVD) degeneration. This is a multifactorial disease induced by genetic and environmental factors and that progresses with aging. Disc degeneration is characterized by a limited ability of IVD cells to produce functional matrix while producing abnormal amoun...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2014.08.008
更新日期:2015-04-01 00:00:00
abstract::Small interfering RNA (siRNA) has a huge potential for the treatment or prevention of various lung diseases. Once the RNA molecules have successfully entered the target cells, they could inhibit the expression of specific gene sequence through RNA interference (RNAi) mechanism and generate therapeutic effects. The big...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2011.02.006
更新日期:2012-01-01 00:00:00
abstract::Pulmonary arterial hypertension (PAH) is a rare, but severe and life-threatening disease characterized by vasoconstriction and remodeling of the pulmonary arterioles, leading to progressive increase in pulmonary vascular resistance and ultimately to right-heart failure. In the last two decades, significant progress in...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2018.06.003
更新日期:2018-08-01 00:00:00
abstract::Clinical pharmacogenomics, integrating genomic information with clinical practices to facilitate the prediction of drug response, has recently emerged as a vital area of public health. In clinical trials, phenotypic data on drug response are often longitudinal, with some patients dropping out early due to physiologica...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章
doi:10.1016/j.addr.2013.03.003
更新日期:2013-06-30 00:00:00
abstract::Endogenous regenerative technology (Endoret) involves the use of patient's own biologically active proteins, growth factors and biomaterial scaffolds for therapeutic purposes. This technology provides a new approach for the stimulation and acceleration of tissue healing and bone regeneration. The versatility and bioco...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2010.01.001
更新日期:2010-06-15 00:00:00
abstract::The administration of radiolabeled compounds with gamma-emitting radionuclides allows real time pursuit of in vivo behavior of the compounds by external counting of the radioactivity. If the compounds possess a specific localization mechanism to target tissue, the image reflects the specific function of the target. Th...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章
doi:10.1016/s0169-409x(98)00102-1
更新日期:1999-04-05 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::A "microdose clinical trial" (microdosing) is one kind of early phase exploratory clinical trial, administering the compound at doses estimated to have no pharmacological or toxicological effects, aimed at screening candidates for further clinical development. This article's objective is to clarify the ethical, legal,...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2011.01.002
更新日期:2011-06-19 00:00:00
abstract::Calcium phosphate cements are used as synthetic bone grafts, with several advantages, such as their osteoconductivity and injectability. Moreover, their low-temperature setting reaction and intrinsic porosity allow for the incorporation of drugs and active principles in the material. It is the aim of the present work ...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2012.01.008
更新日期:2012-09-01 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::With inhalational delivery the alveolar epithelium appears to be the appropriate lung surface to target for the systemic delivery of macromolecules, such as therapeutic proteins. The existence of a high numerical density of smooth-coated or non-coated plasma membrane vesicles or invaginations within the alveolar epith...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/s0169-409x(01)00142-9
更新日期:2001-07-28 00:00:00
abstract::Modern pharmaceutical delivery systems are intended to produce plasma drug concentration versus time profiles that result in optimum therapeutic efficacy and a minimum of drug concentration-dependent adverse effects. To accomplish this requires that the drug delivery rate and temporal profile be based on the pharmacok...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章
doi:10.1016/s0169-409x(98)00028-3
更新日期:1998-09-07 00:00:00
abstract::Heparin and heparan sulfates are closely related linear anionic polysaccharides, called glycosaminoglycans, which exhibit a number of important biological and pharmacological activities. These polysaccharides, having complex structures and polydispersity, are biosynthesized in the Golgi of animal cells. While heparan ...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2015.11.002
更新日期:2016-02-01 00:00:00
abstract::Ultrasound (US) imaging is an exquisite tool for the non-invasive and real-time diagnosis of many different diseases. In this context, US contrast agents can improve lesion delineation, characterization and therapy response evaluation. US contrast agents are usually micrometer-sized gas bubbles, stabilized with soft o...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2013.11.013
更新日期:2014-06-01 00:00:00
abstract::A site-directed method of joining proteins to poly(ethylene glycol) is presented which allows for the preparation of essentially homogeneous PEG-protein derivatives with a single PEG chain conjugated to the amine terminus of the protein. This selectivity is achieved by conducting the reductive alkylation of proteins w...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/s0169-409x(02)00023-6
更新日期:2002-06-17 00:00:00
abstract::Newborn babies can require significant amounts of medication containing excipients intended to improve the drug formulation. Most medicines given to neonates have been developed for adults or older children and contain excipients thought to be safe in these age groups. Many excipients have been used widely in neonates...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章
doi:10.1016/j.addr.2013.11.003
更新日期:2014-06-01 00:00:00
abstract::According to the latest report from the World Health Organization, an estimated 265,000 deaths still occur every year as a direct result of burn injuries. A widespread range of these deaths induced by burn wound happens in low- and middle-income countries, where survivors face a lifetime of morbidity. Most of the deat...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2017.08.001
更新日期:2018-01-01 00:00:00
abstract::Computational Fluid Dynamics (CFD) and Discrete Element Modelling (DEM) studies relevant to inhaled drug delivery are reviewed. CFD is widely used in device design to determine airflow patterns and turbulence levels. CFD is also used to simulate particles and droplets, which are subjected to various forces, turbulence...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2011.10.004
更新日期:2012-03-30 00:00:00
abstract::Despite advances in medicine and biomedical sciences, cancer still remains a major health issue. Complex interactions between tumors and their microenvironment contribute to tumor initiation and progression and also contribute to the development of drug resistant tumor cell populations. The complexity and heterogeneit...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2014.07.002
更新日期:2014-12-15 00:00:00
abstract::In recent years, drug development costs have soared, primarily due to the failure of preclinical animal and cell culture models, which do not directly translate to human physiology. Organ-on-a-chip (OOC) is a burgeoning technology with the potential to revolutionize disease modeling, drug discovery, and toxicology res...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2018.06.001
更新日期:2019-02-01 00:00:00
abstract::Tip-enhanced Raman scattering (TERS) has become a powerful tool for nanoscale structural analysis for several branches of organic, inorganic, and biological chemistry. This highly sensitive technique enables molecular characterization with a lateral resolution far beyond Abbe's diffraction limit and correlates structu...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2015.06.007
更新日期:2015-07-15 00:00:00
abstract::Using oligonucleotide-based drugs to modulate gene expression has opened a new avenue for drug discovery. In particular small interfering RNAs (siRNAs) are being rapidly recognized as promising therapeutic tools, but their poor bioavailability limits the full realization of their clinical potential. In recent years, c...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2012.08.008
更新日期:2013-03-01 00:00:00
abstract::Quantitative structure-activity relationships have long been considered a vital component of drug discovery and development, providing insight into the role of molecular properties in the biological activity of similar and unrelated compounds. Recognition that in vitro bioassay and/or pre-clinical activity are insuffi...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2006.08.002
更新日期:2006-11-30 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