Abstract：
:Clinical trials of gene therapy for cystic fibrosis suggest that current levels of gene transfer efficiency are probably too low to result in clinical benefit, largely as a result of the barriers faced by gene transfer vectors within the airways. The respiratory epithelium has evolved a complex series of extracellular barriers (mucus, lack of receptors, immune surveillance, etc.) aimed at preventing penetration of lumenally delivered materials, including gene therapy vectors. In addition, once in the cell, further hurdles have to be overcome, including DNA degradation, nuclear import and the ability to maintain long-term transgene expression. Strategies to overcome these barriers will be addressed in this review and include the use of: (i) clinically relevant adjuncts to overcome the extra- and intracellular barriers; (ii) less-conventional delivery routes, such as intravenous or in utero administration; (iii) more efficient non-viral vectors and 'stealth' viruses which can be re-administered; and (iv) new approaches to prolong transgene expression by means of alternative promoters or integrating vectors. These advances have the potential to improve the efficiency of gene delivery to the airway epithelium, thus making gene therapy a more realistic option for cystic fibrosis.
journal_name
Adv Drug Deliv Revjournal_title
Advanced drug delivery reviewsauthors
Ferrari S,Geddes DM,Alton EWdoi
10.1016/s0169-409x(02)00145-xsubject
Has Abstractpub_date
2002-12-05 00:00:00pages
1373-93issue
11eissn
0169-409Xissn
1872-8294pii
S0169409X0200145Xjournal_volume
54pub_type
杂志文章,评审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：:Cells cultured in vitro will tend to retain their differentiated phenotype under conditions that resemble their natural in vivo environment, for example, when cultured on polymer scaffolds in tissue culture bioreactors. In this chapter, we define organized cell communities as three-dimensional in vitro grown cell-poly...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(98)00017-9
更新日期：1998-08-03 00:00:00
abstract：:Transdermal and rectal routes of drugs are very important as a useful supplement of oral routes and the direct exposing method of drugs when the systemic and local effects are required. The widespread use of parent cyclodextrins (CDs) and chemically modified CD derivatives (CD derivatives) for in-vitro and in-vivo der...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(98)00056-8
更新日期：1999-03-01 00:00:00
abstract：:Theranostics, the fusion of therapy and diagnostics for optimizing efficacy and safety of therapeutic regimes, is a growing field that is paving the way towards the goal of personalized medicine for the benefit of patients. The use of light as a remote-activation mechanism for drug delivery has received increased atte...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2010.09.002
更新日期：2010-08-30 00:00:00
abstract：:Recreating the beating heart in the laboratory continues to be a formidable bioengineering challenge. The fundamental feature of the heart is its pumping action, requiring considerable mechanical forces to compress a blood filled chamber with a defined in- and outlet. Ventricular output crucially depends on venous loa...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2015.09.001
更新日期：2016-01-15 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：:Bioprinted skin tissue has the potential for aiding drug screening, formulation development, clinical transplantation, chemical and cosmetic testing, as well as basic research. Limitations of conventional skin tissue engineering approaches have driven the development of biomimetic skin equivalent via 3D bioprinting. A...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2018.07.016
更新日期：2018-07-01 00:00:00
abstract：:We review the early developments in drug delivery from 1960 to 1990 with emphasis on the fundamental aspects of the field and how they shaped the collaboration of pharmaceutical scientists, chemists, biologists, engineers and medical scientists towards the development of advanced drug delivery systems. Emphasis is giv...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.09.040
更新日期：2013-01-01 00:00:00
abstract：:Despite recent progress in our knowledge about the development and therapy of colorectal cancer (CRC), it still remains one of the major cancer related deaths throughout the world. With the introduction of new cytotoxic and targeting agents a significant improvement in progression-free and overall survival has been ac...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2008.10.002
更新日期：2009-05-20 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：: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：:High affinity antibodies are crucial both for the discovery and validation of biomarkers for human health and disease and as clinical diagnostic and therapeutic reagents. This review describes some of the latest technologies for the design, mutation and selection of high affinity antibodies that provide a paradigm for...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2006.01.025
更新日期：2006-08-07 00:00:00
abstract：:We have entered a new era in understanding CNS circuitry involved in acute and chronic pain. The ability to objectively measure a pain or analgesic state of the brain using non-invasive methods that define neural activation provides the possibility for top-down approaches to drug discovery. These brain maps represent ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(03)00099-1
更新日期：2003-08-28 00:00:00
abstract：:Numerous drugs are known to deplete mitochondrial DNA (mtDNA) from mammalian cells. These include DNA polymerase gamma and type II topoisomerase inhibitors, lipophilic cationic compounds, and DNA intercalating and non intercalating agents. The effects of these drugs on mtDNA metabolism will be discussed and potential ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(01)00133-8
更新日期：2001-07-02 00:00:00
abstract：:Micro-/nanoscale technologies such as lithographic techniques and microfluidics offer promising avenues to revolutionalize the fields of tissue engineering, drug discovery, diagnostics and personalized medicine. Microfabrication techniques are being explored for drug delivery applications due to their ability to combi...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2011.11.013
更新日期：2012-05-01 00:00:00
abstract：:Gene delivery to bone is useful both as an experimental tool and as a potential therapeutic strategy. Among its advantages over protein delivery are the potential for directed, sustained and regulated expression of authentically processed, nascent proteins. Although no clinical trials have been initiated, there is a s...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.03.013
更新日期：2012-09-01 00:00:00
abstract：:In spite of significant exposure to drugs and chemicals through breast milk, there are very few reports of documented adverse effects on the infant. It is perhaps appropriate to consider that the presence of drugs and chemicals in milk may have a positive effect on developmental processes of the young infant. New expe...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(03)00029-2
更新日期：2003-04-29 00:00:00
abstract：:One of the most common causes of illnesses in humans is from respiratory tract infections caused by bacterial, viral or fungal pathogens. Inhaled anti-infective drugs are crucial for the prophylaxis and treatment of respiratory tract infections. The benefit of anti-infective drug delivery via inhalation is that it aff...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2014.11.004
更新日期：2015-05-01 00:00:00
abstract：:The effective delivery of antigens via the oral route is an extremely desirable goal. Mucosal delivery of antigens stimulates mucosal and systemic immunity without affecting maternal antibodies and reduces the need for sterile needles or trained personnel. To date, there are very few commercially available oral vaccin...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2004.09.009
更新日期：2005-01-10 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：:Many drug molecules possess inadequate physical-chemical characteristics that prevent to surpass the viscous mucus layer present in the surface of mucosal tissues. Due to mucus protective role and its fast turnover, these drug molecules end up being removed from the body before being absorbed and, thus, before exertin...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2017.09.020
更新日期：2018-01-15 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 wide range of polymeric scaffolds have been intensively studied for use as implantable and temporal devices in tissue engineering. Biodegradable and biocompatible scaffolds having a highly open porous structure and good mechanical strength are needed to provide an optimal microenvironment for cell proliferation, mig...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2007.03.015
更新日期：2007-05-30 00:00:00
abstract：:Antiepileptic drugs are commonly given orally for chronic treatment of epilepsy. The treatment of epilepsy requires administration of medications for both acute and chronic treatment using multiple types of formulations. Parenteral routes are used when the oral route is unavailable or a rapid clinical response is requ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/j.addr.2012.01.017
更新日期：2012-07-01 00:00:00
abstract：:PEGylation is a clinically proven strategy for increasing the therapeutic efficacy of protein-based medicines. Our approach to site-specific PEGylation exploits the thiol selective chemistry of the two cysteine sulfur atoms from an accessible disulfide. It involves two key steps: (1) disulfide reduction to release the...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2007.06.014
更新日期：2008-01-03 00:00:00
abstract：:Over recent years the research focus within the field of respiratory drug delivery has broadened to include a wide range of potential applications for inhalation by delivering drugs not just onto the lung mucosa but across it. The range of drugs being assessed is broad and includes both current and novel therapies and...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2007.08.023
更新日期：2007-09-30 00:00:00
abstract：:An essential element of tissue homeostasis is the response to injuries, cutaneous wound healing being the most studied example. In the adults, wound healing aims at quickly restoring the barrier function of the skin, leading however to scar, a dysfunctional fibrotic tissue. On the other hand, in fetuses a scarless tis...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2018.04.011
更新日期：2019-06-01 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
abstract：:Lack of approved pharmaceutical agents and/or pharmacokinetic data in the literature for exotic, wildlife, and zoo species is a major issue for veterinarians. These practitioners must take approved agents (veterinary or human) and extrapolate their use to non-approved species with little or no scientific basis to supp...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(02)00068-6
更新日期：2002-10-04 00:00:00
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
