Read by QxMD icon Read

Human Gene Therapy

Qingxi Zhang, Wanling Chen, Sheng Tan, Tongxiang Lin
Parkinson's disease (PD) is the second most frequent neurodegenerative disease after Alzheimer's disease, which is characterized by low level of dopamine expressing in the striatum and deteriorated dopaminergic neurons (DAn) in Substantia nigra pars compacta (SNpc). Generation of PD-derived DAn including differentiation of human embryonic stem cell (hESC), human neural stem cell (hNSC), human induced pluripotent stem cell (hiPSC) and directly reprogramming provide an ideal tool to model PD, which created the possibilities of mimicking key essential pathological processes charactering single cell changes in vitro...
October 20, 2016: Human Gene Therapy
Cynthia N Fuhrmann
PhD-trained biomedical scientists are moving into an increasingly diverse variety of careers within the sciences. However, our graduate and postdoctoral training programs have historically focused on academic career preparation, and have not sufficiently prepared trainees for transitioning into other scientific careers. Advocates for science have raised the concern that our collective disregard of the broader career development needs for predoctoral and postdoctoral trainees could drive talent away from science in upcoming generations...
October 20, 2016: Human Gene Therapy
Yuxia Yang, Yanju Zhang, Zhenchuan Miao, Junhua Zhou, Jianyuan Luo
The bone marrow (BM) microenvironment, heavily composed of osteoblasts, plays a key role during the normal development of hematopoiesis. Endogenous miR-22 has an important function on the hematopoietic development and osteoblastic differentiation. It is unclear whether miR-22 in osteoblasts from the BM microenvironment also has an important function on the development of hematopoiesis. In this study, we found that the capacity of FBMOB-hTERT cells to expand human cord blood (CB) CD34+ cells and maintain the multipotency of CB CD34+ cells is decreased upon ectopic expression of miR-22...
October 20, 2016: Human Gene Therapy
Kara W Moyes, Nicole Ap Lieberman, Shannon A Kreuser, Harrison Chinn, Conrad Winter, Gail Deutsch, Virginia Hoglund, Reid Watson, Courtney A Crane
In spite of their successes against hematologic malignancies, immunotherapeutic interventions for the treatment of patients with glioblastoma (GBM) have thus far been unsuccessful. This is in part due to the presence of a tumor microenvironment that fosters neoplastic growth and protects the tumor from destruction by the immune system. We have developed a novel genetically engineered macrophage-based platform with the potential to minimize the effects of the suppressive tumor microenvironment and improve innate and adaptive anti-tumor immune responses...
October 19, 2016: Human Gene Therapy
Pavitra Ramachandran, Vivian Lee, Zhangyong Wei, Ji Yun Song, Giulia Casal, Therese Cronin, Keirnan Willett, Rachel Huckfeldt, Jessica I W Morgan, Tomas S Aleman, Albert M Maguire, Jean Bennett
Within the next decade, we will see many gene therapy clinical trials for eye diseases progress, which may lead to treatments for thousands of visually impaired people around the world. To target retinal diseases that affect specific cell types, several recombinant adeno-associated virus (AAV) serotypes have been generated and used successfully in pre-clinical mouse studies. Because there are numerous anatomic, and physiologic differences between the eyes of mice and 'men' and because surgical delivery approaches and immunologic responses also differ between these species, we evaluated the transduction characteristics of two promising new serotypes AAV7m8 and AAV8BP2, in retinas of animals that are most similar to those of humans: non-human primates (NHPs)...
October 18, 2016: Human Gene Therapy
(no author information available yet)
No abstract text is available yet for this article.
October 17, 2016: Human Gene Therapy
(no author information available yet)
No abstract text is available yet for this article.
October 17, 2016: Human Gene Therapy
Peter Gin-Fu Chen, Zhongjie Sun
Cold temperatures are associated with increased prevalence of hypertension. Cold exposure increases endothelin-1 (ET1) production. The purpose of this study is to determine if upregulation of ET1 contributes to cold-induced hypertension (CIH). In vivo RNAi silencing of the ET1 gene was achieved by AAV2 delivery of ET1 short-hairpin siRNA (ET1-shRNA). Four groups of male rats were used. Three groups were given AAV.ET1-shRNA, AAV.SC-shRNA (scrambled shRNA), and phosphate-buffered saline (PBS), respectively, before exposure to a moderately cold environment (6...
October 12, 2016: Human Gene Therapy
Peter A Campochiaro, Andreas K Lauer, Elliott H Sohn, Tahreem A Mir, Stuart Naylor, Matthew C Anderton, Michelle Kelleher, Richard Harrop, Scott Ellis, Kyriacos A Mitrophanous
Neovascular age-related macular degeneration (NVAMD) is a prevalent cause of vision loss. Intraocular injections of VEGF-neutralizing proteins provide benefit, but many patients require frequent injections for a prolonged period. Benefits are often lost over time due to lapses in treatment. New treatments that sustain anti-angiogenic activity are needed. This study tested the safety and expression profile of a lentiviral Equine Infectious Anemia Virus (EIAV) vector expressing endostatin and angiostatin (RetinoStat(®))...
September 26, 2016: Human Gene Therapy
Divya Punwani, Misako Kawahara, Jason Yu, Ukina Sanford, Sushmita Roy, Kiran Patel, Denise A Carbonaro, Andrea D Karlen, Sara Khan, Kenneth G Cornetta, Michael Rothe, Axel Schambach, Donald B Kohn, Harry L Malech, R Scott McIvor, Jennifer M Puck, Morton J Cowan
During B and T lymphocyte maturation, V(D)J recombination is initiated by creation of DNA double-strand breaks. Artemis is an exonuclease essential for their subsequent repair by non-homologous end-joining. Mutations in DCLRE1C, the gene encoding Artemis, cause T-B-NK+ severe combined immunodeficiency (ART-SCID) and also confer heightened sensitivity to ionizing radiation and alkylating chemotherapy. Although allogeneic hematopoietic cell transplantation (HCT) can treat ART-SCID, conditioning regimens are poorly tolerated, leading to early mortality and/or late complications, including short stature, endocrinopathies, and dental aplasia...
September 9, 2016: Human Gene Therapy
Jason Arsenault, Shervin Gholizadeh, Yosuke Niibori, Laura K Pacey, Sebok K Halder, Enea Koxhioni, Ayumu Konno, Hirokazu Hirai, David R Hampson
Fragile X mental retardation protein (FMRP) is absent or highly reduced in Fragile X Syndrome, a genetic disorder causing cognitive impairment and autistic behaviors. Previous proof-of-principle studies have demonstrated that restoring FMRP in the brain using viral vectors can improve pathological abnormalities in mouse models of fragile X. However, unlike small molecule drugs where the dose can readily be adjusted during treatment, viral vector-based biological therapeutic drugs present challenges in terms of achieving optimal dosing and expression levels...
September 7, 2016: Human Gene Therapy
Xing Su, Wenjuan Chen, Shaowen Xiao, Xiaofan Li, Gang Xu, Jianji Pan, Shanwen Zhang
Detection of exogenous p53 gene and target gene expression in cervical cancer cell lines SiHa and C33A infected by rAd-p5 in vitro. The infection of rAd-p53 evidently increased the expression of exogenous p53 gene, p21 gene, and Bax gene. The radio-sensitization rate (SER) of rAd-p53 were 1.19 in SiHa cell and 1.18 in C33A cell in vitro, respectively. To evaluate the effect and safety of recombinant adenovirus-p53 (rAd-p53) transfer combined with radiotherapy (RT) in patients with cervical cancer. rAd-p53 transfer combined with radiotherapy ( group PRT ) in 69 patients with cervical cancer was compared with a control group treated with radiotherapy alone ( group RT ) in 35 patients with cervical cancer...
August 30, 2016: Human Gene Therapy
Ying Chen, Jiali Pu, Baorong Zhang
Neurodegenerative diseases are characterized by protein aggregation and progressive degeneration of neurons, causing severe functional deficiency in cognition, behavior, and movement. Until now, there has been no effective treatment available in the clinic. Considering the selective loss of specific neurons in the human brain in the pathogenesis of these diseases, generating functional neurons in vitro or in vivo to replace the lost neurons represents a novel strategy to treat neurodegenerative diseases. Human embryonic stem cells and induced pluripotent stem cells have good potential for cell replacement therapy...
August 26, 2016: Human Gene Therapy
Christoph Priesner, Krasimira Aleksandrova, Ruth Esser, Nadine Mockel-Tenbrinck, Jana Leise, Katharina Drechsel, Michael Marburger, Andrea Quaiser, Lilia Goudeva, Lubomir Arseniev, Andrew Kaiser, Wolfgang Glienke, Ulrike Koehl
Multiple clinical studies demonstrated that adaptive immunotherapy using redirected T cells against advanced cancer has led to promising results with improved survival of the patients. The continuously increasing interest in those advanced Gene Therapy Medicinal Products (GTMPs) leads to a challenge on the manufacturing side regarding automation, process robustness and cell storage. Therefore, our study addresses the proof of principle in clinical-scale selection, stimulation, transduction and expansion of T cells using the automated closed CliniMACS® Prodigy system...
August 25, 2016: Human Gene Therapy
Iiro Hassinen, Antti Kivelä, Antti Hedman, Antti Saraste, Juhani Knuuti, Juha Ek Hartikainen, Seppo Ylä-Herttuala
Cardiac gene transfer for the treatment of ischemic diseases has suffered from low gene transfer efficiency and inability to target treatment genes to the ischemic myocardium. We have developed a combined method based on electromechanical mapping and radiowater PET imaging to target gene therapy to viable but ischemic and hibernating areas of the myocardium. Electromechanical NOGA mapping produces 3D images of myocardium with both an electric activity map and a myocardial contractility map. These have been converted to 17-segment 2D bull's eye maps which were superimposed onto PET radiowater perfusion imaging maps of the myocardium...
August 23, 2016: Human Gene Therapy
Diane D'Allard, Johnson Liu
Diamond Blackfan anemia (DBA) is a well known inherited bone marrow failure syndrome mostly caused by mutations in ribosomal protein (RP) genes but also rarely in the hematopoietic transcription factor gene, <i>GATA1</i>, or <i>TSR2</i>, a ribosomal protein (Rps26) chaperone gene. About 25% of patients have heterozygous mutations in the <i>RPS19</i> gene, which leads to haploinsufficiency of Rps19 protein in most cases. However, some <i>RPS19</i> missense mutations appear to act in a dominant negative fashion...
August 22, 2016: Human Gene Therapy
Qizhao Wang, Jenni Firrman, Zhongren Wu, Katie A Pokiniewski, C Alexander Valencia, Hairong Wang, Hongying Wei, Zhenjing Zhuang, LinShu Liu, Stephanie L Wunder, Mario P S Chin, Ruian Xu, Yong Diao, Biao Dong, Weidong Xiao
Recombinant adeno-associated viral (rAAV) vectors have recently achieved clinical successes in human gene therapy. However, the commonly observed, heavier particles found in rAAV preparations have traditionally been ignored due to their reported low in vitro transduction efficiency. In this study, the biological properties of regular and high-density rAAV serotype 8 vectors, rAAV(RD) and rAAV(HD), were systemically compared. Results demonstrated that both rAAV(RD) and rAAV(HD) exhibited similar DNA packaging profiles, while rAAV(HD) capsids contained fewer VP1 and VP2 proteins, indicating that the rAAV(HD) particles contained a higher DNA/protein ratio than that of rAAV(RD) particles...
August 22, 2016: Human Gene Therapy
Shirley Hsin-Ju Mei, Claudia Dos Santos, Duncan Stewart
Given the failure of pharmacological interventions in acute respiratory distress syndrome (ARDS), researchers have been actively pursuing novel strategies to treat this devastating, life-threatening condition commonly seen in the Intensive Care Unit. There has been considerable research on harnessing the reparative properties of stem and progenitor cells to develop more effective therapeutic approaches for respiratory diseases with limited treatment options like ARDS. This review will discuss the preclinical literature on the use of stem and progenitor cell therapy and cell-based gene therapy for the treatment of preclinical animal models of acute lung injury (ALI)...
August 16, 2016: Human Gene Therapy
Wenpeng Li, Linjie Guo, Purva Rathi, Ekaterina Marinova, Xiuhua Gao, Meng-Feng Wu, Hao Liu, Gianpietro Dotti, Stephen Gottschalk, Leonid S Metelitsa, Andras Heczey
T cells engineered to express CD19-specific chimeric antigen receptors (CARs) have shown breakthrough clinical successes in patients with B-cell lymphoid malignancies. However, similar therapeutic efficacy of CAR T cells in solid tumors is yet to be achieved. In this study we systematically evaluated a series of CAR constructs targeting glypican-3 (GPC3), which is selectively expressed on several solid tumors. We compared GPC3-specific CARs that encoded CD3ζ (Gz) alone or with costimulatory domains derived from CD28 (G28z), 4-1BB (GBBz), or CD28 and 4-1BB (G28BBz)...
August 16, 2016: Human Gene Therapy
Maria Sofia Falzarano, Domenico D'Amario, Andrea Siracusano, Massimo Massetti, Antonio Amodeo, Federica La Neve, Camilla Reina Maroni, Eugenio Mercuri, Hana Osman, Chiara Scotton, Annarita Armaroli, Rachele Rossi, Rita Selvatici, Filippo Crea, Alessandra Ferlini
A ready source of autologous myogenic cells is of vital importance for drug screening and functional genetic studies in Duchenne Muscular Dystrophy (DMD), a rare disease caused by a variety of dystrophin gene mutations. As stem cells (SCs) can be easily and non-invasively obtained from urine specimens, we set out to determine whether they could be myogenic-induced and useful in DMD research. To this end, we isolated stem cells from the urine of two healthy donors and one patient with DMD, and performed surface-marker characterization, myogenic differentiation (MyoD), and then transfection with antisense oligoribonucletoides to test for exon skipping and protein restoration...
August 16, 2016: Human Gene Therapy
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"