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 spectrum of maternal and fetal phenotypes. Current management of patients with MTP defects include long-term dietary therapy of fasting avoidance, low-fat/high-carbohydrate diet with restriction of long-chain fatty acid intake and substitution with medium-chain fatty acids. These dietary approaches appear promising in the short-term, but the long-term outcome of patients treated with dietary intervention is largely unknown. Potential therapeutic approaches targeted at correcting the metabolic defect will be discussed. We will discuss the potential use of protein transduction domains that cross the mitochondrial membranes for the treatment of mitochondrial disorders. In addition, we discuss the phenotypes of MTP in a heterozygous state and potential ways to intervene to increase hepatic fatty acid oxidative capacity.
journal_name
Adv Drug Deliv Revjournal_title
Advanced drug delivery reviewsauthors
Rector RS,Payne RM,Ibdah JAdoi
10.1016/j.addr.2008.04.014subject
Has Abstractpub_date
2008-10-01 00:00:00pages
1488-96issue
13-14eissn
0169-409Xissn
1872-8294pii
S0169-409X(08)00162-2journal_volume
60pub_type
杂志文章,评审abstract::In the drug development process, it remains a difficult task to regulate the entry of the drugs. However, recent progress in studies of the transporter-mediated influx and efflux of endogenous and exogenous compounds, including synthetic drugs, across the blood-brain barrier (BBB) is beginning to provide a rational ba...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章
doi:10.1016/s0169-409x(98)00084-2
更新日期:1999-04-05 00:00:00
abstract::Poly(ortho esters), POE, are synthetic bioerodible polymers that can be prepared as solid materials, or as viscous, injectable polymers. These materials have evolved through a number of families, and the latest member of this family, POE IV, is particularly well suited to drug delivery since latent acid is integrated ...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/s0169-409x(02)00056-x
更新日期:2002-10-16 00:00:00
abstract::Messenger RNA and small interfering RNA are attractive modalities for curing diseases by complementation or knock-down of proteins. For success of these RNAs, a drug delivery system (DDS) is required to control a pharmacokinetics, to enhance cellular uptake, to overcome biological membranes, and to release the cargo i...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2020.07.001
更新日期:2020-01-01 00:00:00
abstract::Synthetic polymer based Polyfectins (cationic polymer-DNA complex) have received intensive scientific research as they can potentially circumvent problems associated with viral vectors for gene therapy. These cationic macromolecules can readily condense DNA or RNA into stable nanostructures for use in gene delivery. R...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2006.09.008
更新日期:2006-12-01 00:00:00
abstract::Synthetic polymers and nanomaterials display selective phenotypic effects in cells and in the body signal transduction mechanisms involved in inflammation, differentiation, proliferation, and apoptosis. When physically mixed or covalently conjugated with cytotoxic agents, bacterial DNA or antigens, polymers can drasti...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2006.09.019
更新日期:2006-12-30 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
abstract::Many kinds of inorganic nanoparticles (NPs) including semiconductor, metal, metal oxide, and lanthanide-doped NPs have been developed for imaging and therapy applications. Their unique optical, magnetic, and electronic properties can be tailored by controlling the composition, size, shape, and structure. Interaction o...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2012.08.015
更新日期:2013-05-01 00:00:00
abstract::Spray drying is a well-established manufacturing technique which can be used to formulate amorphous solid dispersions (ASDs) which is an effective strategy to deliver poorly water soluble drugs (PWSDs). However, the inherently complex nature of the spray drying process coupled with specific characteristics of ASDs mak...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2015.12.010
更新日期:2016-05-01 00:00:00
abstract::Mitochondria represent both the main source and target of reactive oxygen and nitrogen species (RONS). In view of the large energy expenditure made by neurons during neurotransmission, an intact mitochondrial function is of paramount importance for the correct function of the brain. Accordingly, the search of therapeu...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2009.05.009
更新日期:2009-11-30 00:00:00
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 barr...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/s0169-409x(00)00133-2
更新日期:2001-03-01 00:00:00
abstract::Non-communicable diseases including cancer, cardiovascular disease, diabetes, and neuropathy are chronic in nature. Treatment of these diseases with traditional delivery systems is limited due to lack of site-specificity, non-spatiotemporal release and insufficient doses. Numerous responsive delivery systems which res...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2018.02.008
更新日期:2018-04-01 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::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::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::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::Nano- and microfabrication techniques have been widely explored in the textile, polymer and biomedical arenas, although more recently these systems have attracted considerable interest as drug delivery vehicles with concomitant considerations of physical characterization, scalability, stability and drug release. In th...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2016.01.003
更新日期:2016-05-01 00:00:00
abstract::The complexity of nanoscale interactions between biomaterials and cells has limited the realization of the ultimate vision of nanotechnology in diagnostics and therapeutics. As such, significant effort has been devoted to advancing our understanding of the biophysical interactions of the myriad nanoparticles. Endocyto...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2019.08.004
更新日期:2019-04-01 00:00:00
abstract::This article reviews the latest progresses regarding the applications of carbon nanotubes (CNTs), including single-walled carbon nanotubes (SWNTs) and multi-walled carbon nanotubes (MWNTs), as multifunctional nano-probes for biomedical imaging. Utilizing the intrinsic band-gap fluorescence of semi-conducting single-wa...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2013.10.002
更新日期:2013-12-01 00:00:00
abstract::A great deal of interest has surrounded the activities of monoclonal antibodies (mAbs), and mAb-drug, toxin and radionuclide conjugates for the treatment of human cancers. In the last few years, a number of new mAb-based reagents have been clinically approved (Rituxan, Herceptin, and Panorex), and several others are n...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/s0169-409x(01)00206-x
更新日期:2001-12-31 00:00:00
abstract::Over the past few decades, a range of vesicle-based drug delivery systems have entered clinical practice and several others are in various stages of clinical translation. While most of these vesicle constructs are lipid-based (liposomes), or polymer-based (polymersomes), recently new classes of vesicles have emerged t...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2018.10.012
更新日期:2019-01-01 00:00:00
abstract::Hydrogels have been used primarily in the pharmaceutical field as carriers for delivery of various drugs, peptides and proteins. These systems have included stimuli-responsive gels that exhibit reversible swelling behavior and hence can show modulated release in response to external stimuli such as pH, temperature, io...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/s0169-409x(01)00246-0
更新日期:2002-01-17 00:00:00
abstract::The early impact of nanotechnology on medicine is beginning to get realized, with novel nanoscale therapeutic and diagnostic modalities under development or in clinical practice today. In this commentary the field of "nanomedicine" is briefly reviewed form the perspective of where we were; where we are today; and wher...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2006.09.011
更新日期:2006-12-01 00:00:00
abstract::Mesenchymal stem cells (MSCs) have been developed for a number of indications due to their regenerative and anti-inflammatory phenotypes and their utility is enhanced by the fact that allogeneic transplant is feasible with this cell type. Animal studies and early human cases indicate that this has the potential to be ...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2017.10.003
更新日期:2018-01-01 00:00:00
abstract::Two-photon excitation imaging is the least invasive optical approach to study living tissues. We have established two-photon extracellular polar-tracer (TEP) imaging with which it is possible to visualize and quantify all exocytic events in the plane of focus within secretory tissues. This technology also enables esti...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章
doi:10.1016/j.addr.2006.07.008
更新日期:2006-09-15 00:00:00
abstract::Nanomaterials composed of plant viral components are finding their way into medical technology and health care, as they offer singular properties. Precisely shaped, tailored virus nanoparticles (VNPs) with multivalent protein surfaces are efficiently loaded with functional compounds such as contrast agents and drugs, ...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2018.08.011
更新日期:2019-05-01 00:00:00
abstract::The percentage of new chemical entities synthesised with low aqueous solubility and high therapeutic efficacy is growing, this presents major challenges for the drug delivery scientists. The role of physicochemical properties in identification of suitable drug candidates for oral lipid-based delivery systems is discus...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章
doi:10.1016/j.addr.2007.10.012
更新日期:2008-03-17 00:00:00
abstract::This paper reviews key issues found to affect acceptability and preferences for vaginal products to prevent HIV infection or HIV and pregnancy. We focus on the interplay between the biological and physico-chemical aspects of formulation and the social and behavioral issues that may affect use. The need for an HIV prev...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2015.02.004
更新日期:2015-09-15 00:00:00
abstract::Nucleic acids have gained a lot of interest for the treatment of ocular diseases. The first to enter in clinic has been Vitravene an antisense oligonucleotide for the treatment of cytomegalovirus (CMV) infection and more recently, research on aptamers have led to the marketing of anti-vascular endothelial growth facto...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2006.07.020
更新日期:2006-11-15 00:00:00
abstract::Mid-IR and Raman spectroscopy are versatile tools in pharmaceutics and biopharmaceutics, with a wide field of applications ranging from characterization of drug formulations to elucidation of kinetic processes in drug delivery. After an introduction to the basic principles of IR and Raman spectroscopy, new development...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2005.01.022
更新日期:2005-06-15 00:00:00
abstract::Amorphisation of poorly water-soluble drugs is one approach that can be applied to improve their solubility and thus their bioavailability. Amorphisation is a process that usually requires deliberate external energy input. However, amorphisation can happen both unintentionally, as in process-induced amorphisation duri...
journal_title:Advanced drug delivery reviews
pub_type: 杂志文章,评审
doi:10.1016/j.addr.2015.12.014
更新日期:2016-05-01 00:00:00