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Human Gene Therapy

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https://www.readbyqxmd.com/read/29466872/attenuation-of-egfl7-expression-inhibits-growth-hormone-producing-pituitary-adenomas-growth-and-invasion
#1
Qian Liu, Jianpeng Wang, Hongye Yang, Hua Gao, Chuzhong Li, Xiaolei Lan, Yazhuo Zhang
Invasiveness of growth hormone-producing pituitary adenomas (GHPAs) causes difficulties in safe and complete adenoma removal during surgery and often leads to high recurrence. Epidermal growth factor-like domain 7 (EGFL7) has been shown to be able to promote tumor angiogenesis, growth, invasiveness and metastasis through Notch signaling pathway. We previously demonstrated that EGFL7 was overexpressed in GHPAs. In this study, we report that EGFL7 and Notch2 (positive correlation with EGFL7) are overexpressed in invasive GHPA...
February 21, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29466871/design-of-a-novel-gene-therapy-construct-to-achieve-sustained-brain-derived-neurotrophic-factor-signalling-in-neurons
#2
Andrew Osborne, Aiden Xz Wang, Alessia Tassoni, Peter S Widdowson, Keith R Martin
Brain-derived neurotrophic factor (BDNF) acting through the tropomyosin-related receptor-B (TrkB) is an important signalling system for the maintenance and survival of neurons. Gene therapy using either recombinant adeno-associated viral (AAV) or lentiviral vectors can provide sustained delivery of BDNF to tissues where reduced BDNF signalling is hypothesised to contribute to disease pathophysiology. However, elevation in BDNF at target sites has been shown to lead to a down-regulation of TrkB receptors, thereby reducing the effect of chronic BDNF delivery over time...
February 21, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29463117/micro-dystrophin-gene-therapy-goes-systemic-in-duchenne-muscular-dystrophy-patients
#3
Dongsheng Duan
Whole body systemic gene therapy is likely the most effective way to greatly reduce the disease burden of Duchenne muscular dystrophy (DMD), an X-linked inherited muscle disease that leads to premature death in early adulthood. Genetically, DMD is due to null mutation of the dystrophin gene, one of the largest genes in the genome. Recent studies have shown highly promising improvements in animal models with intravascular delivery of the engineered micro-dystrophin gene by adeno-associated virus (AAV). Several human trials are now started to advance AAV micro-dystrophin therapy to DMD patients...
February 20, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29448836/aav8-gene-therapy-rescues-the-newborn-phenotype-of-a-mouse-model-of-crigler-najjar
#4
Jenny A Greig, Jayme M L Nordin, Christine Draper, Peter Bell, James M Wilson
Adeno-associated viral (AAV) vectors can target the liver, making them an attractive platform for gene therapy approaches that require the correction of hepatocytes. Crigler-Najjar syndrome is an autosomal recessive disorder of bilirubin metabolism that occurs when the liver's uridine diphosphate glucuronosyl transferase 1A1 (UGT1A1) enzyme activity is partially or completely absent. This syndrome is characterized by elevated bilirubin levels in the blood. We developed an AAV8 vector expressing a codon-optimized human version of UGT1A1 from a liver-specific promoter...
February 16, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29433343/cardiac-specific-expression-of-%C3%A2-h2-r15-mini-dystrophin-normalized-all-ecg-abnormalities-and-the-end-diastolic-volume-in-a-23-m-old-mouse-model-of-duchenne-dilated-cardiomyopathy
#5
Nalinda B Wasala, Jin-Hong Shin, Yi Lai, Yongping Yue, Federica Montanaro, Dongsheng Duan
Heart disease is a major health threat for Duchenne/Becker muscular dystrophy patients and carriers. Expression of a 6 to 8-kb mini-dystrophin gene in the heart holds promise to dramatically change the disease course. However the mini-dystrophin gene cannot be easily studied with adeno-associated virus (AAV) gene delivery because the size of the minigene exceeds AAV packaging capacity. We previously studied cardiac protection of the ∆H2-R19 minigene using the cardiac specific transgenic approach. Although this minigene fully normalized skeletal muscle force, it only partially corrected ECG and heart hemodynamics in dystrophin-null mdx mice that had moderate cardiomyopathy...
February 13, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29409356/therapeutic-potential-of-lentivirus-mediated-glucagon-like-peptide-1-glp-1-gene-therapy-for-diabetes
#6
Hale M Tasyurek, Hasan A Altunbas, Mustafa K Balci, Thomas S Griffith, Salih Sanlioglu
Postprandial glucose-induced insulin secretion from the islets of Langerhans is facilitated by glucagon-like peptide-1 (GLP-1) - a metabolic hormone with insulinotropic properties. Among the variety of effects it mediates, GLP-1 induces delta cell secretion of somatostatin, inhibits alpha cell release of glucagon, reduces gastric emptying, and slows food intake. These events collectively contribute to weight loss over time. During type 2 diabetes (T2DM), however, the incretin response to glucose is reduced and accompanied by a moderate reduction in GLP-1 secretion...
February 6, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29409352/hepatocyte-growth-factor-gene-therapy-for-ischemic-diseases-and-tissue-regeneration
#7
Lisheng Wang, Hua Wang, QingLin Zhang, Zhijian Yang, Fanxuan Kong, Chu-Tse Wu
Stem cells and gene therapy have become promising strategies for treating ischemia diseases and regenerating tissue. Hepatocyte growth factor (HGF) is an angiogenic growth factor with multiple functions including promoting angiogenesis, regulating inflammation, inhibiting fibrosis and activating tissue regeneration. Numerous preclinical experiments and clinical trials have demonstrated the feasibility and efficacy of HGF gene therapy in treatment of ischemia diseases and tissue regeneration. This review summarized the current advances of therapeutic angiogenesis using HGF gene transfer and modified stem cells...
February 6, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29409351/cd33-specific-chimeric-antigen-receptor-t-cells-with-different-costimulators-showed-potent-anti-leukemia-efficacy-and-different-phenotype
#8
Saisai Li, Zhongfei Tao, Yingxi Xu, Jia Liu, Na An, Ying Wang, Haiyan Xing, Zheng Tian, Kejing Tang, Xiaolong Liao, Qing Rao, Min Wang, Jianxiang Wang
Acute myeloid leukemia (AML) is a kind of a malignant hematologic tumor caused by uncontrolled repopulation of myeloid hematopoietic stem cells (HSCs). Current therapeutic effects for AML patients are unsatisfactory. Especially relapsed and refractory AML still have poor prognosis. T cell modified by chimeric antigen receptor (CAR) is an immunotherapeutic strategy for malignancies, which has a broad developing prospect. Most of AML cells overexpress the myeloid antigen CD33. Therefore, CD33-specific CAR-T cells with different costimulators (CD28, 4-1BB or both, referred to as CD33 28z...
February 6, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29390873/preclinical-models-in-chimeric-antigen-receptor-engineered-t-cell-therapy
#9
Elizabeth Louise Siegler, Pin Wang
Cancer immunotherapy has enormous potential in inducing long-term remissions in cancer patients, and chimeric antigen receptor-engineered T (CAR-T) cells have been largely successful in treating hematological malignancies in the clinic. CAR-T therapy has not been as effective in treating solid tumors, in part due to the immunosuppressive tumor microenvironment. Additionally, CAR-T therapy can cause dangerous side effects, including off-tumor toxicity, cytokine release syndrome, and neurotoxicity. Animal models of CAR-T therapy often fail to predict such adverse events and frequently overestimate the efficacy of the treatment...
February 1, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29385852/clinical-trial-of-mgmt-p140k-gene-therapy-in-the-treatment-of-paediatric-patients-with-brain-tumours
#10
Belinda Kramer, Radhika Singh, Jessica Wischusen, Rebecca Velickovic, Amanda Rush, Shiloh Middlemiss, Yu-Wooi Ching, Ian Edward Alexander, Geoffrey B McCowage
Gene transfer targeting haematopoietic stem cells (HSC) in children has shown sustained therapeutic benefit in the treatment of genetic diseases affecting the immune system, most notably in the severe combined immuno-deficiencies affecting T cell function. The HSC compartment has also been successfully targeted using gene transfer in children with genetic diseases affecting the central nervous system, such as metachromatic leukodystrophy and adrenoleukodystrophy. The HSC is also a target for genetic modification in strategies aiming to confer drug resistance to chemotherapy agents so as to reduce off-target toxicity, and to allow for chemotherapy dose escalation with the possibility of enhanced therapeutic benefit...
January 31, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29382231/overexpression-of-hepatocyte-growth-factor-mrna-induced-by-gene-transfer-attenuates-neointimal-hyperplasia-after-balloon-injury
#11
Yu He, Li Mei, Ying Jin, Xiaoping Li, Chunxiang Jin
Hepatic growth factor (HGF) has been widely used in studies on arterial remodeling after injury and results turn out to be inconsistent. The changes of endogenous HGF expression after injury also remain controversial. In this study, we clarified the role of exogenous human HGF (hHGF) gene transfer in neointimal hyperplasia and investigated the associated alterations of endogenous HGF and c-Met expressions under endothelial denudation with or without hHGF gene transfer using a balloon-injured rabbit aorta model...
January 30, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29378426/severe-toxicity-in-nonhuman-primates-and-piglets-following-high-dose-intravenous-administration-of-an-aav-vector-expressing-human-smn
#12
Christian Hinderer, Nathan Katz, Elizabeth Lynne Buza, Cecilia Dyer, Tamara Goode, Peter Bell, Laura Richman, James M Wilson
Neurotropic AAV serotypes such as AAV9 have been demonstrated to transduce spinal alpha motor neurons when administered intravenously at high doses. This observation led to the recent successful application of intravenous AAV9 delivery to treat infants with spinal muscular atrophy (SMA), an inherited deficiency of the survival of motor neuron (SMN) protein characterized by selective death of lower motor neurons. To evaluate the efficiency of motor neuron transduction with an AAV9 variant (AAVhu68) using this approach, we treated three juvenile nonhuman primates (NHPs; age 14 months) and three piglets (age 7-30 days) with an intravenous injection of an AAVhu68 vector carrying a human SMN transgene at a dose similar to that employed in the SMA clinical trial...
January 29, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29378415/severe-toxicity-in-non-human-primates-and-piglets-with-systemic-high-dose-administration-of-aav9-like-vectors-putting-patients-first
#13
https://www.readbyqxmd.com/read/29373929/third-generation-her2-chimeric-antigen-receptor-car-expression-on-human-t-cells-improves-with-two-signal-activation
#14
Mumtaz Yaseen Balkhi, Al-Rubaye Dalal, Sylvester Homsy
Patient derived T cells activated ex vivo with CD3/CD28 beads show superior expansion. Therefore, CD3/CD28 beads have huge potential to be used in the clinic for Immunotherapy applications. We devised two protocols to evaluate if the expression of third generation human epidermal growth factor receptor 2 (HER2) CAR can be improved on human T cells activated with CD3/CD28 beads. In protocol one, we used unconcentrated HER2-CAR retroviral supernatants and in the second protocol we used concentrated virus. Our results demonstrated that compared to unconcentrated viral supernatants, transduction with the concentrated virus improved the infection rate of bead activated CD4 T cells from ∿40% to ∿70%, and the fluorescent Intensity values improved from ∿12,000 to ∿28,000 MFI units...
January 26, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29366352/gene-therapy-for-hemophilia-and-duchenne-muscular-dystrophy-in-china
#15
Xionghao Liu, Mujun Liu, Lingqian Wu, Desheng Liang
Gene therapy provides hope for curing monogenic diseases caused by mutations in a single gene. Hemophilia and Duchenne muscular dystrophy (DMD) are ideal target diseases of gene therapy. Important advances have been made in clinical trials, such as AAV vectors in hemophilia and antisense in DMD. However, issues of high does of viral vectors, limited system delivery efficiency of antisense oligonucleotides (AOs) remain to be addressed. In addition, as an alternative strategy to classic gene addition, genome editing based on programmable nucleases has shown promise to in situ correct mutations...
January 24, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29361840/safety-and-efficacy-of-oxb-202-a-genetically-engineered-tissue-therapy-for-the-prevention-of-rejection-in-high-risk-corneal-transplant-patients
#16
Naghmeh Fouladi, Maria Parker, Vicky Kennedy, Katie Binley, Laura McCloskey, Julie Loader, Michelle Kelleher, Kyriacos Mitrophanous, Jt Timothy Stout, Scott Ellis
Due to both the avascularity of the cornea and the relatively immune-privileged status of the eye corneal transplantation is one of the most successful clinical transplant procedures. However in high risk patients, which account for >20% of the 180,000 transplants carried out worldwide each year, the rejection rate is high due to vascularisation of the recipient cornea. The main reason for graft failure is irreversible immunological rejection and it is therefore unsurprising that neovascularisation (both pre- and post-grafting) is a significant risk factor for subsequent graft failure...
January 23, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29357712/recent-progress-on-genetic-diagnosis-and-therapy-for-%C3%AE-thalassemia
#17
Jingzhi Zhang, Jingbin Yan, Fanyi Zeng
Thalassemia is a recessive monogenic hematological disease, due to mutations/deletions in at least one of the globin genes, resulting in a reduced amount of functional hemoglobin. From genetic diagnosis to the recent developments in gene and cell therapy, this disease has attracted much attention throughout the years. We here review the recent progress in the genetic diagnosis and therapeutic attempts in interfering with Thalassemia, especially -thalassemia in China and around the world.
January 22, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29338444/adp53-gendicine-12-years-in-clinic-the-first-approved-gene-therapy-product-for-cancer
#18
Wei-Wei Zhang, Jianglong Li, Dinggang Li, Jiliang Liu, Xiuqin Li, Wei Li, Xiaolong Xu, Michael J Zhang, Lois Chandler, Hong Lin, Aiguo Hu, Wei Xu, Dominic Man-Kit Lam
Gendicine (Recombinant Human p53 Adenovirus, AdRSV-p53), developed by Shenzhen SiBiono GeneTech Company, was approved by CFDA in 2003. It has been used in clinical oncology for 12 years since it entered the market in 2004. As the first-in-class product for cancer gene therapy, Gendicine met its expectation and demonstrated with ample clinical data that it is safe and effective for treating cancer. SiBiono manufactured 41 batches in compliant with CFDA QC/QA requirements. Total 169,571 vials (1.0x1012 vector particles per vial) were used in more than 50,000 patients, among them about 5,000 were international form over 50 nations outside China...
January 16, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29338433/in-vivo-ovarian-cancer-gene-therapy-using-crispr-cas9-system
#19
Zhi-Yao He, Ya-Guang Zhang, Yu-Han Yang, Cui-Cui Ma, Ping Wang, Wei Du, Ling Li, Rong Xiang, Xiang-Rong Song, Xia Zhao, Shaohua Yao, Yu-Quan Wei
CRISPR-Cas9 genome editing technology holds great promise for the field of human gene therapy. However, deficiency of safe and effective delivery systems restricts the biomedical application of CRISPR-Cas9 technique. Here, we use a folate receptor-targeted liposome (F-LP) to deliver a CRISPR plasmid DNA co-expressing Cas9 and single guide RNA targeting the DNA methyltransferase 1 (DNMT1) gene of ovarian cancer. F-LP binds CRISPR plasmid (gDNMT1) efficiently and the formed lipoplex (F-LP/gDNMT1) is stable and safe for injection...
January 16, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29334773/macf1-overexpression-by-transfecting-the-21-kbp-large-plasmid-pegfp-c1a-acf7-promotes-osteoblast-differentiation-and-bone-formation
#20
Yan Zhang, Chong Yin, Lifang Hu, Zhihao Chen, Fan Zhao, Dijie Li, Jianhua Ma, Xiaoli Ma, Peihong Su, Wuxia Qiu, Chaofei Yang, Pai Wang, Siyu Li, Ge Zhang, Liping Wang, Airong Qian, Cory Xian
Microtubule actin crosslinking factor 1 (MACF1) is a large spectraplakin protein known to have crucial roles in regulating cytoskeletal dynamics, cell migration, growth and differentiation. However, its role and action mechanism in bone remain unclear. This study investigated the optimal conditions for effective transfection of the large plasmid PEGFP-C1A-ACF7 (~21 kbp) containing the full-length human MACF1 cDNA and the potential role of MACF1 in bone formation. To enhance MACF1 expression, the large plasmid was transfected into osteogenic cells by electroporation in vitro and mouse calvaria with nanoparticles...
January 15, 2018: Human Gene Therapy
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