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Human Gene Therapy

Shiran Yu, Hai Yuan, Min Yang, Xinran Cao, Jing Chen, Xiaoming Zhou, Bo Dong
(Pro) renin receptor (PRR) is a novel component of the renin-angiotensin system (RAS) that has been demonstrated to be involved in cardiovascular diseases. Recent research reported that diabetic cardiomyopathy (DCM) may be accompanied by high expression of PRR, indicating that PRR may be a potential therapeutic target for DCM. However, the exact mechanisms of PRR in DCM have not been completely clarified. In this study, we hypothesized that PRR is involved in the pathological progression of DCM and can exacerbate myocardial fibrosis and cardiac dysfunction...
January 11, 2019: Human Gene Therapy
Xiubin He, Haihua Xie, Xiexie Liu, Feng Gu
Traditional gene therapy (gene replacement), as the most compelling concepts in clinical medicine, have gained a breakthrough in treating inherited diseases. Adeno-associated virus (AAV) has emerged as a highly promising vector with innate ability, boosting the development of gene replacement and gene targeting. With the recent advance of engineered nucleases that work efficiently in human cells, AAV mediated-genome editing with nucleases have raised hopes for the gene therapy of inherited or non-inherited diseases...
December 27, 2018: Human Gene Therapy
Wenyi Liu, Mingming Liu, Yong Liu, Shiying Li, Chuanhuang Weng, Yan Fu, Juncai He, Yu Gong, Weiping Liu, CongJian Zhao, ZhengQin Yin
To study whether ectopic human melanopsin (hMel) in retinal ganglion cells (RGCs) could restore the visual function in end-stage retinal degeneration, AAV2/8-CMV-hMel/FYP was injected into the intravitreal space of Royal College of Surgeons (RCS) rats. We observed that ectopic hMel/YFP was dominantly expressed in the RGCs of the RCS rat retinae. At 30-45 d after administration of AAV2/8-CMV-hMel/FYP in RCS rats, the FVEP and behavioral results demonstrated that visual function was significantly improved compared with that in the control group, while no improvement in FERG was observed at this time point...
December 22, 2018: Human Gene Therapy
Hui Yan, Jiejie Meng, Shasha Zhang, Hang Zhuang, YuE Song, Xiao Xiao, Dao Wen Wang, Jiangang Jiang
Myostatin, a negative modulator of muscle growth, has been considered as a potential target for treatment of type 2 diabetes. In our previous work, we found that myostatin inhibition by adeno-associated virus (AAV) mediated gene delivery of myostatin propeptide (MPRO) could improve muscle mass and achieve therapeutic effects on glucose regulation and lipid metabolism in db/db mice. In this study, we investigated whether pre-intervention of rAAV-mediated expression of MPRO could lower the incidence of type 2 diabetes...
December 10, 2018: Human Gene Therapy
Hildegard Büning, Uta Griesenbach, Boris Fehse, Seppo Ylä-Herttuala, Nicholas P Anagnou, Victor van Beusechem, Angel Raya, Els Verhoeyen
No abstract text is available yet for this article.
December 4, 2018: Human Gene Therapy
Anil Chekuri, Bhubanananda Sahu, Venkata R M Chavali, Marina Voronchikhina, Angel-Soto Hermida, John J Suk, Akhila N Alapati, Dirk-Uwe Bartsch, Raul Ayala-Ramirez, Juan C Zenteno, Astra Dinculescu, Monica M Jablonski, Shyamanga Borooah, Radha Ayyagari
Patients harboring homozygous c.498_499insC mutations in MFRP demonstrate hyperopia, microphthalmia, retinitis pigmentosa, retinal pigment epithelial (RPE) atrophy, variable degrees of foveal edema and optic disc drusen. The disease phenotype is variable however, with some patients maintaining good central vision and cone function till late in the disease. We developed a knock-in mouse model with the c.498_499insC mutation in Mfrp (Mfrp KI/KI) to understand the effects of these mutations in the retina. Our model shares many of the features of human clinical disease including reduced axial length, hyperopia, retinal degeneration, RPE atrophy and decreased electrophysiological responses...
November 30, 2018: Human Gene Therapy
Yang Wang, Xiaobing Zhou, Zhen Wu, Han Hu, Jing Jin, Yanping Hu, Yuting Dong, Jianwen Zou, Zeyong Mao, Xiaotai Shi, Yan Huo, Jianjun Lyu, Zhizheng Fang, Wen Zhang, Yujie Zhu, Bo Li, Binlei Liu
Dear editor: Here within enclosed is the manuscript entitled "Preclinical safety evaluation of oncolytic herpes simplex virus type 2" by Yang et al., which we would like to submit to Human Gene Therapy for publication. All the authors have read the final version of the manuscript and approved submission of the article to Human Gene Therapy. The authors claim that the work is original research that has not been published previously and not under consideration for publication elsewhere, in whole or part...
November 30, 2018: Human Gene Therapy
Xianqiang Wang, Sen Guo, Rui Zhao, Yanfeng Liu, Guangyun Yang
Hepatoblastoma usually occurs in infant or toddlers. Nowadays, long non-coding RNAs (lncRNAs) have been widely studied in various human cancers. However, the role of lncRNAs in hepatoblastoma still needs to be elucidated. This study aimed to investigate the biological role of a certain lncRNA in hepatoblastoma. According to the TCGA data, upregulation of lncRNA LUCAT1 was closely associated with the poor overall survival of hepatoblastoma patients. Next, the expression patterns of LUCAT1 in hepatoblastoma tissues and cell lines were detected with qRT-PCR...
November 27, 2018: Human Gene Therapy
Constanca Figueiredo, Marco Carvalho Oliveira, Chen Chen-Wacker, Katharina Jansson, Klaus Höffler, Yuliia Yuzefovych, Olena Pogozhykh, Zhu Jin, Mark Kühnel, Danny Jonigk, Bettina Wiegmann, Wiebke Sommer, Axel Haverich, Gregor Warnecke, Rainer Blasczyk
Disparities at the major histocompatibility complex (MHC) antigens and associated minor antigens trigger harmful immune responses, leading to graft rejection after transplantation. This study shows that MHC-silenced cells and tissues are efficiently protected against rejection. In complex vascularized organs, the endothelium is the major interface between donor and recipient. This study therefore aimed to reduce the immunogenicity of the lung by silencing MHC expression on the endothelium. In porcine lungs, short-hairpin RNAs targeting beta-2-microglobulin and class II-transactivator transcripts were delivered by lentiviral vectors during normothermic ex vivo perfusion to silence swine leukocyte antigen (SLA) I and II expression permanently...
November 20, 2018: Human Gene Therapy
Yang Penghui, Sun Fang, Wang Ruilan, Lei Guanglin, Gu Hongjing, Duan Yueqiang, Zhao Zhongpeng, Yang Xiaolan, Wang Zhaohai, Zhang Shaogeng, Wang Xiliang
Oncolytic virotherapy is a promising strategy for the treatment of cancer. Influenza A virus has shown potential as an oncolytic agent. In this study, a recombinant PR8 influenza viral vector, called delNS1-GM-CSF, was generated with a partial deletion in NS and the granulocyte-macrophage colony-stimulating factor (GM-CSF) coding sequence inserted into the influenza nonstructural protein 1 gene. The morphological characteristics of delNS1-GM-CSF were examined. The delNS1-GM-CSF virus replicated well in various cell lines, including MDCK, A549, SMCC7721, and HepG2 cells...
November 20, 2018: Human Gene Therapy
Shuai Zhen, Xu Li
Long noncoding RNAs (LncRNA), a class of transcripts with lengths >200 nt, play a master role in the regulation of cancer pathogenesis. Recently, the CRISPR-Cas9 system has been explored as a revolutionary genome editing tool for molecular biology. Growing evidence shows that LncRNAs can be targeted by the CRISPR-Cas9 system used for evaluating its function. Thus, the CRISPR-Cas9 systems provide a novel gene-editing strategy for the modification of LncRNA expression. This review summarizes current knowledge of the functions and underlying mechanisms of LncRNA by CRISPR-Cas9...
November 20, 2018: Human Gene Therapy
Shuai Zhen, Jiaojiao Lu, Wei Chen, Le Zhao, Xu Li
Targeted therapy produces objective responses in bladder cancer patients, although the responses can be short. Meanwhile, response rates to immune therapy are lower, but the effects are more durable. Based on these findings, it was hypothesized that urothelial carcinoma associated 1 (UCA1)-targeted therapy could synergize with programmed cell death 1 (PD-1) blockade to enhance antitumor activity. To test this hypothesis, the effects of CRISPR-Cas9 targeting of UCA1 and PD-1 were assessed in vitro and in vivo...
November 19, 2018: Human Gene Therapy
Margarita García, Rafael Moreno, Marta Gil-Martin, Manel Cascallò, Maria Ochoa de Olza, Carmen Cuadra, Josep Maria Piulats, Valentin Navarro, Marta Domenech, Ramon Alemany, Ramon Salazar
Oncolytic viruses represent a unique type of agents that combine self-amplification, lytic, and immunostimulatory properties against tumors. A local and locoregional clinical benefit has been demonstrated upon intratumoral injections of an oncolytic herpes virus in melanoma patients, leading to its approval in the United States and Europe for patients without visceral disease (up to stage IVM1a). However, in order to debulk and change the local immunosuppressive environment of tumors that cannot be injected directly, oncolyitc viruses need to be administered systemically...
November 13, 2018: Human Gene Therapy
Joana Serra, Cláudia P A Alves, Liliana Brito, Gabriel A Monteiro, Joaquim M S Cabral, Duarte Miguel F Prazeres, Cláudia L da Silva
Peripheral artery disease (PAD) is a debilitating and prevalent condition characterized by blockage of the arteries, leading to limb amputation in more severe cases. Mesenchymal stem/stromal cells (MSC) are known to have intrinsic regenerative properties that can be potentiated by the introduction of pro-angiogenic genes such as the vascular endothelial growth factor (VEGF). Herein, the use of human bone marrow MSC transiently transfected with minicircles encoding for VEGF is proposed as an ex vivo gene therapy strategy to enhance angiogenesis in PAD patients...
November 13, 2018: Human Gene Therapy
QingQing Hao, XueFei Dong, Xu Chen, Feng Yan, Xiaoyu Wang, HaiShui Shi, Bo Dong
Recent studies have demonstrated that angiotensin-converting enzyme 2 (ACE2) plays an important role in the pathogenesis of abdominal aortic aneurysms (AAAs). However, few studies have reported the direct effect of ACE2 overexpression on the aneurysm. This study hypothesized that the overexpression of ACE2 may prevent the pathogenesis of aneurysms by decreasing RAS activation. Thirty-nine mice were randomly assigned to three groups (n = 13 in each group): the Ad.ACE2 group, the Ad.EGFP group, and a control group...
November 13, 2018: Human Gene Therapy
Ziying Yan, Wei Zou, Zehua Feng, Weiran Shen, Soo-Yeun Park, Xuefeng Deng, Jianming Qiu, John F Engelhardt
The genome of recombinant adeno-associated virus 2 (rAAV2) remains a promising candidate for gene therapy for cystic fibrosis (CF) lung disease, but due to limitations in the packaging capacity and the tropism of this virus with respect to the airways, strategies have evolved for packaging an rAAV2 genome (up to 5.8kb) into the capsid of human bocavirus 1 (HBoV1) to produce a chimeric rAAV2/HBoV1 vector. Although a replication-incompetent HBoV1 genome has been established as a trans helper for capsid complementation, this system remains suboptimal with respect to virion yield...
November 6, 2018: Human Gene Therapy
Michelle E McClements, Alun R Barnard, Mandeep S Singh, Peter Charbel Issa, Zhichun Jiang, Roxana A Radu, Robert E MacLaren
The recent approval in the US of the first adeno-associated viral (AAV) vector for the treatment of an inherited retinal degeneration validates this approach for the treatment of many other diseases. A major limiting factor continues to be the size restriction of the AAV transgene at under 5kb. Stargardt disease is the most prevalent form of recessively inherited blindness and is caused by mutations in ABCA4, which has a coding sequence length of 6.8kb. Dual vector approaches increase the capacity of AAV gene therapy but at the cost of substantially reduced levels of target protein, which may be insufficient to achieve a therapeutic effect...
November 1, 2018: Human Gene Therapy
Yingxi Xu, Saisai Li, Ying Wang, Jia Liu, Xinhe Mao, Haiyan Xing, Zheng Tian, Kejing Tang, Xiaolong Liao, Qing Rao, Dongsheng Xiong, Min Wang, Jianxiang Wang
CD20 is an effective immunotherapy target for CD20+ B-cell malignant cells. Monoclonal antibody, especially Rituximab, has been a conventional strategy in the treatment of B-cell malignancies such as non-Hodgkin's lymphoma (NHL). However, treatment with monoclonal antibodies has not been enough to overcome the refractory/relapsed problems. Chimeric antigen receptor engineered T (CAR-T) cells have revealed excellent therapeutic effect on lymphocytic leukemia in recent years. In this study, a CD20 specific CAR was constructed and the cytotoxic efficacy of CD20 CAR-T cells on B-cell malignant cells was evaluated by CD107a degranulation, pro-inflammation cytokine production and true lytic ability in vitro and in vivo...
November 1, 2018: Human Gene Therapy
Shuaishuai Huang, Yu Ren, Xue Wang, Lissy Lazar, Suya Ma, Guobin Weng, Jinshun Zhao
Chronic renal disease or acute renal injury could result in end-stage renal disease or renal failure. Sonoporation, induced by ultrasound-targeted microbubble destruction (UTMD), has evolved as a new technology for gene delivery. It increases the transfection efficiency of the genes into target kidney tissues. Moreover, UTMD-mediated gene delivery can directly repair the damaged tissues or improve the recruitment and homing of stem cells in the recovery of injured tissues, which has the potential to act as a non-viral and effective method to current gene therapy...
October 31, 2018: Human Gene Therapy
Yimin Xiao, Yanxia Zhang, Yueqiu Chen, Jingjing Li, Zihan Zhang, Yimin Sun, Han Shen, Zhenao Zhao, Zan Huang, Wencheng Zhang, Weiqian Chen, Zhenya Shen
Follistatin-like 1 (Fstl1) protects cardiomyocytes from a broad spectrum of pathologic injuries including myocardial infarction (MI). It is worthy of note that although cardiac Fstl1 is elevated in post-MI microenvironment, its cardioprotective role is still restricted to a limited extent considering the frequency and severity of adverse cardiac remodeling following MI. We therefore propose that intrinsic Fstl1-suppressing microRNA (miRNA) may exist in the heart and its neutralization may further facilitate post-MI recovery...
October 31, 2018: Human Gene Therapy
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