Abstract:
INTRODUCTION:Viral and non-viral vectors have been used as methods of delivery in gene therapy for many CNS diseases. Currently, viral vectors such as adeno-associated viruses (AAV), retroviruses, lentiviruses, adenoviruses and herpes simplex viruses (HHV) are being used as successful vectors in gene therapy at clinical trial levels. However, many disadvantages have risen from their usage. Non-viral vectors like cationic polymers, cationic lipids, engineered polymers, nanoparticles, and naked DNA offer a much safer option and can therefore be explored for therapeutic purposes. AREAS COVERED:This review discusses different types of viral and non-viral vectors for gene therapy and explores clinical trials for CNS diseases that have used these types of vectors for gene delivery. Highlights include non-viral gene delivery and its challenges, possible strategies to improve transfection, regulatory issues concerning vector usage, and future prospects for clinical applications. EXPERT OPINION:Transfection efficiency of cationic lipids and polymers can be improved through manipulation of molecules used. Efficacy of cationic lipids is dependent on cationic charge, saturation levels, and stability of linkers. Factors determining efficacy of cationic polymers are total charge density, molecular weights, and complexity of molecule. All of the above mentioned parameters must be taken care for efficient gene delivery.
journal_name
Expert Opin Drug Delivjournal_title
Expert opinion on drug deliveryauthors
Jayant RD,Sosa D,Kaushik A,Atluri V,Vashist A,Tomitaka A,Nair Mdoi
10.1080/17425247.2016.1188802subject
Has Abstractpub_date
2016-10-01 00:00:00pages
1433-45issue
10eissn
1742-5247issn
1744-7593journal_volume
13pub_type
杂志文章,评审abstract:IMPORTANCE OF THE FIELD:Vascular delivery of several classes of therapeutic agents may benefit from carriage by red blood cells (RBC), for example, drugs that require delivery into phagocytic cells and those that must act within the vascular lumen. The fact that several protocols of infusion of RBC-encapsulated drugs a...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425241003610633
更新日期:2010-04-01 00:00:00
abstract::Background: To overcome the rigorous aseptic process widely adopted by commercial long-acting injections, this work aimed to prepare a sterile MPA injection by manipulating the crystal habit through a bottom-up method. Methods: The habit of the precipitated crystals was modified by changing the process conditions. Whe...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章
doi:10.1080/17425247.2019.1662394
更新日期:2019-10-01 00:00:00
abstract::Introduction: Thymoquinone (TQ), 2-isopropyl-5-methylbenzo-1, 4-quinone, the main active constituent of Nigella sativa (NS) plant, has been proven to be of great therapeutic aid in various in vitro and in vivo conditions. Despite the promising therapeutic activities of TQ, this molecule is not yet in the clinical tria...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1080/17425247.2020.1730808
更新日期:2020-04-01 00:00:00
abstract:INTRODUCTION:The utility of controlled and sustained release of drugs from polymeric systems, both bulk (hydrogels) and colloids (nanoparticles), is a key point that should be addressed. Unfortunately, classic delivery systems are essentially driven by diffusion, which is very quick due to the high concentration gradie...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1080/17425247.2017.1285280
更新日期:2017-11-01 00:00:00
abstract:AIM:Spacer devices are inhalation aids of varying dimension and complexity, specifically designed to overcome problems with the use of pressurised metered dose inhalers (pMDIs). The aim of this review is to examine the current understanding about these inhalation devices and discuss their advantages and disadvantages. ...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425240802637862
更新日期:2009-01-01 00:00:00
abstract::Various techniques have been developed to prepare nanoparticles for the delivery of drugs. The implementation of these techniques for large-scale production is still in its infancy, as it requires defined steps which include excipient compatibility, process feasibility, formulation optimization, process optimization, ...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425240902735806
更新日期:2009-02-01 00:00:00
abstract:INTRODUCTION:Topical drug delivery is a challenging area with many advantages such as avoidance of first passage effect, stabilization of blood concentrations and attainment of local therapeutic effect with fewer side effects. Despite all these advantages, topical drug delivery remains limited to few molecules, since s...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1080/17425247.2016.1182489
更新日期:2016-08-01 00:00:00
abstract:INTRODUCTION:Development of efficient in vivo delivery systems remains a major challenge en route to clinical application of antisense technology, including RNA-cleaving molecules such as deoxyribozymes (DNAzymes). The mechanisms of oligonucleotide uptake and trafficking are clearly dependent on cell type and the type ...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1080/17425247.2017.1266326
更新日期:2017-09-01 00:00:00
abstract:BACKGROUND AND OBJECTIVES:Alendronate sodium (ALS) is the most common drug used for the treatment of osteoporosis. The challenges facing ALS use include: very poor oral bioavailability (0.6%), esophageal ulcers, and complicated instructions for its use. The objective of this research is to utilize nanotechnology to for...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章
doi:10.1517/17425247.2013.799136
更新日期:2013-06-01 00:00:00
abstract:OBJECTIVES:Methotrexate (MTX) is a chemotherapeutic and anti-inflammatory drug that may cause systemic adverse effects. This study investigated kinetics and biodistribution of MTX delivered topically by ablative fractional laser (AFXL). METHODS:In vitro passive diffusion of 10 mg/ml MTX (1 w/v%) was measured from 0.25...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章
doi:10.1517/17425247.2015.1031216
更新日期:2015-07-01 00:00:00
abstract:INTRODUCTION:Nasal drug delivery that exploits the olfactory and trigeminal neuronal pathways to deliver drugs to the brain is being widely explored by pharmaceutical companies, for the delivery of challenging drugs. Low-molecular-weight and lipophilic drugs are effectively absorbed by the intranasal route for efficaci...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2012.636801
更新日期:2012-01-01 00:00:00
abstract:INTRODUCTION:Lymphatic vessels are the preferential route of most solid tumors to spread their metastases in the body. The onset of metastatic nests in draining lymph nodes (LNs) are a significant indicator of cancer progression and a dismaying sign of worsen staging. Therefore, the individuation and elimination of can...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1080/17425247.2018.1446937
更新日期:2018-05-01 00:00:00
abstract:IMPORTANCE OF THE FIELD:Cancer is the most formidable human disease. Owing to the heterogeneity of cancer, a single-treatment modality is insufficient for the complete elimination of cancer cells. Therapeutic strategies from various aspects are needed for cancer therapy. These therapeutic agents should be carefully sel...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2010.510511
更新日期:2010-09-01 00:00:00
abstract:BACKGROUND:Pulmonary cell culture models for the development of new aerosol medicines are attracting increasing interest. Ease of handling, ethical acceptance and high explanatory power are the main advantages of cell culture systems in pulmonary drug research. OBJECTIVE:Pulmonary cell culture models are described and...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.5.6.641
更新日期:2008-06-01 00:00:00
abstract:BACKGROUND:Targeted delivery of antithrombotic (thrombolytic) drugs is expected to increase their efficacy and decrease side effects, especially in the case of thrombolytic enzymes. Liposomes, phospholipid nanosized bubbles with a bilayered membrane structure, have drawn a lot of interest as pharmaceutical carriers for...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425240802497457
更新日期:2008-11-01 00:00:00
abstract:INTRODUCTION:Hepatocellular carcinoma (HCC) is one of the most common lethal malignancies. The prognosis is poor despite progress in early diagnosis. The initial treatment of choice is hepatic resection; unfortunately, not all patients are eligible for liver resection. Moreover, there are significant recurrences. Diffe...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2013.770733
更新日期:2013-05-01 00:00:00
abstract:IMPORTANCE OF THE FIELD:Active targeting of bioactive molecules by physicochemical association with hyaluronic acid (HA) is an attractive approach in current nanomedicine because HA is biocompatible, non-toxic and non-inflammatory. AREAS COVERED IN THIS REVIEW:This review focuses on synthesis, physicochemical characte...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425241003730399
更新日期:2010-06-01 00:00:00
abstract:INTRODUCTION:Since decades, cancer is a major public health problem worldwide. The increasing knowledge of molecular and tumor biology has significantly changed the cancer treatment paradigms during the past few years. AREA COVERED:Conventionally, the first-line treatment of solid tumors is their surgical removal foll...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1080/17425247.2020.1757645
更新日期:2020-06-01 00:00:00
abstract::Cationic lipid/DNA complexes (lipoplexes) represent an attractive alternative to viral vectors for cell transfection in vitro and in vivo but still suffer from relatively low efficiency. Comprehension of the interactions between vectors and DNA as well as cellular pathways and mechanisms in DNA entry into cells and ul...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425240903287153
更新日期:2009-12-01 00:00:00
abstract:INTRODUCTION:Recent advances in pharmacological therapies to treat ocular diseases such as glaucoma, age-related macular degeneration, diabetic macular edema and retinal vascular occlusions have greatly improved the prognosis for these diseases. Due to these advances in pharmacological therapy, there is a great deal of...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2014.935338
更新日期:2014-10-01 00:00:00
abstract:INTRODUCTION:Ultrasound enhancement of thrombolysis (sonothrombolysis) is further potentiated by administration of acoustically active microbubbles, which may be developed into powerful adjuvant therapies for thrombolytic treatment of occlusive conditions such as ischaemic stroke. AREAS COVERED:The role of microbubble...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2014.868434
更新日期:2014-02-01 00:00:00
abstract::The treatment of genetic diseases using therapeutic gene transfer is considered to be a significant development. This development has brought with it certain limitations, and the process of overcoming these barriers has seen a drastic change in gene delivery. Many metal ions such as Mg2+, Mn2+, Ba2+ and, most importan...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章
doi:10.1517/17425247.3.2.235
更新日期:2006-03-01 00:00:00
abstract::FlexTouch® (FT) is a new prefilled insulin pen with the unique characteristic of no extendable push button at any dose setting and consequently a low activation force. Its technical features along with health care professionals' and patients' preferences in comparison to other traditionally used devices for insulin de...
journal_title:Expert opinion on drug delivery
pub_type: 社论
doi:10.1517/17425247.2014.894505
更新日期:2014-05-01 00:00:00
abstract:IMPORTANCE OF THE FIELD:Spontaneously constructed from block copolymers in aqueous media, the polymer micelle has been extensively studied as a potential carrier of poorly water-soluble drugs, but cellular uptake pathways and stability of micelles in blood have not yet been clearly understood. An in-depth insight into ...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425240903380446
更新日期:2010-01-01 00:00:00
abstract:INTRODUCTION:This topic is important as it allows for improved specificity in drug delivery, providing possibilities for reduced side effects, and thereby improved pharmacotherapy. As a wealth of different polymers and mechanisms of action has been suggested, a systematic overview of the field is of current importance....
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2013.846906
更新日期:2013-12-01 00:00:00
abstract:INTRODUCTION:Pressurized metered dose inhalers (pMDIs) are evolving to be a very effective drug delivery option in patients with airway diseases. They offer comparable lung deposition and reduced oropharyngeal deposition similar with the dry powder inhalers. As recommended by the Global Initiative for Asthma guidelines...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2015.987661
更新日期:2015-06-01 00:00:00
abstract::Introduction: Pulmonary drug delivery is a complex field of research combining physics which drive aerosol transport and deposition and biology which underpins efficacy and toxicity of inhaled drugs. A myriad of preclinical methods, ranging from in-silico to in-vitro, ex-vivo and in-vivo, can be implemented.Areas cove...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1080/17425247.2020.1730807
更新日期:2020-04-01 00:00:00
abstract:INTRODUCTION:Macrophages are involved in the normal defense of the body; however, the varying phenotypes of macrophages and imbalance in their ratio lead to the impairment of immune response initiating the production of inflammation. As the role of macrophages in immunological disorders and their surface receptors modu...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1080/17425247.2020.1783237
更新日期:2020-09-01 00:00:00
abstract::Due to the poor solvent properties of hydrofluoroalkanes, suspension is often the only formulation option for respiratory drug delivery. Research in this area has focussed mainly on two main themes over the past 5 years: new design of stabilisers and particle engineering. Among the most important advances, the introdu...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2.4.625
更新日期:2005-07-01 00:00:00
abstract::dl-Methylphenidate (MPH) remains the most widely used pharmacological agent in the treatment of attention-deficit/hyperactivity disorder (ADHD). The predominantly dopaminergic mechanism of the psychostimulant actions has become more clearly defined. Neuroimaging and genetic studies are revealing the underlying neuropa...
journal_title:Expert opinion on drug delivery
pub_type: 杂志文章,评审
doi:10.1517/17425247.2.1.121
更新日期:2005-01-01 00:00:00