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) exhibited efficient intracellular delivery and superior anti-tumor efficiency with simultaneous up and down-regulation of PTEN and VEGF, respectively. The MSN system can be considered as a new platform for cancer gene therapy requiring accurate control of multiple gene expressions.Graphical abstract
Source: Journal of Industrial and Engineering Chemistry - Category: Chemistry Source Type: research

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(CNN) — The US Food and Drug Administration approved a treatment Friday for a genetic disease called spinal muscular atrophy that causes infants’ muscles to waste away, potentially killing them before age 2. And then came the price tag: $2.125 million for a one-time treatment. The gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis. “Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” Dr. Ned Sharpless, the FDA’s acting commissioner, said in a statement Friday. &ldqu...
Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - Category: Consumer Health News Authors: Tags: Health News CNN Novartis Source Type: news
ConclusionTo that end, a KID syndrome cell line (KID-KC) was established from primary keratinocytes of a KID syndrome patient with heterozygous p.D50N mutation, which displayed impaired gap junction intercellular communication and hyperactive hemichannels, confirmed by dye transfer, patch clamp and neurobiotin uptake assays. In KID-KCs, treatment with AS-siRNA led to robust inhibition of the mutant GJB2 allele without altering expression of the wildtype allele. This resulted in correction of both gap junction intercellular communication and hemichannel activity. Furthermore, AS-siRNA treatment caused only low-level off-tar...
Source: Cytotherapy - Category: Cytology Source Type: research
Authors: Sun W, Shen NM, Fu SL Abstract Cervical cancer (CC) develops, after human papillomavirus (HPV), an infection transmitted through sexual contact. Worldwide estimates are around>500,000 CC diagnoses and>300,000 related deaths annually, and CC remains the second most devastating type of cancers in women after breast cancer. Although the vaccine against HPV has reduced the incidence of infection and the treatment efficacy of the early-stage diagnoses has improved, many challenges remain in terms of treatment efficacy, during the late-stage and prevention of chemotherapy resistance development. Thus, new ...
Source: European Review for Medical and Pharmacological Sciences - Category: Drugs & Pharmacology Tags: Eur Rev Med Pharmacol Sci Source Type: research
Authors: Nieddu V, Piredda R, Bexell D, Barton J, Anderson J, Sebire N, Kolluri K, Janes SM, Karteris E, Sala A Abstract Drug-resistant neuroblastoma remains a major challenge in paediatric oncology and novel and less toxic therapeutic approaches are urgently needed to improve survival and reduce the side effects of traditional therapeutic interventions. Mesenchymal stem cells (MSCs) are an attractive candidate for cell and gene therapy since they are recruited by and able to infiltrate tumours. This feature has been exploited by creating genetically modified MSCs that are able to combat cancer by delivering therap...
Source: Oncology Reports - Category: Cancer & Oncology Tags: Oncol Rep Source Type: research
Here, one of the leading researchers working on the biochemistry of senescent cells - and their relevance to aging - considers the state of development of senolytic therapies. These are treatments, largely small molecule drugs at this stage, but also including suicide gene therapies, immunotherapies, and more, that are capable of selectively destroying some fraction of the senescent cells present in old tissues. There is tremendous enthusiasm in the scientific and development communities for the potential to create significant degrees of rejuvenation via this approach. The results in mice are far and away more impressive a...
Source: Fight Aging! - Category: Research Authors: Tags: Daily News Source Type: blogs
The lengthy and somewhat overwrought article I'll point out today is a good example of the way in which journalists fail when writing on the topic of the growing biotechnology industry that is making the first steps towards the medical control of aging. They talk to just a few people, and thus have a very narrow (generously) or absolutely incorrect (more accurately) view of what might be happening, the prospects for the future, and the shape of the field as a whole. In this case the few people are the folk at AgeLab at MIT, and George Church, with a focus on the veterinary deployment of gene therapies by Rejuvenate Bio, an...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
Fight Aging! provides a weekly digest of news and commentary for thousands of subscribers interested in the latest longevity science: progress towards the medical control of aging in order to prevent age-related frailty, suffering, and disease, as well as improvements in the present understanding of what works and what doesn't work when it comes to extending healthy life. Expect to see summaries of recent advances in medical research, news from the scientific community, advocacy and fundraising initiatives to help speed work on the repair and reversal of aging, links to online resources, and much more. This content is...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Publication date: 1 October 2019Source: Chemical Engineering Journal, Volume 373Author(s): Santanu Ghosh, Krishanu Ghosal, Sk Arif Mohammad, Kishor SarkarAbstractTriple negative breast cancer (TNBC) is the most fatal subtype among all other types of breast cancer and there are no targeted chemotherapy for the treatment of TNBC due to the absence of estrogen receptor (ER), progesterone receptor (PR) and human epidermal growth factor receptor-2 (HER-2). To address this problem, we develop a targeted non-viral vector which could efficiently deliver the gene of interest and could also diagnose of TNBC. We have synthesized carb...
Source: Chemical Engineering Journal - Category: Chemistry Source Type: research
Authors: Yao H, Sun Q, Zhu J Abstract The high mortality of colorectal cancer (CRC) is likely caused by early invasion and metastasis. The chemoresistance of tumor cells is the critical reason for treatment failure. The present study aimed to develop targeted solutions to overcome chemotherapy drug resistance in CRC. CCK-8 assay was used to examine SW480 cell viability. SW480 cell apoptosis was examined using flow cytometry. The present study demonstrated that the expression of miR-1271 was significantly decreased in CRC tumors and cell lines compared with control tissues. Furthermore, the expression of microRNA (m...
Source: Experimental and Therapeutic Medicine - Category: General Medicine Tags: Exp Ther Med Source Type: research
Glycogen storage disease type Ib (GSD-Ib) is an autosomal recessive disorder caused by deficiencies in glucose-6-phosphate transporter (G6PT), a ubiquitously expressed endoplasmic reticulum (ER) protein that translocates G6P from the cytoplasm into the ER lumen.   Inside the ER, G6P is hydrolyzed to glucose and phosphate by either the liver/kidney/intestine-restricted glucose-6-phosphatase-α (G6Pase-α or G6PC) or the ubiquitously expressed G6Pase-β.  G6PT and G6Pase are functionally co-dependent and form the G6PT/G6Pase complexes. The G6PT/G6Pase-α co mplex maintains interprandial blood glu...
Source: NIH OTT Licensing Opportunities - Category: Research Authors: Source Type: research
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