Cyclodextrins as potential gene therapy vectors

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Clinical Studies ◽  
Distribution Systems ◽  
Materials Science ◽  
Biological Properties ◽  
Delivery Systems ◽  
Hereditary Diseases ◽  
Delivery Technique ◽  
Wide Range

Cyclodextrins (CDs)-based techniques of delivering nucleic acid have considerable potential in gene therapy. A clinical study using a CD-based delivery technique to distribute siRNA to patients with solid tumors was done. The ribonucleotide reductase (RRM2)-related siRNA utilized in that work was designed to reduce cancer. Besides melanoma research, CD-like delivery systems were examined as therapeutic techniques for hepatoma, glioma, breast cancer, and cardiovascular diseases. Preclinical and clinical studies that have been linked have shown a tremendous amount of therapeutic promise. Several difficulties remain before clinical studies may employ CD-based nucleic acid delivery methods. In addition, easy modification techniques should be researched to ensure the security of the delivery systems. CD's intrinsic advantages, including its exceptional biological properties and unique structure, enable the sensible design of a wide range of distribution systems. CDs are anticipated to be used for clinical gene therapy transformation with the progress of relevant research and ongoing materials science and biology development. The research focuses on cancer therapy, but other hereditary diseases may benefit from increased attention.

Gene therapy promises accurate, targeted administration and overcoming drug resistance in diverse cancer cells

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Drug Resistance ◽  
Nucleic Acid ◽  
Nucleic Acids ◽  
Clinical Research ◽  
Cancer Cells ◽  
Cationic Lipids ◽  
Target Cells ◽  
Genetic Components ◽  
Wide Range

Nucleic acid-based therapeutics such as siRNA and miRNA employ the silencing capabilities of the RNAi mechanism to affect the expression of one gene or several genes in target cells. Nucleic acid-based therapies enable accurate, targeted administration and overcoming drug resistance in diverse cancer cells. Several studies have shown that they can be utilized alongside pharmacological therapy to increase the efficacy of existing therapies. In addition, nucleic acid-based therapies have the potential to widen the spectrum of druggable targets for a range of diseases and emerge as a novel therapeutic technique for treating a number of diseases that are today untreatable. Nucleic acids are dependent on their effective distribution to target cells, which need correct complexation and encapsulation in a delivery mechanism. Although nucleic acids exist in a variety of forms and sizes, their physical and chemical commonality allow them to be loaded into a wide range of delivery vehicles. The primary biomaterials used to encapsulate genetic components were cationic lipids and polymers. Furthermore, the experiments focused particularly on effective transfection in target cells.Recent breakthroughs in NP-based RNA therapeutics have spurred a flood of clinical research, facing many challenges. In vivo, pharmacokinetics of different RNA-based medications must be researched to establish the viability and therapeutic potential of nucleic acid-based therapeutics. The U.S. Food and Drug Administration recently authorized many NP-based gene therapy. In 2019, Novartis authorized Zolgensma (onasemnogene abeparvovec-xioi) to treat spinal muscle atrophy. The first clinical research employing siRNA began in 2004 and is considered a milestone in nucleic acid-based drug development. Thirty clinical investigations have subsequently been completed. In 2018, the US FDA cleared Onpattro (Patisiran, Alnylam Pharmaceuticals) for the treatment of polyneuropathy caused by transthyretin amyloidosis.Several new generations of nucleic acid compositions employing polymer nanoparticles or liposomes are presently undergoing clinical testing. If allowed, the debut of nucleic acid-based treatments would represent a watershed event in immunotherapy. Advances in the design and development of biocompatible nanomaterials would allow us to overcome the above-mentioned problems and so show the potential to deliver nucleic acids in the treatment of a number of illnesses.

Advanced Technologies in DNA-based Nanomedicine

2021 ◽  
Vol 15 ◽  
Author(s):  
Ameneh Mohammadi ◽  
Pooria Gill ◽  
Pedram Ebrahimnejad ◽  
Said Abediankenari ◽  
Zahra Kashi
Keyword(s):  
Drug Delivery ◽  
Gene Therapy ◽  
Drug Delivery Systems ◽  
Delivery Systems ◽  
Dna Nanostructures ◽  
Medical Sciences ◽  
Rational Engineering ◽  
Advanced Technologies ◽  
Wide Range ◽  
Or Gene

: The application of nanotechnology in medicine and pharmaceutical purpose suggested a novel procedure in the nanotechnology terminology as nanomedicine. There is a wide range of applications for nanotechnology in medicine, such as the use of nanocarriers in drug delivery systems. Recently a remarkable attention to DNA has been made through its amazing functionality and its nature as a nanomaterial in biological systems. Since DNA is a biocompatible, the use of DNA as a nanomaterial in medicine has shown a great perspective of rational engineering of DNA nanostructures. According to new approaches in treatment of diseases in gene levels, gene therapy, using DNA as a nanomedicine possesses an important role in the medical sciences as the researchers published enormous papers and patents in the fields, for instance, the applications of DNA and DNA-based nanostructures as drug or gene nanocarriers, DNA-based diagnostics and DNA nanovasccines. Here, some examples of DNA-based nanomedicine in the patent frame were reviewed.

scholarly journals Harnessing Intranasal Delivery Systems of Sumatriptan for the Treatment of Migraine

2022 ◽  
Vol 2022 ◽  
pp. 1-9
Author(s):  
Sara Assadpour ◽  
Mohammad Reza Shiran ◽  
Peyman Asadi ◽  
Javad Akhtari ◽  
Amirhossein Sahebkar
Keyword(s):  
Clinical Studies ◽  
Large Fraction ◽  
Hepatic Metabolism ◽  
Oral Route ◽  
Delivery Systems ◽  
Clinical Effects ◽  
Beneficial Effects ◽  
Wide Range ◽  
Preclinical And Clinical Studies ◽  
Novel Delivery Systems

Sumatriptan (ST) is a commonly prescribed drug for treating migraine. The efficiency of several routes of ST administration has been investigated. Recently, the intranasal route with different delivery systems has gained interest owing to its fast-acting and effectiveness. The present study is aimed at reviewing the available studies on novel delivery systems for intranasal ST administration. The oral route of ST administration is common but complicated with some problems. Gastroparesis in patients with migraine may reduce the absorption and effectiveness of ST upon oral use. Furthermore, the gastrointestinal (GI) system and hepatic metabolism can alter the pharmacokinetics and clinical effects of ST. The bioavailability of conventional nasal liquids is low due to the deposition of a large fraction of the delivered dose of a drug in the nasal cavity. Several delivery systems have been utilized in a wide range of preclinical and clinical studies to enhance the bioavailability of ST. The beneficial effects of the dry nasal powder of ST (AVP-825) have been proven in clinical studies. Moreover, other delivery systems based on microemulsions, microspheres, and nanoparticles have been introduced, and their higher bioavailability and efficacy were demonstrated in preclinical studies. Based on the extant findings, harnessing novel delivery systems can improve the bioavailability of ST and enhance its effectiveness against migraine attacks. However, further clinical studies are needed to approve the safety and efficacy of employing such systems in humans.

scholarly journals The Use of Viral Vectors in Gene Transfer Therapy

2016 ◽  
Vol 8 (03) ◽  
Author(s):  
A. Dziaková ◽  
A. Valenčáková ◽  
E. Hatalová ◽  
J. Kalinová
Keyword(s):  
Gene Therapy ◽  
Plasmid Dna ◽  
Clinical Studies ◽  
Viral Vectors ◽  
Disease Gene ◽  
Major Gene ◽  
Genetic Material ◽  
The Body ◽  
Disease Genes ◽  
Wide Range

Gene therapy is strategy based on using genes as pharmaceuticals. Gene therapy is a treatment that involves altering the genes inside body's cells to stop disease. Genes contain DNA- the code controlling body form and function. Genes that do not work properly can cause disease. Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve the ability of the body to fight disease. Gene therapy holds promise for treating a wide range of diseases, including cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. Various types of genetic material are used in gene therapy; double-stranded DNA (dsDNA), single-stranded DNA (ssDNA), plasmid DNA and antisense oligodeoxynucleotides (ASON). The success of gene therapy depends on assuring the entrance of the therapeutic gene to targeted cells without any form of biodegradation. Commonly used vectors in gene therapy are: adenoviruses (400 clinical studies; 23.8%), retroviruses (344 clinical studies; 20.5%), unenveloped/plasmid DNA (304 clinical studies, 17.7%), adenoassociated viruses (75 clinical studies; 4.5%) and others. In this paper, we have reviewed the major gene delivery vectors and recent improvements made in their design meant to overcome the issues that commonly arise with the use of gene therapy vectors.

Advances in nano-biomaterials and their applications in biomedicine

2021 ◽  
Author(s):  
Yogita Patil-Sen
Keyword(s):  
Tissue Engineering ◽  
Regenerative Medicine ◽  
Biomedical Applications ◽  
Materials Science ◽  
Disease Diagnosis ◽  
Biological Properties ◽  
The Past ◽  
Physico Chemical ◽  
Wide Range ◽  
Biomolecules Detection

Nano0technology has received considerable attention and interest over the past few decades in the field of biomedicine due to the wide range of applications it provides in disease diagnosis, drug design and delivery, biomolecules detection, tissue engineering and regenerative medicine. Ultra-small size and large surface area of nanomaterials prove to be greatly advantageous for their biomedical applications. Moreover, the physico-chemical and thus, the biological properties of nanomaterials can be manipulated depending on the application. However, stability, efficacy and toxicity of nanoparticles remain challenge for researchers working in this area. This mini-review highlights the recent advances of various types of nanoparticles in biomedicine and will be of great value to researchers in the field of materials science, chemistry, biology and medicine.

scholarly journals From traditional pharmacological towards nucleic acid-based therapies for cardiovascular diseases

2020 ◽  
Vol 41 (40) ◽  
pp. 3884-3899 ◽  
Author(s):  
Ulf Landmesser ◽  
Wolfgang Poller ◽  
Sotirios Tsimikas ◽  
Patrick Most ◽  
Francesco Paneni ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Nucleic Acid ◽  
Cardiovascular Diseases ◽  
Genome Editing ◽  
Clinical Studies ◽  
Large Scale ◽  
Therapeutic Targets ◽  
Targeted Therapeutics ◽  
Gene Therapies

Abstract Nucleic acid-based therapeutics are currently developed at large scale for prevention and management of cardiovascular diseases (CVDs), since: (i) genetic studies have highlighted novel therapeutic targets suggested to be causal for CVD; (ii) there is a substantial recent progress in delivery, efficacy, and safety of nucleic acid-based therapies; (iii) they enable effective modulation of therapeutic targets that cannot be sufficiently or optimally addressed using traditional small molecule drugs or antibodies. Nucleic acid-based therapeutics include (i) RNA-targeted therapeutics for gene silencing; (ii) microRNA-modulating and epigenetic therapies; (iii) gene therapies; and (iv) genome-editing approaches (e.g. CRISPR-Cas-based): (i) RNA-targeted therapeutics: several large-scale clinical development programmes, using antisense oligonucleotides (ASO) or short interfering RNA (siRNA) therapeutics for prevention and management of CVD have been initiated. These include ASO and/or siRNA molecules to lower apolipoprotein (a) [apo(a)], proprotein convertase subtilisin/kexin type 9 (PCSK9), apoCIII, ANGPTL3, or transthyretin (TTR) for prevention and treatment of patients with atherosclerotic CVD or TTR amyloidosis. (ii) MicroRNA-modulating and epigenetic therapies: novel potential therapeutic targets are continually arising from human non-coding genome and epigenetic research. First microRNA-based therapeutics or therapies targeting epigenetic regulatory pathways are in clinical studies. (iii) Gene therapies: EMA/FDA have approved gene therapies for non-cardiac monogenic diseases and LDL receptor gene therapy is currently being examined in patients with homozygous hypercholesterolaemia. In experimental studies, gene therapy has significantly improved cardiac function in heart failure animal models. (iv) Genome editing approaches: these technologies, such as using CRISPR-Cas, have proven powerful in stem cells, however, important challenges are remaining, e.g. low rates of homology-directed repair in somatic cells such as cardiomyocytes. In summary, RNA-targeted therapies (e.g. apo(a)-ASO and PCSK9-siRNA) are now in large-scale clinical outcome trials and will most likely become a novel effective and safe therapeutic option for CVD in the near future. MicroRNA-modulating, epigenetic, and gene therapies are tested in early clinical studies for CVD. CRISPR-Cas-mediated genome editing is highly effective in stem cells, but major challenges are remaining in somatic cells, however, this field is rapidly advancing.

scholarly journals Nano- and Microcarriers as Drug Delivery Systems for Usnic Acid: Review of Literature

Pharmaceutics ◽  
2020 ◽  
Vol 12 (2) ◽  
pp. 156 ◽  
Author(s):  
Ana Zugic ◽  
Vanja Tadic ◽  
Snezana Savic
Keyword(s):  
Drug Delivery ◽  
Drug Delivery Systems ◽  
Usnic Acid ◽  
Chemical Properties ◽  
Biological Properties ◽  
Delivery Systems ◽  
Physico Chemical ◽  
Wide Range ◽  
Number Of Publications ◽  
Promising Therapeutic Agent

Usnic acid is one of the most investigated lichen secondary metabolites, with several proven biological properties with potential medical relevance. However, its unfavorable physico-chemical properties, as well as observed hepatotoxicity, have discouraged wide-range utilization of usnic acid as a promising therapeutic agent. In accordance with the growing research interest in the development of nanotechnology, especially in the arena of preparations based on natural sources of medicinal compounds, usnic acid incorporated into nano- and microsized colloidal carriers has been a subject of a large number of publications. Therefore, this review discusses the overall results of the studies dealing with usnic acid encapsulated into lipid-based, polymeric and nonorganic micro- and/or nanocarriers, as potential drug delivery systems for this natural compound, in an attempt to introduce its usage as a potential antitumor, antimicrobial, wound-healing, antioxidative and anti-inflammatory drug.

Evaluation of the nucleopolyhedrovirus of Anticarsia gemmatalis as a vector for gene therapy in mammals

2020 ◽  
Vol 20 ◽  
Author(s):  
Cintia N. Parsza ◽  
Diego L. Mengual Gómez ◽  
Jorge Alejandro Simonin ◽  
Mariano Nicolás Belaich ◽  
Pablo Daniel Ghiringhelli
Keyword(s):  
Gene Therapy ◽  
Mammalian Cells ◽  
Insect Cells ◽  
Autographa Californica ◽  
Anticarsia Gemmatalis ◽  
Agricultural Pests ◽  
Mammalian Cell Lines ◽  
Delivery Vector ◽  
Wide Range ◽  
Insect Pathogens

Background: Baculoviruses are insect pathogens with important biotechnological applications that transcend their use as biological controllers of agricultural pests. One species, Autographa californica multiple nucleopolhyedrovirus (AcMNPV) has been extensively exploited as a molecular platform to produce recombinant proteins and as a delivery vector for genes in mammals, because it can transduce a wide range of mammalian cells and tissues without replicating or producing progeny. Objective/Method: To investigate if the budded virions of Anticarsia gemmatalis multiple nucleopolhyedrovirus (AgMNPV) species has the same ability, the viral genome was modified by homologous recombination into susceptible insect cells to integrate reporter genes and then it was evaluated on mammalian cell lines in comparative form with respect to equivalent viruses derived from AcMNPV. Besides, the replicative capacity of AgMNPV´s virions in mammals was determined. Results: The experiments carried out showed that the recombinant variant of AgMNPV transduces and support the expression of delivered genes but not replicates in mammalian cells. Conclusion: Consequently, this insect pathogen is proposed as an alternative of non-infectious viruses in humans to explore new approaches in gene therapy and other applications based on the use of mammalian cells.

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