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Human Gene Therapy. Clinical Development

journal
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
#1
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...
May 16, 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
#2
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...
April 17, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28609190/gene-therapy-briefs
#3
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
#4
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
#5
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
#6
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
#7
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
#8
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
#9
(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
#10
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
#11
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
https://www.readbyqxmd.com/read/28285544/improvement-of-adeno-associated-virus-mediated-liver-transduction-efficacy-by-regional-administration-in-macaca-fascicularis
#12
Nerea Zabaleta, David Salas, Maria Paramo, Mirja Hommel, Valerie Sier-Ferreira, Ruben Hernandez-Alcoceba, Jesus Prieto, Jose I Bilbao, Gloria Gonzalez-Aseguinolaza
The liver is a central organ in metabolism and can be affected by numerous inherited metabolic disorders. Recombinant adeno-associated virus (AAV)-based gene therapy represents a promising therapeutic approach for such diseases. AAVs have been demonstrated to be safe, and resulted in high and long-term expression in preclinical and clinical studies. However, there are still some concerns regarding the expression levels that can be achieved and the percentage of hepatocytes that can be transduced. Because of the cell-autonomous nature of most metabolic liver disorders, a high percentage of hepatocytes needs to be corrected in order to achieve a therapeutic effect...
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28279081/persistent-expression-of-dopamine-synthesizing-enzymes-15-years-after-gene-transfer-in-a-primate-model-of-parkinson-s-disease
#13
Yoshihide Sehara, Ken-Ichi Fujimoto, Kunihiko Ikeguchi, Yuko Katakai, Fumiko Ono, Naomi Takino, Mika Ito, Keiya Ozawa, Shin-Ichi Muramatsu
Restoring dopamine production in the putamen through gene therapy is a straightforward strategy for ameliorating motor symptoms for Parkinson's disease (PD). In a 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP) toxicity-based primate model of PD, we previously showed the safety and efficacy of adeno-associated viral (AAV) vector-mediated gene delivery to the putamen of three dopamine-synthesizing enzymes (tyrosine hydroxylase [TH], aromatic l-amino acid decarboxylase [AADC], and guanosine triphosphate cyclohydrolase I [GCH]) up to 10 months postprocedure...
June 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319449/non-clinical-study-examining-aav8-tbg-hldlr-vector-associated-toxicity-in-chow-fed-wild-type-and-ldlr-rhesus-macaques
#14
Jenny A Greig, Maria P Limberis, Peter Bell, Shu-Jen Chen, Roberto Calcedo, Daniel J Rader, James M Wilson
Vectors based on adeno-associated virus serotype 8 (AAV8) have been evaluated in several clinical trials of gene therapy for hemophilia B with encouraging results. In preparation for a Phase 1 clinical trial of AAV8 gene therapy for the treatment of homozygous familial hypercholesterolemia (HoFH), the safety of the clinical candidate vector, AAV8.TBG.hLDLR, was evaluated in wild-type rhesus macaques and macaques heterozygous for a nonsense mutation in the low-density lipoprotein receptor (LDLR) gene (LDLR(+/-))...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319448/rationale-and-design-of-a-phase-1-clinical-trial-to-evaluate-hsv-g207-alone-or-with-a-single-radiation-dose-in-children-with-progressive-or-recurrent-malignant-supratentorial-brain-tumors
#15
Alicia M Waters, James M Johnston, Alyssa T Reddy, John Fiveash, Avi Madan-Swain, Kara Kachurak, Asim K Bag, G Yancey Gillespie, James M Markert, Gregory K Friedman
Primary central nervous system tumors are the most common solid neoplasm of childhood and the leading cause of cancer-related death in pediatric patients. Survival rates for children with malignant supratentorial brain tumors are poor despite aggressive treatment with combinations of surgery, radiation, and chemotherapy, and survivors often suffer from damaging lifelong sequelae from current therapies. Novel innovative treatments are greatly needed. One promising new approach is the use of a genetically engineered, conditionally replicating herpes simplex virus (HSV) that has shown tumor-specific tropism and potential efficacy in the treatment of malignant brain tumors...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319447/gene-therapy-briefs
#16
Alex Philippidis
No abstract text is available yet for this article.
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319446/good-laboratory-practice-preclinical-safety-studies-for-gsk2696273-mlv-vector-based-ex-vivo-gene-therapy-for-adenosine-deaminase-deficiency-severe-combined-immunodeficiency-in-nsg-mice
#17
Nicola Carriglio, Jan Klapwijk, Raisa Jofra Hernandez, Michela Vezzoli, Franck Chanut, Rhiannon Lowe, Draghici Elena, Melanie Nord, Paola Albertini, Patrizia Cristofori, Jane Richards, Hazel Staton, Jonathan Appleby, Alessandro Aiuti, Aisha V Sauer
GSK2696273 (autologous CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] enzyme) is a gamma-retroviral ex vivo gene therapy of bone marrow-derived CD34+ cells for the treatment of adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID). ADA-SCID is a severe monogenic disease characterized by immunologic and nonimmunologic symptoms. Bone-marrow transplant from a matched related donor is the treatment of choice, but it is available for only a small proportion of patients...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319445/nonclinical-pharmacology-toxicology-study-of-aav8-tbg-mldlr-and-aav8-tbg-hldlr-in-a-mouse-model-of-homozygous-familial-hypercholesterolemia
#18
Jenny A Greig, Maria P Limberis, Peter Bell, Shu-Jen Chen, Roberto Calcedo, Daniel J Rader, James M Wilson
The homozygous form of familial hypercholesterolemia (HoFH) is an excellent model for developing in vivo gene therapy in humans. The success of orthotropic liver transplantation in correcting the metabolic abnormalities in HoFH suggests that the correction of low-density lipoprotein receptor (LDLR) expression in hepatocytes via gene therapy should be sufficient for therapeutic efficacy. Vectors based on adeno-associated virus serotype 8 (AAV8) have been previously developed for liver-targeted gene therapy of a number of genetic diseases, including HoFH...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28263671/jurassic-park-gene-therapy-and-neuroscience-an-interview-with-feng-zhang-phd
#19
James M Wilson
No abstract text is available yet for this article.
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28106474/correction-to-hum-gene-ther-clin-devel-2016-27-4-123-126
#20
(no author information available yet)
No abstract text is available yet for this article.
March 2017: Human Gene Therapy. Clinical Development
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