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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Lipid-based siRNA Nanodelivery Systems: a Learning Process for Improving Transfer from Concepts to Clinical Applications.
CONCLUSION: Formulation design should be increasingly addressed with industrial criteria; it should be based on quality by design and on the estimation of critical attributes that affect performance, and supported by a range of characterization techniques and appropriate analytical methods. PMID: 30160217 [PubMed - as supplied by publisher]
Source: Current Clinical Pharmacology - August 29, 2018 Category: Drugs & Pharmacology Authors: Perez SE, Carlucci AM Tags: Curr Clin Pharmacol Source Type: research

Tunability of Biodegradable Poly(amine-co-ester) Polymers for Customized Nucleic Acid Delivery and Other Biomedical Applications.
Abstract Gene therapy promises to treat diseases that arise from genetic abnormalities by correcting the underlying cause of the disease rather than treating the associated symptoms. Successful transfer of nucleic acids into cells requires efficient delivery vehicles that protect the cargo and can penetrate the appropriate cellular barriers before releasing their contents. Many viral vectors and synthetic polycationic vectors for nucleic acid delivery do not translate well from in vitro to in vivo applications due to their instability and toxicity. We synthesized and characterized a library of biocompatible low ch...
Source: Biomacromolecules - August 15, 2018 Category: Biochemistry Authors: Kauffman AC, Piotrowski-Daspit AS, Nakazawa KH, Jiang Y, Datye A, Saltzman WM Tags: Biomacromolecules Source Type: research

Current non-viral siRNA delivery systems as a promissing treatment of skin diseases.
CONCLUSION: The treatment of skin diseases based on topical delivery of siRNA, which act by inhibiting the expression of target transcripts, offers many potential therapeutic advantages for suppressing genes into the skin. PMID: 30084329 [PubMed - as supplied by publisher]
Source: Current Pharmaceutical Design - August 7, 2018 Category: Drugs & Pharmacology Authors: Rosa J, Suzuki I, Kravicz M, Caron A, Pupo AV, Praca FG, Bentley MVLB Tags: Curr Pharm Des Source Type: research

Nanostructured Hyaluronic acid-based materials for the delivery of siRNA.
CONCLUSION: To overcome the problems associated with these emerging genetic tools, investigators have employed glycosaminoglycan HA-based biopolymers. This biopolymer offers a variety of properties such as biodegradability, biocompatibility, aqueous solubility, viscoelasticity, and non-immunogenicity. PMID: 30084325 [PubMed - as supplied by publisher]
Source: Current Pharmaceutical Design - August 7, 2018 Category: Drugs & Pharmacology Authors: Shah K, Chawla S, Agrawal A, Reddy G, Maheshwari R, Kalia K, Tekade RK Tags: Curr Pharm Des Source Type: research

Destination Penis? Erectile Dysfunction As Possible Future Indication Of Therapeutic Gene Delivery.
Abstract Erectile Dysfunction (ED) is a common health problem in roughly 50% of males of advanced age (40-70 years old). Recent attention related gene therapy to ED cases; this received much interest to further progress gene therapy ideals to ED treatment. This review is an attempt to analyze key challenges and to emphasize primary areas, including mostly preclinical and few clinical trials, cellular target(s), and different viral vectors/nanoparticles for gene delivery in ED. While overexpression of target genes can be silenced by RNA interference (RNAi), down-regulation of these mechanisms has been implicated in...
Source: Current Gene Therapy - July 29, 2018 Category: Genetics & Stem Cells Authors: Gur S, Abdel-Mageed AB, Sikka SC, Bartolome AR, Hellstrom WJG Tags: Curr Gene Ther Source Type: research

Polyester-based nanoparticles for nucleic acid delivery
Publication date: Available online 25 July 2018Source: Materials Science and Engineering: CAuthor(s): Jing Zhao, Guojun Weng, Jianjun Li, Jian Zhu, Junwu ZhaoAbstractGene therapy is promising for the treatment of inherited diseases and complex diseases such as chronic infections and cancer. The advancement in science and technology has extended the scope of gene therapy from gene transfer to the delivery of a variety of nucleic acids such as mRNA, antisense oligonucleotides (ASOs), siRNA and miRNA. Nanoparticle delivery systems can efficiently protect the nucleic acids from enzymatic degradation and immune recognition, fac...
Source: Materials Science and Engineering: C - July 26, 2018 Category: Materials Science Source Type: research

Micelle-like nanoparticles as siRNA and miRNA carriers for cancer therapy.
Abstract Gene therapy has emerged as an alternative in the treatment of cancer, particularly in cases of resistance to chemo and radiotherapy. Different approaches to deliver genetic material to tumor tissues have been proposed, including the use of small non-coding RNAs due to their multiple mechanisms of action. However, such promise has shown limits in in vivo application related to RNA's biological instability and stimulation of immunity, urging the development of systems able to overcome those barriers. In this review, we discuss the use of RNA interference in cancer therapy with special attention to the role...
Source: Biomedical Microdevices - July 12, 2018 Category: Biomedical Engineering Authors: Costa DF, Torchilin VP Tags: Biomed Microdevices Source Type: research

Nanoscale polyelectrolyte complexes encapsulating mRNA and long-chained siRNA for combinatorial cancer gene therapy
Publication date: 25 August 2018Source: Journal of Industrial and Engineering Chemistry, Volume 64Author(s): Myung Goo Kim, Sung Duk Jo, Ji Hoon Jeong, Sun Hwa KimAbstractTo precisely regulate target genes that are abnormally expressed in cancers, we suggest an RNA-mediated multigene targeting system that co-encapsulates siRNA against vascular endothelial growth factor (VEGF) and mRNA encoding phosphatase and tensin homolog (PTEN). Polymerized long-chain siRNAs (L-siRNAs) formed stable and condensed nanocomplexes with mRNAs using thiolated glycol chitosans (tGCs) as gene carriers. The mRNA/L-siRNA/tGC nanocomplexes (MSNs) ...
Source: Journal of Industrial and Engineering Chemistry - July 11, 2018 Category: Chemistry Source Type: research

Potential of siRNA-albumin complex against cancer
Publication date: Available online 27 April 2018Source: Chemico-Biological InteractionsAuthor(s): Na Liu, Yan-Hua Qi, Chuan-Tao Cheng, Wen Bin Yang, Anshoo Malhotra, Qi ZhouAbstractRNA interference is a highly specific as well as efficient technology for gene therapy application in molecular oncology. The present study was planned to develop an efficient and stable tumor selective delivery mechanism for siRNA gene therapy for the purpose of both diagnosis as well as therapy. We have used 20 Male wistar rats for the formation of colon cancer model and utilized albumin as carrier molecule for the delivery of siRNA against va...
Source: Chemico Biological Interactions - July 11, 2018 Category: Biochemistry Source Type: research

Evaluation of siRNA and Cationic Liposomes Complexes as a Model for in vitro siRNA Delivery to Cancer Cells
This study aims to investigate the effect of different molar charge ratios (R+/-) between positive charges from microfluidics-produced cationic liposomes (CL) (egg phosphatidylcholine, 1,2-dioleoyl-3-trimethylammonium-propane and 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine) and negative charges from siRNA and on physico-chemical and morphological properties of the lipoplexes (CL/siRNA) as well as their in vitro luciferase silencing effect in HeLa cells. R+/- 3.27 was found to be the optimum point for complexation. This finding was confirmed by gel retardation and siRNA accessibility assays. According to Cryo-TEM analysis...
Source: Colloids and Surfaces A: Physicochemical and Engineering Aspects - July 11, 2018 Category: Chemistry Source Type: research

Potential of siRNA-albumin complex against cancer
Publication date: Available online 27 April 2018Source: Chemico-Biological InteractionsAuthor(s): Na Liu, Yan-Hua Qi, Chuan-Tao Cheng, Wen Bin Yang, Anshoo Malhotra, Qi ZhouAbstractRNA interference is a highly specific as well as efficient technology for gene therapy application in molecular oncology. The present study was planned to develop an efficient and stable tumor selective delivery mechanism for siRNA gene therapy for the purpose of both diagnosis as well as therapy. We have used 20 Male wistar rats for the formation of colon cancer model and utilized albumin as carrier molecule for the delivery of siRNA against va...
Source: Chemico Biological Interactions - July 5, 2018 Category: Biochemistry Source Type: research

Nanoscale polyelectrolyte complexes encapsulating mRNA and long-chained siRNA for combinatorial cancer gene therapy
Publication date: 25 August 2018Source: Journal of Industrial and Engineering Chemistry, Volume 64Author(s): Myung Goo Kim, Sung Duk Jo, Ji Hoon Jeong, Sun Hwa KimAbstractTo precisely regulate target genes that are abnormally expressed in cancers, we suggest an RNA-mediated multigene targeting system that co-encapsulates siRNA against vascular endothelial growth factor (VEGF) and mRNA encoding phosphatase and tensin homolog (PTEN). Polymerized long-chain siRNAs (L-siRNAs) formed stable and condensed nanocomplexes with mRNAs using thiolated glycol chitosans (tGCs) as gene carriers. The mRNA/L-siRNA/tGC nanocomplexes (MSNs) ...
Source: Journal of Industrial and Engineering Chemistry - July 5, 2018 Category: Chemistry Source Type: research

Lipid-based nanocarriers for siRNA delivery: challenges, strategies and the lessons learned from the DODAX:MO liposomal system.
Abstract The possibility of using the RNA interference (RNAi) mechanisms in gene therapy was one of the scientific breakthroughs of the last century. Despite the extraordinary therapeutic potential of this approach, the need for an efficient gene carrier is hampering the translation of the RNAi technology to the clinical setting. Although a diversity of nanocarriers have been described, liposomes continue to be one of the most attractive siRNA vehicles due to their relatively low toxicity, facilitated siRNA complexation, high transfection efficiency and enhanced pharmacokinetic properties. This review focuses on R...
Source: Current Drug Targets - July 3, 2018 Category: Drugs & Pharmacology Authors: Oliveira ACN, Fernandes J, Goncalves A, Gomes AC, Oliveira R Tags: Curr Drug Targets Source Type: research

Engineered Exosomes for Targeted Transfer of siRNA to HER2 Positive Breast Cancer Cells.
Abstract Exosomes are the best options for gene targeting, because of their natural, nontoxic, non-immunogenic, biodegradable, and targetable properties. By engineering exosome-producing cells, ligands can be expressed fusing with exosomal surface proteins for targeting cancer cell receptors. In the present study, HER2-positive breast cancer cells were targeted with a modified exosome producing engineered HEK293T cell. For this purpose, the HEK293T cells were transduced by a lentiviral vector bearing-LAMP2b-DARPin G3 chimeric gene. Stable cells expressing the fusion protein were selected, and the exosomes produced...
Source: Applied Biochemistry and Biotechnology - June 28, 2018 Category: Biochemistry Authors: Limoni SK, Moghadam MF, Moazzeni SM, Gomari H, Salimi F Tags: Appl Biochem Biotechnol Source Type: research