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 7, 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

Effect of Tumor Suppressor MiR-34a Loaded on ZSM-5 Nanozeolite in Hepatocellular Carcinoma: In Vitro and In Vivo Approach.
CONCLUSION: Our results suggested that miR-34a is a powerful candidate in HCC treatment, and that AEG-1, and SOX-9 are novel oncotargets of miR-34a in HCC. Results also demonstrated that our nano-formulation may serve as a candidate approach for miR-34a restoration for HCC therapy, and generally for safe gene delivery. PMID: 31701846 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 7, 2019 Category: Genetics & Stem Cells Authors: Salah Z, Abd El Azeem EM, Youssef HF, Gamal-Eldeen AM, Farrag AR, El-Meliegy E, Soliman B, Elhefnawi M Tags: Curr Gene Ther Source Type: research

Non-coding RNA associated Competitive Endogenous RNA Regulatory Network: Novel Therapeutic Approach in Liver Fibrosis.
Abstract Liver fibrosis or scarring is the most common pathological feature caused by chronic liver injury, and is widely considered one of the primary causes of morbidity and mortality. It is primarily characterised by hepatic stellate cells (HSC) activation and excessive extracellular matrix (ECM) protein deposition. Overwhelming evidence suggests that the dysregulation of several noncoding RNAs (ncRNAs) mainly long non-coding RNAs (lncRNAs), microRNAs (miRNAs) and circular RNAs (circRNAs) contributes to activation of HSC and progression of liver fibrosis. These ncRNA not bind only to their target genes for the ...
Source: Current Gene Therapy - November 6, 2019 Category: Genetics & Stem Cells Authors: Riaz F, Li D Tags: Curr Gene Ther Source Type: research

Suicide Gene Therapy Against Malignant Gliomas by the Local Delivery of Genetically Engineered Umbilical Cord Mesenchymal Stem Cells as Cellular Vehicles.
CONCLUSION: This research suggested that the MSCTK/GCV system exerts a strong bystander effect on GBM tumor cells, and this system may be a promising assistant method for GBM postoperative therapy. PMID: 31657679 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 27, 2019 Category: Genetics & Stem Cells Authors: Wei D, Hou J, Zheng K, Jin X, Xie Q, Cheng L, Sun X Tags: Curr Gene Ther Source Type: research

The promise of miRNA replacement therapy for Hepatocellular carcinoma.
CONCLUSION: tumor suppressor miRNAs can act as novel therapeutics for HCC and more studies should be directed toward these promising therapeutics. PMID: 31657677 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 22, 2019 Category: Genetics & Stem Cells Authors: Elhefnawi M, Salah Z, Soliman B Tags: Curr Gene Ther Source Type: research

Nano-enhanced optical gene delivery to retinal degenerated mice.
CONCLUSION: The successful delivery and expression of MCO in targeted retina after in-vivo NOD in the mice models of retinal degeneration opens new vista for re-photosensitizing retina with geographic atrophies such as in dry age-related macular degeneration. PMID: 31625475 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 17, 2019 Category: Genetics & Stem Cells Authors: Batabyal S, Gajjeraman S, Bhattacharya S, Wright W, Mohanty S Tags: Curr Gene Ther Source Type: research

Current RNA-based Therapeutics in Clinical Trials.
Abstract Long-term research on various types of RNAs has led to further understanding of diverse mechanisms, which eventually resulted in the rapid development of RNA-based therapeutics as powerful tools in clinical disease treatment. Some of the developing RNA drugs obey the antisense mechanisms including antisense oligonucleotides, small interfering RNAs, microRNAs, small activating RNAs, and ribozymes. These types of RNAs could be utilized to inhibit/activate gene expression or change splicing to provide functional proteins. In the meantime, some others based on different mechanisms like modified messenger RNAs...
Source: Current Gene Therapy - October 2, 2019 Category: Genetics & Stem Cells Authors: Zhou LY, Qin Z, Zhu YH, He ZY, Xu T Tags: Curr Gene Ther Source Type: research

Effects of Huangkui Capsule on the Expression of SPARC in the Kidney Tissue of a Rat Model with Diabetic Nephropathy.
This study was performed to investigate the effects of HKC on DN in SD rats model and its molecular mechanism. Our results showed that, the rats treated with HKC had an improved general state and reduced creatinine, blood urea nitrogen and 24-hour urinary protein levels. The deterioration of renal function was delayed due to treatment with HKC. We utilized HE staining to observe that HKC can improve histopathological findings in the kidney tissues of DN rats, including kidney fibrosis. Results of western blot and qRT-PCR showed that HKC can inhibit the expressions of SPARC in the rat model of DN. PMID: 31553295 [PubMe...
Source: Current Gene Therapy - September 25, 2019 Category: Genetics & Stem Cells Authors: Yang X, Luo M, Jiang Q, Wang Y Tags: Curr Gene Ther Source Type: research

A Mendelian Randomization Study of Infant Length and Type 2 Diabetes Mellitus Risk.
CONCLUSIONS: We validated that the shorter IL contributes no additional risk to T2DM. The sensitivity analysis and the MR-Egger regression analysis also provided adequate evidence the above result was not due to any heterogeneity or pleiotropic effect of IVs. PMID: 31553296 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - September 25, 2019 Category: Genetics & Stem Cells Authors: Zhuang H, Zhang Y, Yang S, Cheng L, Liu SL Tags: Curr Gene Ther Source Type: research

Identifying Alzheimer's disease-related miRNA Based on semi-Clustering.
Abstract miRNA is not only found in brain tissue, but also ins cerebrospinal fluid as well as in plasma and serum. More and more scholars are trying to use it as a specific biomarker for Alzheimer's Disease (AD). Multiple studies have indicated that miRNAs are associated with poor axonal growth and loss of synaptic structures, both of which are early events in AD. The overall loss of miRNA may be associated with aging, increasing the incidence of AD, and may also be involved in the disease through some specific molecular mechanisms. We used genes as a bridge to connect AD and miRNAs. Firstly, protein-protein inter...
Source: Current Gene Therapy - September 24, 2019 Category: Genetics & Stem Cells Authors: Zhao T, Wang D, Hu Y, Zhang N, Zang T, Wang Y Tags: Curr Gene Ther Source Type: research

Hemophilia Gene Therapy: From Bench to Bed Side.
Abstract Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete cure for hemophilia. This review will address the latest gene therapy research in hemophilia A and B including gene therapy tools, genetic strategies and animal models, and summarizes results of recent clinical trials. We will also discuss potential solutions to the current barriers in gene therapy for hemophilia. PMID: 31549954 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - September 24, 2019 Category: Genetics & Stem Cells Authors: Guo XL, Chung TH, Qin Y, Zheng J, Zheng H, Sheng L, Wynn T, Chang LJ Tags: Curr Gene Ther Source Type: research

A Recurrent Rare SOX9 Variant (M469V) is Associated with Congenital Vertebral Malformations.
CONCLUSION: This SOX9 mutation (p.M469V) may contribute to CVM without other systematic deformity, which provided important implications and better understanding of phenotypic variability in SOX9-related skeletal deformities. PMID: 31549955 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - September 24, 2019 Category: Genetics & Stem Cells Authors: Wu N, Wang L, Hu J, Zhao S, Liu B, Li Y, Du H, Zhang Y, Li X, Yan Z, Wang S, Wang Y, Zhang J, Wu Z, Qiu G Tags: Curr Gene Ther Source Type: research

Prediction of Disease Comorbidity Using HeteSim Scores based on Multiple Heterogeneous Networks.
Abstract Accumulating experimental studies have indicated that disease comorbidity causes additional pain to patients and leads to the failure of standard treatments compared to patients who have a single disease. Therefore, the accurate prediction of potential comorbidity is essential to design more efficient treatment strategies. However, only a few disease comorbidities have been discovered in the clinic. In this work, we propose PCHS, an effective computational method for predicting comorbidity using HeteSim scores. PCHS utilizes the HeteSim measure to calculate the relatedness score for different disease pair...
Source: Current Gene Therapy - September 17, 2019 Category: Genetics & Stem Cells Authors: Chen X, Shi W, Deng L Tags: Curr Gene Ther Source Type: research

Interaction of Cell and Gene Therapy with the Immune System.
Abstract PMID: 31470774 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - September 2, 2019 Category: Genetics & Stem Cells Authors: Yang C Tags: Curr Gene Ther Source Type: research

Biodegradable Polyester of Poly (Ethylene glycol)-sebacic Acid as a Backbone for β-Cyclodextrin-polyrotaxane: A Promising Gene Silencing Vector.
CONCLUSION: The combinatory effect of various factors such as biodegradability, favourable complexation ability, near zero zeta potentials, good cytotoxicity properties of poly (ethylene glycol)-sebacic acid based β-Cyclodextrin-polyrotaxane makes it a potential gene delivery vector for therapeutic applications. PMID: 31393245 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - August 7, 2019 Category: Genetics & Stem Cells Authors: Ghodke S, Mahajan P, Gupta K, Ver Avadhani C, Dandekar P, Jain R Tags: Curr Gene Ther Source Type: research

The Therapeutic Potential and Role of miRNA, lncRNA, and circRNA in Osteoarthritis.
CONCLUSION: In this paper, we briefly introduced the role of miRNA, lncRNA, and circRNA in the occurrence and development of OA and as a new target for gene therapy. PMID: 31333128 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - July 15, 2019 Category: Genetics & Stem Cells Authors: Wu Y, Shen B, Zeng Y Tags: Curr Gene Ther Source Type: research

Functional Immunoregulation by Heme Oxygenase 1 in Juvenile Autoimmune Diseases.
Abstract Autoimmune disease is an inflammatory disease in which the human body's autoimmune system attacks normal cells, resulting in decreased immune function and abnormal immune function, which eventually leads to tissue damage or organ dysfunction. It is estimated that 5-8% of the world's population is at risk of autoimmune diseases. In the field of medicine, especially in pediatrics, the research data of autoimmune diseases are still insufficient. Some prevalent juvenile autoimmune diseases, such as schoenlein-henoch purpura (HSP), systemic juvenile idiopathic arthritis (SJIA), mucocutaneous lymph node syndrom...
Source: Current Gene Therapy - July 9, 2019 Category: Genetics & Stem Cells Authors: Zhang X, Shi S, Shen J, Zhao M, He Q Tags: Curr Gene Ther Source Type: research

The therapeutic potential of small activating RNAs for colorectal carcinoma.
Abstract Small double strand RNA has been recognized as master regulator of gene expression. In contrast to the evolutionary conserved RNA interference machinery, which degrades or inhibits the translation of target mRNAs, the RNA activation is a gene regulation mechanism that facilitates the exact opposite effect. It is triggered by double strand RNA termed small activating RNA and acts through a similar mechanism as RNAi, while activates the specific gene on a target dependent manner. Recently, small RNA molecule mediated expression regulation of specific genes has been extensively studied in the cancer research...
Source: Current Gene Therapy - July 8, 2019 Category: Genetics & Stem Cells Authors: Zheng B, Mai QY, Jiang JX, Zhou QQ Tags: Curr Gene Ther Source Type: research

Understanding Gene Therapy in Acute Respiratory Distress Syndrome.
Abstract Acute respiratory distress syndrome (ARDS) and its complications remain life-threatening conditions for critically ill patients. The present therapeutic strategies such as prone positioning ventilation strategies, nitric oxide inhalation, restrictive intravenous fluid management, and extra corporeal membrane oxygenation (ECMO) do not contribute much to improving the mortality of ARDS. The advanced understanding of the pathophysiology of acute respiratory distress syndrome suggests that gene-based therapy may be an innovative method for this disease. Many scientists have made beneficial attempts to regulat...
Source: Current Gene Therapy - July 2, 2019 Category: Genetics & Stem Cells Authors: Zhang XP, Zhang WT, Qiu Y, Ju MJ, Tu GW, Luo Z Tags: Curr Gene Ther Source Type: research

The Synergy between CRISPR and Chemical Engineering.
Abstract Gene therapy and transgenic research have advanced quickly in recent years due to the development of CRISPR technology. The rapid development of CRISPR technology has been largely benefited by chemical engineering. Firstly, chemical or synthetic substance enables spatiotemporal and conditional control of Cas9 or dCas9 activities. It prevents leaky expression of CRISPR components, as well as minimizes toxicity and off-target effects. Multi-input logic operations and complex genetic circuits can also be implemented via multiplexed and orthogonal regulation of target genes. Secondly, rational chemical modifi...
Source: Current Gene Therapy - June 30, 2019 Category: Genetics & Stem Cells Authors: Lau CH, Tin C Tags: Curr Gene Ther Source Type: research

The Relationship between Ferroptosis and Tumors - A Novel Landscape for Therapeutic Approach.
Abstract Ferroptosis is a newly discovered form of iron-dependent oxidative cell death characterized by accumulating lipid-based reactive oxygen species (ROS) to a lethal level. It is distinct from other forms of cell death including apoptosis, necrosis and auto-phage in the aspects of morphology, biochemistry and genetics. Ferroptosis could be triggered by system xc- inhibitors or GHx4 inhibitors, as well as drugs such as sorafenib, SAS, and ART. Recently, studies have demonstrated that ferroptosis was critical in regulating tumor growth, such as hepatocellular carcinoma(HCC), renal cell carcinoma(RCC), non-small...
Source: Current Gene Therapy - June 28, 2019 Category: Genetics & Stem Cells Authors: Xia X, Fan X, Zhao M, Zhu P Tags: Curr Gene Ther Source Type: research

HMGB1: A generalist in immunity.
Abstract High mobility group box 1 (HMGB1), a highly conserved protein, presents in almost all somatic cells and is necessary for transcription regulation during cell development. Recent studies indicate that HMGB1 is important for inflammation, tissue regeneration and cancer, especially the balance between autophagy and apoptosis. HMGB1 shows a challenge that can be used for therapeutic improvement while preventing its pathological role. Here, we pay attention to the immuno-regulatory effect of HMGB1. PMID: 31223085 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 21, 2019 Category: Genetics & Stem Cells Authors: Zhao J, Sun T, Wu S, Liu Y Tags: Curr Gene Ther Source Type: research

Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy.
CONCLUSION: In conclusion, a single systemic injection of rAAV9-coGAA ameliorates vacuolar accumulation and prevents autophagic dysregulation. PMID: 31223086 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 21, 2019 Category: Genetics & Stem Cells Authors: McCall AL, Stankov SG, Cowen G, Cloutier D, Zhang Z, Yang L, Clement N, Falk DJ, Byrne BJ Tags: Curr Gene Ther Source Type: research

Preface.
Abstract PMID: 31190638 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 14, 2019 Category: Genetics & Stem Cells Authors: Chang LJ, Lewin AS Tags: Curr Gene Ther Source Type: research

Gene Therapy of Anderson-Fabry Disease.
Abstract PMID: 31190639 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 14, 2019 Category: Genetics & Stem Cells Authors: Tuttolomondo A, Simonetta I, Pinto A Tags: Curr Gene Ther Source Type: research

Gene Therapy on the Road.
Abstract PMID: 31190640 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 14, 2019 Category: Genetics & Stem Cells Authors: Feng YM Tags: Curr Gene Ther Source Type: research

Gene Therapy of Anderson-Fabry Disease
PMID: 31154750 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - June 3, 2019 Category: Genetics & Stem Cells Authors: Tuttolomondo A, Simonetta I, Pinto A Tags: Curr Gene Ther Source Type: research

Preface
PMID: 31071761 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 10, 2019 Category: Genetics & Stem Cells Authors: Chang LJ, Lewin AS Tags: Curr Gene Ther Source Type: research

Gene Therapy on the Road
PMID: 31071762 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 10, 2019 Category: Genetics & Stem Cells Authors: Feng YM Tags: Curr Gene Ther Source Type: research

Novel p53 delivery systems in the treatment of head and neck cancer.
CONCLUSION: Powerful delivery systems for reproduced p53 expression and activation in cancer cells would be a promising treatment to achieve therapeutic goal for HNSCC. PMID: 31074365 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - May 10, 2019 Category: Genetics & Stem Cells Authors: Lu Y, Wang H Tags: Curr Gene Ther Source Type: research

Novel Combination Oncolytic Adenoviral Gene Therapy Armed with Dm-dNK and CD40L for Breast Cancer.
CONCLUSION: Our data suggest enhanced, selective tumor cell killing using combined gene therapy with conditionally replicating adenovirus containing Dm-dNK suicide gene and exogenous CD40 ligation (CD40L-CD40). PMID: 30848201 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - March 6, 2019 Category: Genetics & Stem Cells Authors: Wang Q, Yang M, Zhang Y, Zhong L, Zheng X Tags: Curr Gene Ther Source Type: research

Acknowledgements to Reviewers.
Authors: Abstract PMID: 30563442 [PubMed - in process] (Source: Current Gene Therapy)
Source: Current Gene Therapy - December 21, 2018 Category: Genetics & Stem Cells Tags: Curr Gene Ther Source Type: research

Gene-based therapeutic tools in the treatment of Cornea Disease.
DISCUSSION: In the text, we first introduce the pathological development of corneal disease in the aspects of neovascularization and inflammation. We summarize how MSCs become an ideal candidate in cell therapy for treating injured corneal, focusing on cell biology, property and features. We provide an updated review of gene-based therapies in animals and preclinical studies in the aspects of controlling target gene expression, safety and efficacy. Gene transfer vectors are potent to induce candidate protein expression. Delivered by vectors, MSCs are equipped with certain characters by expressing protein of interest, which...
Source: Current Gene Therapy - December 13, 2018 Category: Genetics & Stem Cells Authors: Lu XX, Zhao SZ Tags: Curr Gene Ther Source Type: research

Functional Improvement of Chimeric Antigen Receptor Through Intrinsic Interleukin-15R α Signaling.
Functional Improvement of Chimeric Antigen Receptor Through Intrinsic Interleukin-15Rα Signaling. Curr Gene Ther. 2018 Nov 15;: Authors: Nair S, Wang JB, Tsao ST, Liu Y, Zhu W, Slayton WB, Moreb JS, Dong L, Chang LJ Abstract Recent studies of CD19-specific chimeric antigen receptor (CAR)-modified T cells (CARTs) have demonstrated unprecedented successes in treating refractory and relapsed B cell malignancies. The key to the latest CART therapy advances can be attributed to the improved co-stimulatory signals in the CAR design. Here we established several novel CARs by incorporating T cell signal...
Source: Current Gene Therapy - November 15, 2018 Category: Genetics & Stem Cells Authors: Nair S, Wang JB, Tsao ST, Liu Y, Zhu W, Slayton WB, Moreb JS, Dong L, Chang LJ Tags: Curr Gene Ther Source Type: research

Comparative Activity of Adenosine Deaminase Acting on RNA (ADARs) Isoforms for Correction of Genetic Code in Gene Therapy.
This study provides information that should facilitate the rational use of ADAR variants for genetic restoration and treatment of genetic diseases. PMID: 30426900 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 14, 2018 Category: Genetics & Stem Cells Authors: Azad TA, Qulsum U, Tsukahara T Tags: Curr Gene Ther Source Type: research

Design of artificial immunogens containing melanoma-associated T-cell epitopes.
CONCLUSION: The described approach may become a common platform for designing not only vaccines against oncological diseases but also immunoprophylactic vaccines against infectious diseases. PMID: 30421674 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 12, 2018 Category: Genetics & Stem Cells Authors: Borobova EA, Antonets DV, Starostina EV, Karpenko LI, Ilyichev AA, Bazhan SI Tags: Curr Gene Ther Source Type: research

Circular RNAs Serve as Novel Biomarkers and Therapeutic Targets in Cancers.
Abstract Circular RNAs (circRNAs) are a class of non-coding RNAs (ncRNAs) that structurally form closed loops without 5'-end cap and 3'-end poly(A) tail unlike linear RNAs. CircRNAs are widely present in eukaryotic cells and are presented with capabilities and characteristics of structural stability, high abundance and cell-/tissue-specific expression. A growing body of research now suggests that the aberrant expression of circRNA is intimately relevant to the occurrence and development of cancer. In this review, we mainly discuss the differentially expressed circRNAs in cancer tissues, plasma and exosomes, which ...
Source: Current Gene Therapy - November 9, 2018 Category: Genetics & Stem Cells Authors: Fang S, Pan J, Zhou C, Tian H, He J, Shen W, Jin X, Meng X, Jiang N, Gong Z Tags: Curr Gene Ther Source Type: research

Clinical-Grade Oncolytic Adenovirus Purification Using Polysorbate 20 as an Alternative for Cell Lysis.
Peixoto C Abstract Oncolytic virus therapy is currently considered as a promising therapeutic approach for cancer treatment. Adenovirus is well-known and extensively characterized as an oncolytic agent. The increasing number of clinical trials using this virus generates the demand for the development of a well-established purification approach. Triton X-100 is commonly used in cell lysis buffer preparations. The addition of this surfactant in the list of substances with very high concern of the Registration, Evaluation, Authorization and Restriction of Chemicals (REACH) regulation promoted the research for effect...
Source: Current Gene Therapy - November 9, 2018 Category: Genetics & Stem Cells Authors: Moleirinho MG, Rosa S, Carrondo MJT, Silva RJS, Hagner-McWhirter Å, Ahlén G, Lundgren M, Alves PM, Peixoto C Tags: Curr Gene Ther Source Type: research

Emerging Therapeutic Approaches for Diamond Blackfan Anemia.
We present different DNA- and RNA-based technologies as well as new candidate pharmacological treatments and discuss their relevance and potential applicability for the cure of DBA. PMID: 30411682 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 9, 2018 Category: Genetics & Stem Cells Authors: Aspesi A, Borsotti C, Follenzi A Tags: Curr Gene Ther Source Type: research

Clinical Observation of Patients with Leber's Hereditary Optic Neuropathy before Gene Therapy.
CONCLUSION: Most patients of Leber's hereditary optic neuropathy gradually stabilize visual function with prolonged onset time, and the lower possibility of spontaneous vision recovery provides a basis for future evaluation of the effectiveness of gene therapy. PMID: 30394208 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 5, 2018 Category: Genetics & Stem Cells Authors: Yuan J, Zhang Y, Liu H, Tian Z, Li X, Zheng Y, Gao Q, Song L, Xiao X, Sun J, Wang Z, Li B Tags: Curr Gene Ther Source Type: research

Human Disease System Biology.
Abstract PMID: 30387394 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - November 1, 2018 Category: Genetics & Stem Cells Authors: Cheng L, Hu Y Tags: Curr Gene Ther Source Type: research

ExomiRs: A Novel Strategy in Cancer Diagnosis and Therapy.
Abstract Exosomes play a critical role in intercellular communication between cancer cells and their environments. These secreted nanovesicles can transfer different cargos such as mRNAs, proteins and microRNA (miRNA) to recipient cells. Exosomal miRNAs (exomiRs) derived from tumor cells have emerged as key players in cancer promotion via impairment of the immune system response, tumor growth, metastasis, angiogenesis, and chemotherapeutic drug resistance. Moreover, since dysregulation of miRNA expression in tumor cells can be reflected by distinct profiles of exomiRs extracted from the bodily fluids of cancer pat...
Source: Current Gene Therapy - October 17, 2018 Category: Genetics & Stem Cells Authors: Gorji-Bahri G, Hashemi A, Moghimi HR Tags: Curr Gene Ther Source Type: research

Human Disease System Biology.
Abstract In recent years, genome-wide association studies (GWAS) and next-generation sequencing technologies have been widely used to detect the common genetic variants of diseases. Despite these successes, the majority of genetic architecture of human complex diseases remains unknown. In the post-genome era, the major challenge is to mine novel disease risks from multi-level omics data using system biology methods, which may expand our knowledge of the causes of genetic disease. PMID: 30306867 [PubMed - as supplied by publisher] (Source: Current Gene Therapy)
Source: Current Gene Therapy - October 9, 2018 Category: Genetics & Stem Cells Authors: Cheng L, Hu Y Tags: Curr Gene Ther Source Type: research

Identifying Keystone Species In The Microbial Community Based On Cross-sectional Data.
Abstract In microbial communities, the keystone species have a greater impact on the performance and dynamics of ecosystem than that of other species, in which we can see from the results losing gut microbiome causes some specific diseases. A mass of ongoing studies aim at identifying links between microbial community structure and human diseases. In this paper, we are introducing a valid keystone species identification method, in which a new Spread Intensity (SI) algorithm is used. Because the accuracies of current keystone species identification algorithms are difficult to evaluate for the high diversity and unc...
Source: Current Gene Therapy - October 8, 2018 Category: Genetics & Stem Cells Authors: Li M, Zhang J, Wu B, Zhou Z, Xu Y Tags: Curr Gene Ther Source Type: research

An Observation on the Role of Autophagy in Patients with Endometriosis of Different Stages during Secretory Phase and Proliferative Phase.
Abstract Autophagy exists widely in various physiological and pathological conditions. Lots of investigations have verified that the autophagic activity is always related to the occurrence and the development of cancer. Endometriosis (EMs) is a disease that endometrium-like tissues abnormally grow outside the uterus and also considered to possess the characters of tumor because of its malignant biological behavior. Recently, several studies have already revealed that autophagy may play a potential role in proliferative-phase EMs. However, the function of autophagic activity in secretory-phase EMs is still unclear....
Source: Current Gene Therapy - October 8, 2018 Category: Genetics & Stem Cells Authors: Li M, Lu MS, Liu ML, Deng S, Tang XH, Han C, Wang HL, Li PL Tags: Curr Gene Ther Source Type: research

EZH2 RIP-seq Identifies Tissue-specific Long Non-coding RNAs.
Abstract Polycomb repressive complex 2 (PRC2) catalyzes histone methylation at H3 Lys27, and plays crucial roles during development and diseases in numerous systems. Its catalytic subunit EZH2 represents a key nuclear target for long non-coding RNAs (lncRNAs) that emerging to be a novel class of epigenetic regulator and participate in diverse cellular processes. LncRNAs are characterized by high tissue-specificity; however, little is known about the tissue profile of the EZH2-interacting lncRNAs. Here we performed a global screening for EZH2-binding lncRNAs in tissues including brain, lung, heart, liver, kidney, i...
Source: Current Gene Therapy - October 8, 2018 Category: Genetics & Stem Cells Authors: Wang Y, Xie Y, Li L, He Y, Zheng D, Yu P, Yu L, Tang L, Wang Y, Wang Z Tags: Curr Gene Ther Source Type: research

Advances in gene therapy for erectile dysfunction: promises and challenges.
Abstract Erectile dysfunction (ED) is a major health problem in men. Over the past few decades, oral phosphodiesterase type 5 (PDE5) inhibitors have been adopted as the recommended strategy to treat ED in the majority of cases. However, these oral medications require on-demand access and are not effective in some hard-to-treat populations. Other alternative treatments are also not satisfactory. In recent years, a novel therapeutic approach against ED, gene therapy, has shown great potential in preclinical models designed to provide long-term improvement of erectile function and the underlying conditions. Gene ther...
Source: Current Gene Therapy - October 4, 2018 Category: Genetics & Stem Cells Authors: Yu B, Wu C, Li T, Qin F, Yuan J Tags: Curr Gene Ther Source Type: research

Nanomedicine for gene delivery for the treatment of cardiovascular diseases.
Abstract Myocardial infarction (MI) is the most severe ischemic heart disease and directly leads to heart failure till death. Target molecules have been identified in the event of MI including increasing angiogenesis, promoting cardiomyocyte survival, improving heart function and restraining inflammation and myocyte activation and subsequent fibrosis. All of which are substantial in cardiomyocyte protection and preservation of cardiac function. To modulate target molecule expression, virus and non-virus-mediated gene transfer have been investigated. Despite successful in animal models of MI, virus-mediated gene tr...
Source: Current Gene Therapy - October 3, 2018 Category: Genetics & Stem Cells Authors: Yan C, Quan XJ, Feng YM Tags: Curr Gene Ther Source Type: research

Neuroprotection by Human Dental Pulp Mesenchymal Stem Cells: From Billions to Nano.
Abstract Mesenchymal stem cell (MSC) therapy in recent years have gained significant attention. Though the functional outcomes following MSC therapy for neurodegenerative diseases are convincing, various mechanisms for the functional recovery are being debated. Nevertheless, recent studies convincingly demonstrated that recovery following MSC therapy could be reiterated with MSC secretome per se thereby shifting the dogma from cell therapy to cell "based" therapy. In addition to various functional proteins, stem cell secretome also includes extracellular membrane vesicles like exosomes. Exosomes which ar...
Source: Current Gene Therapy - September 13, 2018 Category: Genetics & Stem Cells Authors: Venugopal C, Shobha K, Rai KS, Pinnelli VB, Kutty BM, Dhanushkodi A Tags: Curr Gene Ther Source Type: research