journal
MENU ▼
Read by QxMD icon Read
search

Human Gene Therapy. Clinical Development

journal
https://www.readbyqxmd.com/read/30226396/a-phase-i-trial-of-oncolytic-adenovirus-icovir-5-administered-intravenously-to-melanoma-patients
#1
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
https://www.readbyqxmd.com/read/30230938/the-rac-retires-after-a-job-well-done
#2
James M Wilson
No abstract text is available yet for this article.
September 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/30230937/gene-therapy-briefs
#3
(no author information available yet)
No abstract text is available yet for this article.
September 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/30216089/university-flunk-out-to-genomics-pioneer-an-interview-with-george-church-phd
#4
James M Wilson
No abstract text is available yet for this article.
September 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/30187779/development-of-methodology-and-study-protocol-safety-and-efficacy-of-a-single-subretinal-injection-of-raav-hcnga3-in-patients-with-cnga3-linked-achromatopsia-investigated-in-an-exploratory-dose-escalation-trial
#5
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
https://www.readbyqxmd.com/read/30180756/mirna-451-inhibits-glioma-cell-proliferation-and-invasion-through-the-mtor-hif-1%C3%AE-vegf-signaling-pathway-by-targeting-cab39
#6
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
https://www.readbyqxmd.com/read/30156440/long-term-follow-up-of-a-phase-i-ii-study-of-prosavin-a-lentiviral-vector-gene-therapy-for-parkinson-s-disease
#7
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
https://www.readbyqxmd.com/read/29869533/the-current-state-of-advanced-therapy-medicinal-products-in-the-czech-republic
#8
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
https://www.readbyqxmd.com/read/29869540/gene-therapy-for-osteoarthritis-pharmacokinetics-of-intra-articular-self-complementary-adeno-associated-virus-interleukin-1-receptor-antagonist-delivery-in-an-equine-model
#9
Rachael S Watson Levings, Ted A Broome, Andrew D Smith, Brett L Rice, Eric P Gibbs, David A Myara, E Viktoria Hyddmark, Elham Nasri, Ali Zarezadeh, Padraic P Levings, Yuan Lu, Margaret E White, E Anthony Dacanay, Gregory B Foremny, Christopher H Evans, Alison J Morton, Mathew Winter, Michael J Dark, David M Nickerson, Patrick T Colahan, Steven C Ghivizzani
Toward the treatment of osteoarthritis (OA), the authors have been investigating self-complementary adeno-associated virus (scAAV) for intra-articular delivery of therapeutic gene products. As OA frequently affects weight-bearing joints, pharmacokinetic studies of scAAV gene delivery were performed in the joints of the equine forelimb to identify parameters relevant to clinical translation in humans. Using interleukin-1 receptor antagonist (IL-1Ra) as a secreted therapeutic reporter, scAAV vector plasmids containing codon-optimized cDNA for equine IL-1Ra (eqIL-1Ra) were generated, which produced eqIL-1Ra at levels 30- to 50-fold higher than the native sequence...
June 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29869535/self-complementary-adeno-associated-virus-mediated-interleukin-1-receptor-antagonist-gene-delivery-for-the-treatment-of-osteoarthritis-test-of-efficacy-in-an-equine-model
#10
Rachael S Watson Levings, Andrew D Smith, Ted A Broome, Brett L Rice, Eric P Gibbs, David A Myara, E Viktoria Hyddmark, Elham Nasri, Ali Zarezadeh, Padraic P Levings, Yuan Lu, Margaret E White, E Anthony Dacanay, Gregory B Foremny, Christopher H Evans, Alison J Morton, Mathew Winter, Michael J Dark, David M Nickerson, Patrick T Colahan, Steven C Ghivizzani
The authors are investigating self-complementary adeno-associated virus (scAAV) as a vector for intra-articular gene-delivery of interleukin-1 receptor antagonist (IL-1Ra), and its therapeutic capacity in the treatment of osteoarthritis (OA). To model gene transfer on a scale proportional to the human knee, a frequent site of OA incidence, studies were focused on the joints of the equine forelimb. Using AAV2.5 capsid and equine IL-1Ra as a homologous transgene, a functional ceiling dose of ∼5 × 1012 viral genomes was previously identified, which elevated the steady state levels of eqIL-1Ra in synovial fluids by >40-fold over endogenous production for at least 6 months...
June 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29664709/non-clinical-efficacy-and-safety-studies-on-g1xcgd-a-lentiviral-vector-for-ex-vivo-gene-therapy-of-x-linked-chronic-granulomatous-disease
#11
Christian Brendel, Michael Rothe, Giorgia Santilli, Sabine Charrier, Stefan Stein, Hana Kunkel, Daniela Abriss, Uta Müller-Kuller, Bobby Gaspar, Ute Modlich, Anne Galy, Axel Schambach, Adrian J Thrasher, Manuel Grez
Chronic granulomatous disease (CGD) is a debilitating primary immunodeficiency affecting phagocyte function due to the absence of nicotinamide dinucleotide phosphate (NADPH) oxidase activity. The vast majority of CGD patients in the Western world have mutations within the X-linked CYBB gene encoding for gp91phox (NOX2), the redox center of the NADPH oxidase complex (XCGD). Current treatments of XCGD are not entirely satisfactory, and prior attempts at autologous gene therapy using gammaretrovirus vectors did not provide long-term curative effects...
June 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29624465/practical-implications-of-factor-ix-gene-transfer-for-individuals-with-hemophilia-b-a-clinical-perspective
#12
Wolfgang Miesbach, Eileen K Sawyer
Gene therapy for severe hemophilia is on the cusp of entering clinical practice. However, there is limited clinical experience in this area given that gene transfer is a relatively recent technology. Therefore, this clinical perspective article will review the evidence supporting gene therapy in this field, examine ways to open a dialogue about gene therapy with patients in the clinic setting, and present a case of a participant in a recent clinical trial of gene therapy for hemophilia. Clinical trials in hemophilia using adeno-associated virus (AAV) vectors to transfer functional factor IX (FIX) have reported increases in FIX activity to functional levels, reduced bleed frequency, and a lessening or abrogation of the need for costly FIX replacement...
June 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29641281/a-multicenter-double-blind-phase-iii-clinical-trial-to-evaluate-the-efficacy-and-safety-of-a-cell-and-gene-therapy-in-knee-osteoarthritis-patients
#13
Myung-Ku Kim, Chul-Won Ha, Yong In, Sung-Do Cho, Eui-Sung Choi, Jeong-Ku Ha, Ju-Hong Lee, Jae-Doo Yoo, Seong-Il Bin, Choong-Hyeok Choi, Hee-Soo Kyung, Myung-Chul Lee
The aim of this study was to test the clinical efficacy of TissueGene-C (TG-C), a cell and gene therapeutic for osteoarthritis consisting of non-transformed and transduced chondrocytes (3:1) retrovirally transduced to overexpress transforming growth factor-β1. A total of 163 Kellgren-Lawrence grade 3 patients with knee osteoarthritis were randomly assigned to receive intra-articular TG-C or placebo. Primary efficacy measures included criteria for subjective assessment by International Knee Documentation Committee (IKDC) and pain severity by Visual Analog Scale (VAS) for 52 weeks...
March 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29641280/gene-therapy-briefs
#14
Alex Philippidis
No abstract text is available yet for this article.
March 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29641279/interview-with-jean-bennett-md-phd
#15
James M Wilson
No abstract text is available yet for this article.
March 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29624457/low-seroprevalence-of-neutralizing-antibodies-targeting-two-clade-f-aav-in-humans
#16
Jeff L Ellsworth, Michael O'Callaghan, Hillard Rubin, Albert Seymour
To assess the therapeutic utility of AAVHSC15 and AAVHSC17, two recently described Clade F adeno-associated viruses (AAVs), the seroprevalence of neutralizing antibodies (NAbs) to these AAVs was assessed in a representative human population and compared to that of AAV9. NAb levels were measured in 100 unique human sera of different races (34, Black, 33 Caucasian, and 33 Hispanic) and sex (49% female, 51% male) collected within the United States. Fifty-six sera were tested in Huh7 cells and 44 sera were tested in 2V6...
March 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29409358/aavrh-10-mediated-apoe2-central-nervous-system-gene-therapy-for-apoe4-associated-alzheimer-s-disease
#17
Jonathan B Rosenberg, Michael G Kaplitt, Bishnu P De, Alvin Chen, Thomas Flagiello, Christiana Salami, Eduard Pey, Lingzhi Zhao, Rodolfo J Ricart Arbona, Sebastien Monette, Jonathan P Dyke, Douglas J Ballon, Stephen M Kaminsky, Dolan Sondhi, Gregory A Petsko, Steven M Paul, Ronald G Crystal
Alzheimer's disease (AD) is a progressive degenerative neurological disorder affecting nearly one in nine elderly people in the United States. Population studies have shown that an inheritance of the apolipoprotein E (APOE) variant APOE4 allele increases the risk of developing AD, whereas APOE2 homozygotes are protected from late-onset AD. It was hypothesized that expression of the "protective" APOE2 variant by genetic modification of the central nervous system (CNS) of APOE4 homozygotes could reverse or prevent progressive neurologic damage...
March 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29228803/in-vitro-human-placental-studies-to-support-adenovirus-mediated-vegf-d-%C3%AE-n%C3%AE-c-maternal-gene-therapy-for-the-treatment-of-severe-early-onset-fetal-growth-restriction
#18
Michelle Desforges, Alexandra Rogue, Nick Pearson, Carlo Rossi, Elena Olearo, Roy Forster, Mark Lees, Neil J Sebire, Susan L Greenwood, Colin P Sibley, Anna L David, Paul Brownbill
Severe fetal growth restriction (FGR) affects 1 in 500 pregnancies, is untreatable, and causes serious neonatal morbidity and death. Reduced uterine blood flow (UBF) is one cause. Transduction of uterine arteries in normal and FGR animal models using an adenovirus (Ad) encoding VEGF isoforms increases UBF and improves fetal growth in utero. Understanding potential adverse consequences of this therapy before first-in-woman clinical application is essential. The aims of this study were to determine whether Ad...
March 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29486610/a-multicenter-double-blind-phase-iii-clinical-trial-to-evaluate-the-efficacy-and-safety-of-a-cell-and-gene-therapy-in-knee-osteoarthritis-patients
#19
Myung-Ku Kim, Chul-Won Ha, Yong In, Sung-Do Cho, Eui-Sung Choi, Jeong-Ku Ha, Ju-Hong Lee, Jae-Doo Yoo, Seong-Il Bin, Choong-Hyeok Choi, Hee-Soo Kyung, Myung-Chul Lee
OBJECTIVE: To test the clinical efficacy of TissueGene-C (TG-C), a cell and gene therapeutic for osteoarthritis consisting of non-transformed and transduced chondrocytes (3:1), retrovirally transduced to overexpress TGF-β1. DESIGN: We randomly assigned 163 with knee osteoarthritis to receive intra-articular TG-C or placebo in Kellgren-Lawrence grade 3 patients for clinical trial. Primary efficacy measures included criteria for subjective assessment by International Knee Documentation Committee (IKDC) and pain severity by Visual Analog Scale (VAS) for 52 weeks...
February 27, 2018: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29486604/low-seroprevalence-of-neutralizing-antibodies-targeting-two-clade-f-aav-in-humans
#20
Jeff L Ellsworth, Michael OCallaghan, Hillard Rubin, Albert Seymour
To assess the therapeutic utility of AAVHSC15 and AAVHSC17, two recently described Clade F adeno-associated viruses, the seroprevalence of neutralizing antibodies to these AAVs was assessed in a representative human population and compared to that of AAV9. Neutralizing antibody levels were measured in 100 unique human sera of different races (34:33:33, Black:Caucasian:Hispanic) and sex (49% Female, 51% Male) collected within the United States. Fifty-six sera were tested in HuH7 cells and 44 sera were tested in 2V6...
February 27, 2018: Human Gene Therapy. Clinical Development
journal
journal
47666
1
2
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"