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Human Gene Therapy. Clinical Development

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https://www.readbyqxmd.com/read/29131688/carl-june-speaks-of-his-pioneering-efforts-that-led-to-the-first-food-and-drug-administration-approved-gene-therapy-product
#1
James M Wilson
No abstract text is available yet for this article.
November 13, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29130351/the-gene-therapy-resource-program-a-decade-of-dedication-to-translational-research-by-the-national-heart-lung-and-blood-institute
#2
Terence R Flotte, Eric Daniels, Janet Benson, Jenee M Bevett-Rose, Kenneth Cornetta, Margaret Diggins, Julie Johnston, Susan Sepelak, Johannes Van Der Loo, James M Wilson, Cheryl L McDonald
Over a ten-year period, the Gene Therapy Resource Program (GTRP) of the National Heart Lung and Blood Institute (NHLBI) has provided a set of core services to investigators to facilitate the clinical translation of gene therapy. These services have included a preclinical (research-grade) vector production core; current Good Manufacturing Practice (cGMP) clinical-grade vector cores for recombinant adeno-associated virus (rAAV) and lentivirus vectors; a pharmacology and toxicology core; and a coordinating center to manage program logistics and to provide regulatory and financial support to early phase clinical trials...
November 12, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29064732/differential-prevalence-of-antibodies-against-adeno-associated-virus-in-healthy-children-and-patients-with-mucopolysaccharidosis-iii-perspective-for-aav-mediated-gene-therapy
#3
Haiyan Fu, Aaron S Meadows, Ricardo J Pineda, Krista L Kunkler, Kristen V Truxal, Kim L McBride, Kevin M Flanigan, Douglas M McCarty
Recombinant adeno-associated virus (AAV) vectors are promising gene therapy tools. However, pre-existing antibodies (Abs) to many useful AAV serotypes pose a critical challenge for the translation of gene therapies. As part of AAV gene therapy program for treating mucopolysaccharidosis (MPS) III patients, the seroprevalence profiles of AAV1-9 and rh74 were investigated in MPS IIIA/IIIB patients and in healthy children. Using enzyme-linked immunosorbent assay for αAAV-IgG, significantly higher seroprevalence was observed for AAV1 and AAVrh74 in 2- to 7-year-old MPS III patients than in healthy controls...
October 24, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29020838/safety-and-efficacy-of-aav5-vectors-expressing-human-or-canine-cngb3-in-cngb3-mutant-dogs
#4
Guo-Jie Ye, András M Komáromy, Caroline Zeiss, Roberto Calcedo, Christine D Harman, Kristin L Koehl, Gabriel A Stewart, Simone Iwabe, Vince A Chiodo, William W Hauswirth, Gustavo D Aguirre, Jeffrey D Chulay
Achromatopsia is an inherited retinal disorder of cone photoreceptors characterized by markedly reduced visual acuity, extreme light sensitivity, and absence of color discrimination. Approximately 50% of cases are caused by mutations in the cone photoreceptor-specific cyclic nucleotide gated channel beta subunit (CNGB3) gene. Studies in CNGB3-mutant dogs showed that subretinal injection of an AAV vector expressing human CNGB3, which has 76% amino acid identity with canine CNGB3, driven by a 2.1 kb human red cone opsin promoter (PR2...
October 11, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28922943/differential-prevalence-of-antibodies-against-adeno-associated-virus-in-healthy-children-and-patients-with-mucopolysaccharidosis-iii-perspective-for-aav-mediated-gene-therapy
#5
Haiyan Fu, Aaron S Meadows, Ricardo J Pineda, Krista L Kunkler, Kristen V Truxal, Kim L McBride, Kevin Flanigan, Douglas M McCarty
Recombinant AAV vectors are promising gene therapy tools. However, pre-existing antibodies (Abs) to many useful AAV serotypes pose a critical challenge for the translation of gene therapies. As part of AAV gene therapy program for treating MPS III patients, we investigated the seroprevalence profiles of AAV1-9 and rh74 in MPS IIIA/IIIB patients and in healthy children. Using ELISA for αAAV-IgG, we observed significantly higher sero-prevalence for AAV1 and AAVrh74 in 2-7y-old MPS III patients than in healthy controls...
September 19, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28915081/the-story-of-rna-interference-as-a-new-therapeutic-paradigm-from-nobel-laureate-craig-mello
#6
James M Wilson
No abstract text is available yet for this article.
September 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28876135/gene-therapy-briefs
#7
Alex Philippidis
No abstract text is available yet for this article.
September 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28726496/a-preclinical-study-in-rhesus-macaques-for-cystic-fibrosis-to-assess-gene-transfer-and-transduction-by-aav1-and-aav5-with-a-dual-luciferase-reporter-system
#8
William B Guggino, Janet Benson, JeanClare Seagrave, Ziying Yan, John Engelhardt, Guangping Gao, Thomas J Conlon, Liudmila Cebotaru
Cystic fibrosis (CF) is an autosomal recessive disease that is potentially treatable by gene therapy. Since the identification of the gene encoding CF transmembrane conductance regulator, a number of preclinical and clinical trials have been conducted using the first generation of adeno-associated virus, AAV2. All these studies showed that AAV gene therapy for CF is safe, but clinical benefit was not clearly demonstrated. Thus, a new generation of AAV vectors based on other serotypes is needed to move the field forward...
September 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28726495/use-of-adeno-associated-virus-vector-for-cardiac-gene-delivery-in-large-animal-surgical-models-of-heart-failure
#9
Michael G Katz, Anthony S Fargnoli, Thomas Weber, Roger J Hajjar, Charles R Bridges
The advancement of gene therapy-based approaches to treat heart disease represents a need for clinically relevant animal models with characteristics equivalent to human pathologies. Rodent models of cardiac disease do not precisely reproduce heart failure phenotype and molecular defects. This has motivated researchers to use large animals whose heart size and physiological processes more similar and comparable to those of humans. Today, adeno-associated viruses (AAV)-based vectors are undoubtedly among the most promising DNA delivery vehicles...
September 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28510497/clinical-development-and-commercialization-of-advanced-therapy-medicinal-products-in-the-european-union-how-are-the-product-pipeline-and-regulatory-framework-evolving
#10
Tomáš Boráň, Margarida Menezes-Ferreira, Ilona Reischl, Patrick Celis, Nicolas Ferry, Bernd Gänsbacher, Hartmut Krafft, Michele Lipucci di Paola, Dariusz Sladowski, Paula Salmikangas
The research and development of advanced therapy medicinal products (ATMPs) has been active in Europe and worldwide during recent years. Yet, the number of licensed products remains low. The main expected legal change in the near future in the European Union (EU) concerns the regulation on clinical trials (536/2014), which will come into force in 2018. With this new framework, a more harmonized and swift process for approval of clinical trials is anticipated, which is expected to support the entry of new innovations into the EU market...
September 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28447889/bone-marrow-as-a-hematopoietic-stem-cell-source-for-gene-therapy-in-sickle-cell-disease-evidence-from-rhesus-and-scd-patients
#11
Naoya Uchida, Atsushi Fujita, Matthew M Hsieh, Aylin C Bonifacino, Allen E Krouse, Mark E Metzger, Robert E Donahue, John F Tisdale
Steady state bone marrow (BM) is the preferred hematopoietic stem cell (HSC) source for gene therapy in sickle cell disease (SCD) due to the recognized risk of vaso-occlusive crisis during granulocyte colony-stimulating factor mobilization. We previously established clinically relevant HSC gene transfer in the rhesus model following transplantation of mobilized peripheral blood (PB) CD34(+) cells transduced with lentiviral vectors. In this study, we examined steady state bone marrow (BM) in the rhesus competitive repopulation model and demonstrate similar gene marking in vitro and in vivo, as compared with mobilized PB CD34(+) cells...
September 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28609190/gene-therapy-briefs
#12
Alex Philippidis
No abstract text is available yet for this article.
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28609189/a-new-kid-on-the-playground-of-crispr-dmd-therapy
#13
Dongsheng Duan
No abstract text is available yet for this article.
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28537450/investor-outlook-the-unanswered-questions
#14
Joshua Schimmer, Steven Breazzano
The year 2016 was an exciting one for the field, with several notable successes outweighing a few setbacks. As the number of patients treated successfully (and safely) with gene therapy grows, the totality of evidence points to a robust platform with utility in orphan/ultra-orphan diseases as well as broader indications, and with hopefully increasing predictability of results. This year promises to feature more patients treated, more clinical data, and more gene therapy products in registration-enabling studies...
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28530842/the-past-present-and-future-of-gene-therapy-from-nobel-laureate-david-baltimore
#15
James M Wilson
No abstract text is available yet for this article.
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28514874/oncolytic-recombinant-vesicular-stomatitis-virus-vsv-is-nonpathogenic-and-nontransmissible-in-pigs-a-natural-host-of-vsv
#16
Lauro Velazquez-Salinas, Shruthi Naik, Steven J Pauszek, Kah-Whye Peng, Stephen J Russell, Luis L Rodriguez
Vesicular stomatitis virus (VSV) is a negative-stranded RNA virus that naturally causes disease in livestock including horses, cattle and pigs. The two main identified VSV serotypes are New Jersey (VSNJV) and Indiana (VSIV). VSV is a rapidly replicating, potently immunogenic virus that has been engineered to develop novel oncolytic therapies for cancer treatment. Swine are a natural host for VSV and provide a relevant and well-established model, amenable to biological sampling to monitor virus shedding and neutralizing antibodies...
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28478700/safety-and-efficacy-evaluation-of-raav2tyf-pr1-7-hcnga3-vector-delivered-by-subretinal-injection-in-cnga3-mutant-achromatopsia-sheep
#17
Elisha Gootwine, Ron Ofri, Eyal Banin, Alexey Obolensky, Edward Averbukh, Raaya Ezra-Elia, Maya Ross, Hen Honig, Alexander Rosov, Esther Yamin, Guo-Jie Ye, David R Knop, Paulette M Robinson, Jeffrey D Chulay, Mark S Shearman
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector expressing the human CNGA3 gene designated AGTC-402 (rAAV2tYF-PR1.7-hCNGA3) for the treatment of achromatopsia, an inherited retinal disorder characterized by markedly reduced visual acuity, extreme light sensitivity, and absence of color discrimination. The results are herein reported of a study evaluating safety and efficacy of AGTC-402 in CNGA3-deficient sheep. Thirteen day-blind sheep divided into three groups of four or five animals each received a subretinal injection of an AAV vector expressing a CNGA3 gene in a volume of 500 μL in the right eye...
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28410447/correction-to-hum-gene-ther-clin-devel-2017-28-1-17-27
#18
(no author information available yet)
No abstract text is available yet for this article.
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28363269/establishing-a-large-animal-model-for-in-vivo-reprogramming-of-bile-duct-cells-into-insulin-secreting-cells-to-treat-diabetes
#19
Caitlin M Hill, Anannya Banga, Juan E Abrahante, Ce Yuan, Lucas A Mutch, Jody Janecek, Timothy O'Brien, Melanie L Graham, James R Dutton
Type 1 diabetes manifests as autoimmune destruction of beta cells requiring metabolic management with an exogenous replacement of insulin, either by repeated injection of recombinant insulin or by transplantation of allogeneic islets from cadaveric donors. Both of these approaches have severe limitations. Repeated insulin injection requires intensive blood glucose monitoring, is expensive, and is associated with decreased quality-of-life measures. Islet transplantation, while highly effective, is severely limited by shortage of donor organs...
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28335614/therapeutic-targeting-of-protein-kinase-ck2-gene-expression-in-feline-oral-squamous-cell-carcinoma-a-naturally-occurring-large-animal-model-of-head-and-neck-cancer
#20
Claire M Cannon, Janeen H Trembley, Betsy T Kren, Gretchen M Unger, M Gerard O'Sullivan, Ingrid Cornax, Jaime F Modiano, Khalil Ahmed
Protein kinase CK2 (CK2) is a highly promising target for cancer therapy, and anti-CK2 gene expression therapy has shown effectiveness in rodent models of human head and neck cancer (HNC). To date, there has been no large-animal model of cancer in which to further explore anti-CK2 therapies. Feline oral squamous cell carcinoma (FOSCC) has been proposed as a large-animal model for human HNC, and we have previously shown that CK2 is a rational target in FOSCC. Here we have tested the hypothesis that a novel tenfibgen-coated tumor-specific nanocapsule carrying RNA interference (RNAi) oligonucleotides targeting feline CK2α and CK2α' (TBG-RNAi-fCK2αα') would be safe in cats with FOSCC; assessment of target inhibition and tumor response were secondary aims...
June 2017: Human Gene Therapy. Clinical Development
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