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Human Gene Therapy. Clinical Development

Chen Wang, Wang Zheng, Dan Yao, Qianping Chen, Lin Zhu, Junlin Zhang, Yan Pan, Jianghong Zhang, Chunlin Shao
Glioblastomas (GBMs) are the most prevalent brain tumor and exhibit poor prognosis. Radiotherapy is an important strategy for GBMs patients, however, this care remains palliative because of GBMs' radioresistance. Glioma stem cells (GSCs), as a subpopulation residing at the apex of the hierarchy, have been believed to be a pivotal population in radioresistance and recurrence of GBMs. To know the key genes involved in radioresistance of GSCs, the gene expression profiles of GSE54660 and GSE60921 were downloaded from Gene Expression Omnibus (GEO) for genetic and transcriptomic analysis to identify the potential biomarker genes differentially expressed between GSCs and GBMs...
February 12, 2019: Human Gene Therapy. Clinical Development
Jenny Greig, Jayme Nordin, Melanie Smith, Scott N Ashley, Christine Draper, Yanqing Zhu, Peter Bell, Elizabeth Lynne Buza, James M Wilson
Wilson disease (WD), an autosomal recessive disease caused by mutations in a copper-transporting P-type ATPase (Atp7b), causes severe liver damage. This disease is currently treated with the lifelong use of copper chelation therapy, which has side effects and does not fix copper metabolism. Here, we thoroughly characterized a mouse model of WD, the toxic milk (txJ) mouse, and used the model to test a gene therapy approach for treating WD. WD mice accumulated copper in the liver from birth; severe copper accumulation and concurrent liver disease were evident by two months of age...
January 29, 2019: Human Gene Therapy. Clinical Development
Liang Han, Yanping Hao, Jianhua Wang, Zhengjiang Wang, Hongmei Yang, Xudong Wu
Gastric cancer (GC) is the second primary cause of cancer-associated mortality around the world. Long non-coding RNAs (lncRNAs) are critical modulators of multiple cellular processes, and their abnormal expression and/or function are related to a variety of diseases, including cancer. Nowadays, various lncRNAs have been unveiled to exert a functional role in GC, but more still remain to be identified since the therapies for GC patients are limited. Herein, we discover LINC02465, a novel recognized lncRNA, is upregulated and correlated with tumour size, tumour stage, lymph node metastasis and differentiation in gastric cancer...
January 11, 2019: Human Gene Therapy. Clinical Development
Shanwei Shi, Bo Li, Yang Dong, Yang Ge, Xing Qu, Liguang Lu, Yihang Yuan, Longjiang Li, Yi Li
BACKGROUND: Malignant melanoma is an aggressive tumor with high fatality and poor prognosis, mainly due to the lack of efficient treatment methods. The present study was performed to investigate the potential antitumor effects of recombinant adenovirus p53 (rAd-p53) on human malignant melanoma. MATERIALS AND METHODS: The optimal viral titer on a human malignant melanoma (A-375) cell line was determined for the rAd-p53 treatment. The invasive abilities, apoptosis, variations in the cell cycle and molecular expression levels of A-375 cells were detected after infected by rAd-p53...
January 8, 2019: Human Gene Therapy. Clinical Development
Gbolahan Amusa, Taylor Feehley, J Kipchirchir Bitok, Geulah Livshits, Natalya Gertsik
The era of gene therapy has begun. In recent years, potentially breakthrough datasets and rapidly expanding company pipelines have begun to overshadow the unfulfilled promise characteristic of the gene therapy sector in decades prior. One barometer for progress in the space can be seen in stock markets, where NASDAQ-listed in vivo gene therapy companies we follow have increased from 4 companies with $1.9 billion in market capitalization on January 31, 2014, to 24 companies with $30.5 billion in market capitalization on October 31, 2018...
December 14, 2018: Human Gene Therapy. Clinical Development
James M Wilson
No abstract text is available yet for this article.
December 2018: Human Gene Therapy. Clinical Development
James M Wilson
No abstract text is available yet for this article.
December 2018: Human Gene Therapy. Clinical Development
Alex Philippidis
No abstract text is available yet for this article.
December 2018: Human Gene Therapy. Clinical Development
Yu Hua Li, Ling Wang, Tao Zhu, Shipo Wu, Liqiang Feng, Ping Cheng, Jing Jing Liu, Junzhi Wang
The 2014 Ebola outbreak in West Africa had brought great threat to the public health worldwide. There were no approved anti-viral therapy and vaccine available to control the disease at that time. Several kinds of Ebola vaccines were under development urgently in the world, among which the novel recombinant adenovirus type-5 vector-based Ebola vaccine( Ad5-EBOV)-the first Ebola vaccine based on the 2014 Zaire Guinea epidemic strain was developed in China and its safety and immunogenicity were demonstrated in China and SierraLeone...
November 1, 2018: Human Gene Therapy. Clinical Development
Xi Xiang, Qianying Leng, Yuanjiao Tang, Liyun Wang, Jianbo Huang, Yi Zhang, Li Qiu
Ultrasound-targeted microbubble destruction (UTMD), which has been successfully used for the treatment of many diseases, offers a promising noninvasive approach for target-specific gene delivery. This study investigated the UTMD delivery of insulin-like growth factor 1 (IGF-1) cDNA and transforming growth factor beta (TGF-β) short hairpin RNA for Achilles tendon injury in rats. Briefly, 168 rats with an injured Achilles tendon were randomly divided into seven groups: (1) IGF-1 + UTMD, (2) TGF-β + UTMD, (3) IGF-1 + TGF-β + UTMD, (4) control, (5) IGF-1, (6) TGF-β, and (7) IGF-1 + TGF-β...
October 25, 2018: Human Gene Therapy. Clinical Development
Yicong Ye, Xiliang Zhao, Yiyun Lu, Bo Long, Shuyang Zhang
BACKGROUND: Atherosclerosis (AS) is a complex, chronic inflammatory disease that is characterized by plaque build-up within arterial vessel walls. Preclinical trials have suggested that vorinostat, a pan-histone deacetylase inhibitor (HDACi), reduces vascular inflammation and AS, but the underlying protective mechanism has not been fully elucidated. The present study aimed to identify altered gene expression profiles in aortic tissues from ApoE-/- mice after vorinostat treatment. METHODS: Male ApoE-/- mice fed a high fat diet were treated with either vorinostat or vehicle and aortic plaque area was quantified 8 weeks after treatment...
October 4, 2018: Human Gene Therapy. Clinical Development
Chunjuan Song, Thomas J Conlon, Wen-Tao Deng, Kirsten E Coleman, Ping Zhu, Cayrn Plummer, Savitri Mandapati, Mailin Van Hoosear, Kari B Green, Peter Sonnentag, Alok K Sharma, Adrian Timmers, Paulette Robinson, David R Knop, William W Hauswirth, Jeffrey David Chulay, Mark S Shearman, Guo-Jie Ye
AGTC is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The vector contains a codon-optimized human RPGR cDNA (RPGRco) driven by a photoreceptor-specific promoter (G protein-coupled receptor kinase 1, GRK1) and is packaged in an AAV2 capsid with three surface tyrosine residues changed to phenylalanine (AAV2tYF). We conducted a safety and potency study of this vector administered by subretinal injection in the naturally occurring RPGR-deficient Rd9 mouse model...
October 3, 2018: Human Gene Therapy. Clinical Development
Margarita Garcia, Rafael Moreno, Marta Gil, Manel Cascallo, Maria Ochoa de Olza, Carmen Cuadra, Josep Maria Piulat, 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 USA 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...
September 18, 2018: Human Gene Therapy. Clinical Development
James M Wilson
No abstract text is available yet for this article.
September 2018: Human Gene Therapy. Clinical Development
(no author information available yet)
No abstract text is available yet for this article.
September 2018: Human Gene Therapy. Clinical Development
James M Wilson
No abstract text is available yet for this article.
September 2018: Human Gene Therapy. Clinical Development
Nadine A Kahle, Tobias Peters, Ditta Zobor, Laura Kuehlewein, Susanne Kohl, Ahmad Zhour, Annette Werner, Immanuel P Seitz, Vithiyanjali Sothilingam, Stylianos Michalakis, Martin Biel, Marius Ueffing, Eberhart Zrenner, Karl U Bartz-Schmidt, M Dominik Fischer, Barbara J C Wilhelm
Achromatopsia is an autosomal recessively inherited congenital defect characterized by a lack of cone photoreceptor function, leading to severely impaired vision. In this clinical study, achromatopsia patients were treated with a single subretinal injection of rAAV.hCNGA3 to restore cone function. The focus of this trial was on the safety of the treatment. After surgery, patients were monitored in eight extensive visits during the first year, followed by a 4-year follow-up period with annual visits. For essential complementation of the standard ophthalmological and systemic examinations, disease-specific methods were developed to assess the safety, efficacy, and patient-reported outcomes in this trial...
September 2018: Human Gene Therapy. Clinical Development
Yang Nan, Hongbao Guo, Liyun Guo, Le Wang, Bingcheng Ren, Kai Yu, Qiang Huang, Yue Zhong
MicroRNAs (miRNAs) are widely expressed and regulate most biological functions. According to several research groups, miR-451 expression is decreased in glioma cells. A previous study also confirmed that miRNA-451 inhibits the PI3K/AKT signaling pathway by directly targeting CAB39, which inhibits glioma cell growth and proliferation and induces apoptosis. However, the specific regulatory mechanism is unclear. Mammalian target of rapamycin (mTOR) is a central regulator of the differentiation, proliferation, and migration of a variety of cells...
September 2018: Human Gene Therapy. Clinical Development
Stéphane Palfi, Jean Marc Gurruchaga, Hélène Lepetit, Katy Howard, G Scott Ralph, Sarah Mason, Gaëtane Gouello, Philippe Domenech, Philip C Buttery, Philippe Hantraye, Nicola J Tuckwell, Roger A Barker, Kyriacos A Mitrophanous
Parkinson's disease is typically treated with oral dopamine replacement therapies. However, long-term use is complicated by motor fluctuations from intermittent stimulation of dopamine receptors and off-target effects. ProSavin, a lentiviral vector based gene therapy that delivers local and continuous dopamine, was previously shown to be well tolerated in a Phase I/II first-in-human study, with significant improvements in motor behavior from baseline at 1 year. Here, patients with Parkinson's disease from the open-label trial were followed up in the long term to assess the safety and efficacy of ProSavin after bilateral injection into the putamen...
September 2018: Human Gene Therapy. Clinical Development
Zuzana Kočí, Tomáš Boráň, Petr Krůpa, Šárka Kubinová
Advanced therapy medicinal products (ATMPs) represent a new generation of biopharmaceuticals that comprise gene therapy medicinal products (GTMPs), somatic cell therapy products (CTMPs), tissue engineered products (TEPs), and combined advanced therapy medicinal products (cATMPs). The joint effort of the academia-industry-regulatory triangle translated scientific progress into ten authorized ATMPs in the European Community. This notion holds promise for the whole field of ATMP therapies that have been increasingly evaluated in a number of clinical studies, also in the Czech Republic (CR)...
September 2018: Human Gene Therapy. Clinical Development
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