Recombinant adeno-associated virus 9-mediated expression of Kallistatin suppresses lung tumor growth in mice.
CONCLUSION: The results indicate that this therapeutic strategy is a promising approach for clinical cancer therapy and impli-cate rAAV9-Kal as a candidate for gene therapy of lung cancer. PMID: 33183200 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 11, 2020 Category: Genetics & Stem Cells Authors: Qu W, Zhao J, Wu Y, Xu R, Liu S Tags: Curr Gene Ther Source Type: research

Epigenetic modifications in acute lymphoblastic leukemia: From cellular mechanisms to therapeutics.
CONCLUSION: According to the literature review, leukemogenesis of ALL is extensively influenced by epigenetic modifica-tions, particularly DNA hyper-methylation, histone modification, and miRNA alteration. PMID: 33183201 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 11, 2020 Category: Genetics & Stem Cells Authors: Fathi E, Farahzadi R, Montazersaheb S, Bagheri Y Tags: Curr Gene Ther Source Type: research

Standardizing the effective correlated dosage of olanzapine and empagli-flozin in female Wistar rats.
CONCLUSION: We conclude that Ola-4 and EMPA-20 were the most effective dosage for experimental purposes in female Wistar rats. The findings of this study standardized the effective correlated dosage of olanzapine and empagliflozin in female Wistar rats that will help un-derstand the underlying molecular and behavioral mechanisms. PMID: 33183202 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 11, 2020 Category: Genetics & Stem Cells Authors: Ashraf GM, Alghamdi BS, Alshehri FS, Alam MZ, Tayeb HO, Tarazi FI Tags: Curr Gene Ther Source Type: research

Challenges of Gene Therapy for Neurodegenerative Disorders.
PMID: 33153421 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 5, 2020 Category: Genetics & Stem Cells Authors: Uddin MS, Khan ZA, Sumsuzzman DM, Perveen A, Ashraf GM Tags: Curr Gene Ther Source Type: research

Gene Therapy for Neuroprotection and Neurorestoration (Part II).
PMID: 33054704 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 20, 2020 Category: Genetics & Stem Cells Authors: Ashraf GM, Uddin MS Tags: Curr Gene Ther Source Type: research

Promising Anti-stroke Signature of Voglibose: Investigation through In- Silico Molecular Docking and Virtual Screening in In-Vivo Animal Studies.
Abstract BACKGROUND: Postprandial hyperglycemia considered to be a major risk factor for cerebrovascular complications. OBJECTIVE: The current study was designed to elucidate the beneficial role of voglibose via in-silico in vitro to in-vivo studies in improving the postprandial glycaemic state by protection against strokeprone type 2 diabetes. MATERIALS AND METHODS: In-Silico molecular docking and virtual screening were carried out with the help of iGEMDOCK+ Pymol+docking software and Protein Drug Bank database (PDB). Based on the results of docking studies, in-vivo investigation was carried out for pos...
Source: Current Gene Therapy - October 20, 2020 Category: Genetics & Stem Cells Authors: Shah P, Chavda V, Patel S, Bhadada S, Ashraf GM Tags: Curr Gene Ther Source Type: research

CRISPR/Cas9 genome editing tool: A Promising Tool for Therapeutic Applications on respiratory diseases.
Abstract Respiratory diseases are one of the prime topics of concern in the current era due to improper diagnostics tools. Gene-editing therapy like Clustered regularly interspaced palindromic repeats-associated nuclease 9 (CRISPR/Cas9) is gaining popularity in pulmonary research opening up doors to invaluable insights on underlying mechanisms. CRISPR/Cas9 can be considered as a potential gene editing tool with a scientific community that is helping in the advancement of knowledge in respiratory health and therapy. As an appealing therapeutic tool, we hereby explore the advanced research on the application of CRIS...
Source: Current Gene Therapy - October 12, 2020 Category: Genetics & Stem Cells Authors: Shaikh SB, Bhandary YP Tags: Curr Gene Ther Source Type: research

Nanoparticle-Based Gene Therapy Intervention for Stroke Treatment: a Systematic Review.
In conclusion, due to the inability of brain regeneration and the importance of genes in stroke-related complications, gene therapy seems to be a suitable treatment strategy. The use of suitable nanoparticles for transportation ensures the efficiency and usefulness of this method. PMID: 33045966 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 12, 2020 Category: Genetics & Stem Cells Authors: Ghasemi S, Alavian K, Alavian F Tags: Curr Gene Ther Source Type: research

A five-gene signature for predicting the prognosis of colorectal cancer.
In this study, we established a new prognostic model for CRC. Four groups of CRC data were accessed from GEO database, and then differential analysis (logFoldChange>1, adjustP
Source: Current Gene Therapy - October 12, 2020 Category: Genetics & Stem Cells Authors: Hong J, Lin X, Hu X, Wu X, Fang W Tags: Curr Gene Ther Source Type: research

Overexpressing Runx2 of BMSCs improve the repairment of knee cartilage defects.
Abstract BACKGROUND: Recruitment of gene modify bone marrow mesenchymal stem cells (BMSCs) has been considered an alternative to single cell injection in articular cartilage repair. PURPOSE: The aim of this study was to investigate the effect whether of runt-related transcription factor 2(Runx2) overexpression bone marrow mesenchymal stem cells in vivo could improve the quality of repaired tissue of a knee cartilage defect in a rabbit model. METHODS: Thirty-two New Zealand rabbits were randomly divided into four groups.The blank group (Con) don't received anything, the model group (Mo) was administered s...
Source: Current Gene Therapy - October 4, 2020 Category: Genetics & Stem Cells Authors: Hu J, Zou WZ, Li L, Shi ZS, Liu XZ, Cai HT, Yang AF, Sun DM, Xu LL, Yang Y, Li ZH Tags: Curr Gene Ther Source Type: research

Delivery Systems for RNA Interference Therapy: Current Technologies and Limitations.
Abstract In recent years, RNA interference technology has been extensively studied for its therapeutic potential against a wide variety of diseases. It aims to silence the expression of undesired genes associated with the target disease by administration of RNA interference agents. However, these agents (nucleic acids) are unstable in the circulatory system and lack target specificity. Drug delivery systems are therefore crucial for successful practice of the technique. A wide array of delivery systems has been developed to conquer these challenges, such as viral vectors, inorganic drug carriers, polymeric carrier...
Source: Current Gene Therapy - October 4, 2020 Category: Genetics & Stem Cells Authors: Wang Y Tags: Curr Gene Ther Source Type: research

Mesenchymal stem cells promote caspase expression in Molt-4 leukemia cells via GSK-3 α/β and ERK1/2 signaling pathways as a therapeutic strategy.
CONCLUSIONS: It was concluded that MSCs co-cultured with Molt-4 cells could be involved in the promotion of Molt-4 cell apoptosis and cell senescence via caspase-8, 9 cascade and GSK-3α/β and ERK1/2 signaling pathways. PMID: 33019931 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 4, 2020 Category: Genetics & Stem Cells Authors: Fathi E, Vietor I Tags: Curr Gene Ther Source Type: research

In-silico Molecular Interaction of Short Synthetic Lipopeptide/Importinalpha and In-vitro Evaluation of Transgene Expression Mediated by Liposome-Based Gene Carrier.
CONCLUSION: Our study for the first time has shown that the fully synthesized short lipopeptide Pal-CKKHH is able to interact firmly with the Importin-α he lipopeptide is able to condense DNA molecules efficiently, facilitate transgene expression, expedite nuclear uptake process, and hence has the characteristics of a potential transfection agent. PMID: 33019928 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 3, 2020 Category: Genetics & Stem Cells Authors: Tarwadi T, Jazayeri JA, Pambudi S, Arbianto AD, Rachmawati H, Kartasasmita RE, Asyarie S Tags: Curr Gene Ther Source Type: research

Genetic engineering of AAV capsid gene for gene therapy application.
Abstract Adeno-associated virus (AAV) is a promising vector for in vivo gene therapy because of its excellent safety profile and ability to mediate stable gene expression in human subjects. However, there are still numerous challenges that need to be resolved before this gene delivery vehicle is used in clinical applications, such as the inability of AAV to effectively target specific tissues, preexisting neutralizing antibodies in human populations, and a limited AAV packaging capacity. Over the past two decades, much genetic modification work has been performed with the AAV capsid gene, resulting in a large numb...
Source: Current Gene Therapy - September 28, 2020 Category: Genetics & Stem Cells Authors: Liu Y, Zhang X, Yang L Tags: Curr Gene Ther Source Type: research

Stem Cell Transplantation Improves Ovarian Function through Paracrine Mechanisms.
Abstract The ovary serves as the source of oocytes for the maintenance of female fertility and is a major supplier of sex hormones for endocrine homeostasis. Various circumstances such as genetic defects, autoimmune disorders, natural aging and environmental toxins can damage the ovaries leading to diminished ovarian function, and there are currently no effective treatment regimens for such loss of function. Stem cells show promising for treating many refractory diseases, and stem cell transplantation has been shown to be effective and safe as a new therapeutic method for ovarian injuries and ovarian aging in both...
Source: Current Gene Therapy - September 27, 2020 Category: Genetics & Stem Cells Authors: Jiao W, Mi X, Qin Y, Zhao S Tags: Curr Gene Ther Source Type: research

Gene Therapy (Part II).
PMID: 32951571 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - September 23, 2020 Category: Genetics & Stem Cells Authors: He ZY, Jia XB Tags: Curr Gene Ther Source Type: research

Applications of Recombinant Adenovirus-p53 Gene Therapy for Cancers in the Clinic in China.
Abstract Suppression of TP53 function is nearly ubiquitous in human cancers, and a significant fraction of cancers have mutations in the TP53 gene itself. Therefore, the wild-type TP53 gene has become an important target gene for transformation research of cancer gene therapy. In 2003, the first anti-tumor gene therapy drug rAd-p53 (recombinant human p53 adenovirus), trade name Gendicine™, was approved by the China Food and Drug Administration (CFDA) for treatment of head and neck squamous cell carcinoma (HNSCC) in combination with radiotherapy. The recombinant human TP53 gene is delivered into cancer cells ...
Source: Current Gene Therapy - September 23, 2020 Category: Genetics & Stem Cells Authors: Xia Y, Li X, Sun W Tags: Curr Gene Ther Source Type: research

γ-Aminobutyric Acid Promotes Osteogenic Differentiation of Mesenchymal Stem Cells by Inducing TNFAIP3.
CONCLUSION: Our results suggested that GABA treatment positively regulated osteogenic differentiation by upregulating TNFAIP3, while no obvious effect on osteoclastic differentiation was detected. Therefore, our results provide a potential gene therapy for the treatment of osteoporosis and low bone mineral density. PMID: 32951573 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - September 23, 2020 Category: Genetics & Stem Cells Authors: Li H, Wu Y, Huang N, Zhao Q, Yuan Q, Shao B Tags: Curr Gene Ther Source Type: research

Exploring the role of gene therapy for neurological disorders.
Abstract Gene therapy is one the frontier fields of medical breakthroughs that poses as an effective solution to previously incurable diseases. The delivery of the corrective genetic material or a therapeutic gene into the cell restores the missing gene function and cures a plethora of diseases, incurable by the conventional medical approaches. This discovery holds the potential to treat many neurodegenerative disorders such as muscular atrophy, multiple sclerosis, Parkinson's disease (PD) and Alzheimer's disease (AD) among others. Gene therapy proves as a humane, cost effective alternative to the exhaustive often...
Source: Current Gene Therapy - September 16, 2020 Category: Genetics & Stem Cells Authors: Puranik N, Yadav D, Chauhan PS, Kwak M, Jin JO Tags: Curr Gene Ther Source Type: research

miRNA-146a Improves Immunomodulatory Effects of MSC-derived Exosomes in Rheumatoid Arthritis.
CONCLUSION: Based on the findings here, Exosomes appears to promote the direct intracellular transfer of miRNAs between cells and to represent a possible therapeutic strategy for RA.the manipulation of MSC-derived exosomes with anti-inflammatory miRNA may increase Treg cell populations and anti-inflammatory cytokines. Ultimately, such modulation may promote the recovery of appropriate T-cell responses in inflammatory situations such as RA. PMID: 32938348 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - September 15, 2020 Category: Genetics & Stem Cells Authors: Tavasolian F, Hosseini AZ, Soudi S, Naderi M Tags: Curr Gene Ther Source Type: research

Recombinant human p53 adenovirus injection (rAd-p53) combined with chemotherapy for 4 cases of high-grade serous ovarian cancer.
Abstract High-grade serous ovarian carcinoma (HGSOC) is one of the most common ovarian epithelial carcinomas. It is highly invasive, easily recurs after systemic treatment, and has a poor prognosis. Despite many new chemotherapeutic drugs and trials of combinations of different regimens that have been used in treatment attempts, there has been no meaningful progress in the treatment of HGSOC. With the development of gene sequencing technology, gene therapy has become a new direction for tumors treatment. It's reported that the P53 has a very high mutation rate in HGSOC, which provides a theoretical basis for the a...
Source: Current Gene Therapy - August 24, 2020 Category: Genetics & Stem Cells Authors: Qu H, Xia Y, Li X Tags: Curr Gene Ther Source Type: research

The emerging role of stem cells in regenerative dentistry.
Bordignon P Abstract Progress of modern dentistry is accelerating at a spectacular speed in the scientific, technological and clinical are-as. Practical examples are the advancement in the digital field, which has guaranteed an average level of prosthetic practices for all patients, as well as other scientific developments, including research on stem cell biology. Given their plasticity, de-fined as the ability to differentiate into specific cell lineages with capacity of almost unlimited self-renewal and release of trophic/immunomodulatory factors, stem cells have gained significant scientific and commercial inte...
Source: Current Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Capparè P, Tetè G, Sberna MT, Panina-Bordignon P Tags: Curr Gene Ther Source Type: research

Gene Therapy in the Management of Parkinson's Disease: Potential of GDNF as a Promising Therapeutic Strategy.
Abstract The limitations of conventional treatment therapies in Parkinson's disorder, a common neurodegenerative disorder, lead to the development of an alternative gene therapy approach. Multiple treatment options targeting dopaminergic neuronal regeneration, production of enzymes linked with dopamine synthesis, subthalamic nucleus neurons, regulation of astrocytes and microglial cells and potentiating neurotrophic factors, were established. Viral vector-based dopamine delivery, prodrug approaches, fetal ventral mesencephalon tissue transplantation and dopamine synthesizing enzyme encoding gene delivery are signi...
Source: Current Gene Therapy - August 16, 2020 Category: Genetics & Stem Cells Authors: Behl T, Kaur I, Kumar A, Mehta V, Zengin G, Arora S Tags: Curr Gene Ther Source Type: research

Targeted delivery for neurodegenerative disorders using gene therapy vectors: Gen next therapeutic goals.
Abstract The technique of gene therapy, ever since its advent nearly fifty years ago, has been utilized by scientists as a po-tential treatment option for various disorders. This review discusses some of the major neurodegenerative diseases (NDDs) like Alzheimer's disease (AD), Parkinson's disease (PD), Motor neuron diseases (MND), Spinal muscular atrophy (SMA), Huntington's disease (HD), Multiple sclerosis (MS) etc. and their underlying genetic mechanisms along with therole that gene therapy can play in combating them. The pathogenesis and the molecular mechanisms specifying the altered gene ex-pression of each o...
Source: Current Gene Therapy - August 16, 2020 Category: Genetics & Stem Cells Authors: Singh M, Singh SP, Yadav D, Agarwal M, Agarwal S, Agarwal V, Swargiary G, Srivastava S, Tyagi S, Kaur R, Mani S Tags: Curr Gene Ther Source Type: research

Growth Retardation of Poorly Transfectable Tumor by Multiple Injections of plasmids Encoding PE40 Based Targeted Toxin Complexed with Poly-ethylenimine.
CONCLUSION: Despite this, three intratumoral injections of a plasmid- polyethylenimine complex resulted in a substantial growth retardation of poorly transfectable D2F2/E2 tumor in mice. There were no significant differences in anti-tumor prop-erties between DNA constructs with telomerase or CAG promoters in vivo. PMID: 32807050 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - August 15, 2020 Category: Genetics & Stem Cells Authors: Khodarovich Y, Rakhmaninova D, Kagarlitskiy G, Baryshnikova A, Deyev S Tags: Curr Gene Ther Source Type: research

Gene Therapy for Hemophilia A: Where We Stand.
Abstract Hemophilia A (HA) is a hereditary hemorrhagic disease caused by a deficiency of coagulation factor VIII (FVIII) in blood plasma. Patients with HA usually suffer from spontaneous and recurrent bleeding in joints and muscles, or even intracerebral hemorrhage, which might lead to disability or death. Although the disease is currently manageable via delivery of plasma-derived or recombinant FVIII, this approach is costly, and neutralizing antibodies may be generated in a large portion of patients, which render the regimens ineffective and inaccessible. Given the monogenic nature of HA and that a slight increa...
Source: Current Gene Therapy - August 4, 2020 Category: Genetics & Stem Cells Authors: Zhou M, Hu Z, Zhang C, Wu L, Li Z, Liang D Tags: Curr Gene Ther Source Type: research

NF1, Neurofibromin and Gene Therapy: Prospects of Next Generation Therapy.
Abstract Neurofibromatosis type 1 [NF1] is an autosomal dominant genetic disorder affecting multiple organs. NF1 is well known for its various clinical manifestations including café-au-late macules, Lisch nodules, bone deformity and neurofibromas. However, there is no effective therapy for NF1. Current therapies are aimed at alleviating NF1 clinical symptoms but not curing the disease. By altering pathogenic genes, gene therapy regulates cell activities at the nucleotide level. In this review, we described the structure and functions of neurofibromin domains, including GAP-related domain [GRD], cysteine-ser...
Source: Current Gene Therapy - August 4, 2020 Category: Genetics & Stem Cells Authors: Cui XW, Ren JY, Gu YH, Li QF, Wang ZC Tags: Curr Gene Ther Source Type: research

Gene therapy, a novel therapeutic tool for neurological disorders: Current progress, challenges and future prospective.
Abstract Neurological disorders are one of the major threat for health care system as it puts enormous socioeconomic burden. All aged population are susceptible to one or other neurological problems with symptoms of neuroinflammation, neurodegeneration and cognitive dysfunction. At present available pharmacotherapeutics are insufficient to treat these diseased conditions and in most cases they provide only palliative effect. It was also found that the molecular etiology of neurological disorders are directly linked with the alteration in genetic makeup which can be inherited or triggered by the injury, environment...
Source: Current Gene Therapy - July 14, 2020 Category: Genetics & Stem Cells Authors: Iqubal A, Iqubal MK, Khan A, Ali J, Baboota S, Haque SE Tags: Curr Gene Ther Source Type: research

Omics Data and Artificial Intelligence: New Challenges for Gene Therapy.
PMID: 32603274 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - July 2, 2020 Category: Genetics & Stem Cells Authors: Cheng L Tags: Curr Gene Ther Source Type: research

Gene Therapy (Part I).
PMID: 32603275 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - July 2, 2020 Category: Genetics & Stem Cells Authors: Jia XB, He ZY Tags: Curr Gene Ther Source Type: research

Systemic Therapy for Hepatocellular Carcinoma: Advances and Hopes.
Abstract The majority of patients with hepatocellular carcinoma (HCC) are diagnosed at advanced stage that can only benefit from systemic treatments. Although HCC is highly treatment resistant, significant achievements have been made in the molecular targeted therapy and immunotherapy of HCC. In addition to regorafenib, cabozantinib and ramucirumab were approved for the second-line targeted treatment by FDA after disease progression on sorafenib. Nivolumab failed to demonstrate remarkable benefit in overall survival (OS) as a first-line therapy, while pembrolizumab did not achieve pre-specified statistical signifi...
Source: Current Gene Therapy - June 26, 2020 Category: Genetics & Stem Cells Authors: Zhang CH, Li M, Lin YP, Gao Q Tags: Curr Gene Ther Source Type: research

CD24, A Review of its Role in Tumor Diagnosis, Progression and Therapy.
Abstract CD24, is a mucin-like GPI-anchored molecules. By immunohistochemistry, it is widely detected in many solid tumors such as breast cancers, genital system cancers, digestive system cancers, neural system cancers and so on. The functional roles of CD24 are either fulfilled by combination with ligands or participate in signal transduction, which mediate the initiation and progression of neoplasms. However, the character of CD24 remains to be intriguing because there are still opposite voices about the impact of CD24 on tumors. In preclinical studies, CD24 target therapies including monoclonal antibodies, targ...
Source: Current Gene Therapy - June 22, 2020 Category: Genetics & Stem Cells Authors: Ni YH, Zhao X, Wang W Tags: Curr Gene Ther Source Type: research

Dissecting the therapeutic relevance of gene therapy in NeuroAIDS, an evolving epidemic.
Abstract NeuroAIDS, a disease incorporating both infectious and neurodegenerative pathways, is still a formidable challenge for the researchers to deal with. The primary concern for the treatment of neuroAIDS still remains the inaccessibility of the viral reservoir, making it indispensable for novel techniques to be continuously innovated. Since brain serves as a reservoir for viral replication, it is pragmatic and a prerequisite to overcome the related barriers in order to improve the drug delivery to the brain. The current treatment ideology is based on the combinatorial approach of mocktail of anti-retroviral d...
Source: Current Gene Therapy - June 14, 2020 Category: Genetics & Stem Cells Authors: Nabi B, Rehman S, Pottoo FH, Baboota S, Ali J Tags: Curr Gene Ther Source Type: research

Mesenchymal Stem Cells: A New Generation of Therapeutic Agent Vehicles in Gene Therapy.
Abstract In recent years, mesenchymal stem cells (MSCs) as a new tool for therapeutic gene delivery in clinics have attracted much attention. Its advantages contain longer lifespan, better isolation, and higher transfection efficiency and proliferation rate. MSCs are the preferred approach for cell-based therapies because of their in vitro self-renewal capacity, migrating especially to tumor tissues, as well as anti-inflammatory and immunomodulatory properties. Therefore, they have considerable efficiency in genetic engineering for future clinical applications in cancer gene therapy and other diseases. For improvi...
Source: Current Gene Therapy - June 6, 2020 Category: Genetics & Stem Cells Authors: Gharbavi M, Sharafi A, Ghanbarzadeh S Tags: Curr Gene Ther Source Type: research

Nanoparticle based gene therapy approach: A pioneering rebellion in the management of psychiatric disorders.
Abstract The neuropsychiatric illnesses have been enigmatic, with no effective treatment to date. The complexity and heterogeneity of psychiatric disorders are daunting for the development of novel treatment modalities. The conventional treatment approaches are less effective and are associated with several side effects, thus creating the need for the development of more innovative strategies. Since psychiatric disorders are known to exhibit genetic linkage, gene therapy has attracted the attention of the researchers worldwide. The delivery of nucleic acids is a complex process requiring the transport of genetic m...
Source: Current Gene Therapy - June 6, 2020 Category: Genetics & Stem Cells Authors: Rehman S, Nabi B, Pottoo FH, Baboota S, Ali J Tags: Curr Gene Ther Source Type: research

Integrated Analysis of mRNA-seq and miRNA-seq to Identify c-MYC, YAP1 and miR-3960 as Major Players in the Anticancer Effects of Caffeic Acid Phenethyl ester in Human Small Cell Lung Cancer Cell Line.
CONCLUSION: We demonstrated the anti-cancer effect of CAPE in human SCLC cells and studied the mechanism by acquiring a comprehensive transcriptome profile of CAPE-treated cells. PMID: 32445454 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 22, 2020 Category: Genetics & Stem Cells Authors: Mo F, Luo Y, Fan D, Zeng H, Zhao Y, Luo M, Liu X, Ma X Tags: Curr Gene Ther Source Type: research

Transfection of TGF- β shRNA by using ultrasound-targeted microbubble destruction to inhibit the early adhesion repair of rats wounded Achilles tendon in vitro and in vivo.
Transfection of TGF-β shRNA by using ultrasound-targeted microbubble destruction to inhibit the early adhesion repair of rats wounded Achilles tendon in vitro and in vivo. Curr Gene Ther. 2020 May 16;: Authors: Huang S, Xi X, Qiu L, Wang L, Zhu B, Guo R, Tang X Abstract BACKGROUND: Tendon injury is a major orthopedic disorder. Ultrasound-targeted microbubble destruction (UTMD) provides a promising method for gene transfection, which can be used for the treatment of injured tendons. OBJECTIVE: The purpose of this study was to investigate the optimal transforming growth factor beta (TGF-β...
Source: Current Gene Therapy - May 15, 2020 Category: Genetics & Stem Cells Authors: Huang S, Xi X, Qiu L, Wang L, Zhu B, Guo R, Tang X Tags: Curr Gene Ther Source Type: research

Artificial RNA editing with ADAR for gene therapy.
Abstract Editing mutated genes is a potential way for the treatment of the genetic diseases. G-to-A mutations are common in mammals, and can be treated by adenosine-to-inosine (A-to-I) editing, a type of substitutional RNA editing. Molecular mechanism of A-to-I editing involves the hydrolytic deamination of an adenosine to an inosine base; this reaction is mediated by RNA-specific deaminases, adenosine deaminases acting on RNA (ADARs), family protein. Here, we review recent findings regarding the application of ADARs to restoring the genetic code along with different approaches involved in the process of artificia...
Source: Current Gene Therapy - May 15, 2020 Category: Genetics & Stem Cells Authors: Bhakta S, Tsukahara T Tags: Curr Gene Ther Source Type: research

Potential Prognostic Predictors and Molecular Targets for Skin Melanoma Screened by Weighted Gene Co-expression Network Analysis.
Abstract BACKGROUND: Among skin cancers, malignant skin melanoma is the leading cause of death. Identification of gene markers of malignant skin melanoma associated with survival may provide new clues for prognosis prediction and treatment. OBJECTIVES: This research aimed to screen out potential prognostic predictors and molecular targets for malignant skin melanoma. METHODS: Information regarding gene expression in skin melanoma and patients' clinical traits was obtained from the Gene Expression Omnibus database. Weighted gene co-expression network analysis (WGCNA) was applied to build co-expression mod...
Source: Current Gene Therapy - May 15, 2020 Category: Genetics & Stem Cells Authors: Chen S, Liu Z, Li M, Huang Y, Wang M, Zeng W, Wei W, Zhang C, Gong Y, Guo L Tags: Curr Gene Ther Source Type: research

Combined Analysis of Clinical Data on HGF Gene Therapy to Treat Critical Limb Ischemia in Japan.
CONCLUSIONS: The findings indicated that intramuscular injection of naked HGF plasmid tended to improve the resting pain and significantly decreased the size of the ischemic ulcer in the patients with CLI who did not have any alternative therapy such as endovascular treatment (EVT) or bypass graft surgery. An HGF gene therapy product, CollategeneTM , was recently launched with conditional and time-limited approval in Japan to treat ischemic ulcer in patients with CLI. Further clinical trials would provide new therapeutic options for patients with CLI. PMID: 32416690 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 15, 2020 Category: Genetics & Stem Cells Authors: Morishita R, Shimamura M, Takeya Y, Nakagami H, Chujo M, Ishihama T, Yamada E, Rakugi H Tags: Curr Gene Ther Source Type: research

Gene Therapy for Neuroprotection and Neurorestoration (Part I).
PMID: 32375609 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 9, 2020 Category: Genetics & Stem Cells Authors: Ashraf GM, Uddin MS Tags: Curr Gene Ther Source Type: research

Evaluation of BMP-2 minicircle DNA for enhanced bone engineering and regeneration.
CONCLUSION: We have designed a highly bioactive BMP-2 minicircle plasmid with the potential to fulfil clinical requirements for non-viral gene therapy in the field of bone regeneration. PMID: 32338217 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - April 26, 2020 Category: Genetics & Stem Cells Authors: Zimmermann A, Hercher D, Regner B, Frischer A, Sperger S, Redl H, Hacobian A Tags: Curr Gene Ther Source Type: research

Prader-Willi syndrome: molecular mechanism and epigenetic therapy.
Abstract Prader-Willi syndrome (PWS) is an imprinted neurodevelopmental disease characterized by cognitive impairments, developmental delay, hyperphagia, obesity, and sleep abnormalities. It's caused by lack of expression of the paternally active genes in the PWS imprinting center on chromosome 15 (15q11.2-q13). Owing to the imprinted gene regulation, the same genes in the maternal chromosome 15q11-q13 are intact in structure but repressed at the transcriptional level because of the epigenetic mechanism. The specific molecular defect underlying PWS provides an opportunity to explore epigenetic therapy to reactivat...
Source: Current Gene Therapy - April 22, 2020 Category: Genetics & Stem Cells Authors: Mian-Ling Z, Yun-Qi C, Chao-Chun Z Tags: Curr Gene Ther Source Type: research

Promising gene therapy using an adenovirus vector carrying REIC/Dkk-3 gene for the treatment of biliary cancer.
CONCLUSIONS: Ad-SGE-REIC induced apoptosis and inhibited tumor growth in biliary cancer cells. REIC/Dkk-3 gene therapy using Ad-SGE-REIC is an attractive therapeutic tool for biliary cancer. PMID: 32148193 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - March 8, 2020 Category: Genetics & Stem Cells Authors: Tanaka E, Uchida D, Shiraha H, Kato H, Oyama A, Iwamuro M, Watanabe M, Kumon H, Okada H Tags: Curr Gene Ther Source Type: research

Gene Therapy Approaches in an Autoimmune Demyelinating Disease: Multiple Sclerosis.
Abstract Multiple sclerosis (MS) is the most common autoimmune demyelinating disease of the central nervous system (CNS). It is a multifactorial disease which develops in an immune-mediated way under the influences of both genetic and environmental factors. Demyelination is observed in the brain and spinal cord leading to neuro-axonal damage in patients with MS. Due to the infiltration of different immune cells such as T-cells, B-cells, monocytes and macrophages, focal lesions are observed in MS. Currently available medications treating MS are mainly based on two strategies; i) to ease specific symptoms or ii) to ...
Source: Current Gene Therapy - March 4, 2020 Category: Genetics & Stem Cells Authors: Islam MA, Kundu S, Hassan R Tags: Curr Gene Ther Source Type: research

Limb Girdle Muscular Dystrophy and Therapy: Current progress in Cell and Gene-based approaches.
Abstract The limb-girdle muscular dystrophies (LGMD) are genetically heterogeneous disorders, responsible for muscle wasting and severe form of dystrophies. Despite the critical developments in the insight and information of pathomechanisms of limb-girdle muscular dystrophy, any definitive treatments do not exist, and current strategies are only based on the improvement of the signs of disorder and to enhance the life quality without resolving an underlying cause. There is a crucial relationship between pharmacological therapy and different consequences; therefore, other treatment strategies will be required. New ...
Source: Current Gene Therapy - February 16, 2020 Category: Genetics & Stem Cells Authors: Taheri F, Taghizadeh E, Pour MJR, Rostami D, Renani PG, Rastgar-Moghadam A, Hayat SMG Tags: Curr Gene Ther Source Type: research

Characterization of human epidermal melanocytes transfected with two combinations of transcription factors.
CONCLUSION: Human epidermal melanocytes do not require ectopic SOX-2 expression for conversion into iPSCs, and may serve as an alternative source for deriving patient-specific iPSCs with fewer genetic elements. PMID: 32072883 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - February 9, 2020 Category: Genetics & Stem Cells Authors: Cheng S, Li D, Zhang RZ, Zhu J, Wang L, Liu Q, Chen RH, Liu XM Tags: Curr Gene Ther Source Type: research

Effect of Proteasome Inhibitors on the AAV-mediated Transduction Efficiency in Retinal Bipolar Cells.
CONCLUSION: Doxorubicin could enhance the AAV transduction efficiency in retinal bipolar cells in vivo. The potential long-term cytotoxicity caused by doxorubicin to retinal neurons could be partially mitigated by dexrazoxane. The coapplication of doxorubicin and dexrazoxane may serve as a potential adjuvant regimen for improving AAV transduction efficiency in retinal bipolar cells. PMID: 32072884 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - February 9, 2020 Category: Genetics & Stem Cells Authors: Cui S, Ganjawala TH, Abrams GW, Pan ZH Tags: Curr Gene Ther Source Type: research

Gene therapy for Angelman Syndrome: Contemporary approaches and future endeavors.
CONCLUSION: Understanding UBE3A imprinting unravels the path to an etiologic treatment of AS. Gene therapy models tested on mice appeared less effective than anticipated pointing out that activation of paternal UBE3A cannot counteract the existing CNS defects. On the other hand, targeting abnormal downstream cell signaling pathways has provided promising rescue effects. Perhaps, combined reinstatement of paternal UBE3A expression with abnormal signaling pathways-oriented treatment is expected to provide better therapeutic effects. However, AS gene therapy remains debatable in pharmacoeconomics and ethics context. PMID...
Source: Current Gene Therapy - January 6, 2020 Category: Genetics & Stem Cells Authors: Tsagkaris C, Papakosta V, Miranda AV, Zacharopoulou L, Danilchenko V, Matiashova L, Dhar A Tags: Curr Gene Ther Source Type: research

Computational and Biological Methods for Gene Therapy.
PMID: 31762421 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 27, 2019 Category: Genetics & Stem Cells Authors: Cheng L Tags: Curr Gene Ther Source Type: research