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Human Gene Therapy. Clinical Development

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https://www.readbyqxmd.com/read/27855487/transfer-of-therapeutic-genes-into-fetal-rhesus-monkeys-using-recombinant-adeno-associated-type-i-viral-vectors
#1
Thomas J Conlon, Cathryn S Mah, Christina A R Pacak, Mary B Rucker Henninger, Kirsten E Erger, Marda L Jorgensen, Charles C Lee, Alice F Tarantal, Barry J Byrne
Neuromuscular disorders such as Pompe disease (glycogen storage disease, type II), result in early and potentially irreversible cellular damage with a very limited opportunity for intervention in the newborn period. Pompe disease is due to deficiency in acid α-glucosidase (GAA) leading to lysosomal accumulation of glycogen in all cell types, abnormal myofibrillogenesis, respiratory insufficiency, neurological deficits, and reduced contractile function in striated muscle. Previous studies have shown that fetal delivery of recombinant adeno-associated virus (rAAV) encoding GAA to the peritoneal cavity of Gaa-/- mice resulted in high-level transduction of the diaphragm...
November 17, 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27604429/safety-study-intraventricular-injection-of-a-modified-oncolytic-measles-virus-into-measles-immune-hcd46-transgenic-ifn%C3%AE-rko-mice
#2
Sangeet Lal, Kah-Whye Peng, Michael B Steele, Nathan Jenks, Hong Ma, Gary Kohanbash, Joanna J Phillips, Corey Raffel
The modified Edmonston vaccine strain of measles virus (MV) has shown potent oncolytic efficacy against various tumor types and is being investigated in clinical trials. Our laboratory showed that MV effectively kills medulloblastoma tumor cells in both localized disease and when tumor cells are disseminated through cerebrospinal fluid (CSF). Although the safety of repeated intracerebral injection of modified MV in rhesus macaques has been established, the safety of administering MV into CSF has not been adequately investigated...
September 7, 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27632772/gene-therapy-briefs
#3
Alex Philippidis
No abstract text is available yet for this article.
September 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27632771/investor-outlook-gene-therapy-picking-up-steam-at-a-crossroads
#4
Joshua Schimmer, Steven Breazzano
The gene therapy field continues to pick up steam with recent successes in a number of different therapeutic indications that highlight the potential for the platform. As the field continues to make progress, a growing data set of long-term safety and efficacy data will continue to define gene therapy's role, determining ultimately how widely it may be used beyond rare, serious diseases with high unmet needs. New technologies often take unanticipated twists and turns as patient exposure accumulates, and gene therapy may be no exception...
September 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27556334/luigi-naldini-on-his-lifelong-involvement-with-the-development-of-gene-therapy
#5
James M Wilson
No abstract text is available yet for this article.
September 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27549987/translation-and-reimbursement-the-twin-challenges-for-cell-and-gene-therapies-reflections-of-an-ex-regulator
#6
Gopalan Narayanan
No abstract text is available yet for this article.
September 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27532609/safety-studies-in-tumor-and-non-tumor-bearing-mice-in-support-of-clinical-trials-using-oncolytic-vsv-ifn%C3%AE-nis
#7
Lianwen Zhang, Michael B Steele, Nathan Jenks, Jacquelyn Grell, Lukkana Suksanpaisan, Shruthi Naik, Mark J Federspiel, Martha Q Lacy, Stephen J Russell, Kah-Whye Peng
Oncolytic VSV-IFNβ-NIS is selectively destructive to tumors. Here, we present the IND enabling preclinical rodent studies in support of clinical testing of vesicular stomatitis virus (VSV) as a systemic therapy. Efficacy studies showed dose-dependent tumor regression in C57BL/KaLwRij mice bearing syngeneic 5TGM1 plasmacytomas after systemic VSV administration. In contrast, the virus was effective at all doses tested against human KAS6/1 xenografts in SCID mice. Intravenous administration of VSV-mIFNβ-NIS is well tolerated in C57BL/6 mice up to 5 × 10(10) TCID50 (50% tissue culture infective dose)/kg with no neurovirulence, no cytokine storm, and no abnormalities in tissues...
September 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27470285/optimizing-transgene-configuration-and-protein-fusions-to-maximize-dopamine-production-for-the-gene-therapy-of-parkinson-s-disease
#8
Hannah J Stewart, G Scott Ralph, Liang Fong-Wong, Iain Strickland, Laura McCloskey, Lucy Barnes, Ian Blount, Owen Wells, Christelle J M Truran, Alan J Kingsman, Stéphane Palfi, Kyriacos A Mitrophanous
Pharmacological dopamine replacement therapies provide the most well-established treatments for Parkinson's disease (PD). However, these long-term treatments can lead to motor complications and off-target effects. ProSavin(®), a lentiviral vector (LV)-based gene therapy approach aimed at restoring local and continuous dopamine production, through delivery of three enzymes in the dopamine biosynthesis pathway, was demonstrated to be safe and well-tolerated in a phase I/II clinical study of patients with advanced PD...
September 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27763769/standing-on-the-shoulders-of-stem-cell-gene-therapists-history-hyperbole-and-hope-for-the-future
#9
Jason Gardner
No abstract text is available yet for this article.
August 30, 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27575553/standing-on-the-shoulders-of-stem-cell-gene-therapists-history-hyperbole-and-hope-for-the-future
#10
Jason Gardner
A new type of medicine was approved in Europe at the end of May that culminated from the successful convergence of two fields of science: stem cell transplantation and gene therapy. Strimvelis, a patient-specific gene-modified stem cell medicine for ADA-SCID (a fatal immunometabolic disorder similar to the bubble-boy disease), was developed by scientists at the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan, then later partnered with GSK. The journey took over 25 years of dedicated work from many groups and involved a pivotal trial with 12 children and their brave families...
August 30, 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27314914/aav-natural-infection-induces-broad-cross-neutralizing-antibody-responses-to-multiple-aav-serotypes-in-chimpanzees
#11
Roberto Calcedo, James M Wilson
Cross-sectional studies of primates have revealed that natural neutralizing antibody (NAb) responses to adeno-associated viruses (AAV) span multiple serotypes. This differs from the phenotype of the NAb response to an AAV vector delivered to seronegative nonhuman primates that is typically restricted to the administered AAV serotype. To better understand the mechanism by which natural AAV infections result in broad NAb responses, we conducted a longitudinal study spanning 10 years in which we evaluated serum-circulating AAV NAb levels in captive-housed chimpanzees...
June 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27314913/design-of-a-phase-i-clinical-trial-to-evaluate-m032-a-genetically-engineered-hsv-1-expressing-il-12-in-patients-with-recurrent-progressive-glioblastoma-multiforme-anaplastic-astrocytoma-or-gliosarcoma
#12
Daxa M Patel, Paul M Foreman, L Burt Nabors, Kristen O Riley, G Yancey Gillespie, James M Markert
M032 is a second-generation oncolytic herpes simplex virus (oHSV) that selectively replicates in tumor cells. M032 kills tumor cells directly through oncolytic replication and then proceeds to infect tumor cells in proximity, continuing the process of tumor destruction. In addition to this direct oncolytic activity, the virus carries a therapeutic payload-thus acting as a gene therapy vector-and causes the tumor cell to synthesize and secrete the immunity-stimulating protein interleukin-12 (IL-12) before cell death...
June 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27314912/measurement-challenges-for-car-t-biomanufacturing-highlights-from-a-meeting-sponsored-by-the-national-institute-of-standards-and-technology-nist
#13
Sheng Lin-Gibson, Kelley C Rogers, Anne L Plant
No abstract text is available yet for this article.
June 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27314911/recollections-from-a-pioneer-who-provided-the-foundation-for-the-success-of-gene-therapy-in-treating-severe-combined-immune-deficiencies
#14
James M Wilson
No abstract text is available yet for this article.
June 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27267566/sequenom-the-u-s-supreme-court-and-personalized-medicine
#15
Cathy A Kodroff
No abstract text is available yet for this article.
June 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27267410/gene-therapy-briefs
#16
(no author information available yet)
No abstract text is available yet for this article.
June 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27267267/investor-outlook-rising-from-the-ashes-gsk-s-european-approval-of-strimvelis-for-ada-scid
#17
Joshua Schimmer, Steven Breazzano
GlaxoSmithKline's (GSK) and partner San Raffaele Telethon Institute for Gene Therapy's recent positive European approval for Strimvelis for treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) represents the second EU-approved gene therapy and the first γ-retrovirus and first ex vivo gene therapy. In this article we discuss the significance and implications of this historic approval for the broader gene therapy field.
June 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27247123/aav-natural-infection-induces-broad-cross-neutralizing-antibody-responses-to-multiple-aav-serotypes-in-chimpanzees
#18
Roberto Calcedo, James M Wilson
Cross-sectional studies of primates have revealed that natural neutralizing antibody (NAb) responses to adeno-associated viruses (AAV) span multiple serotypes. This differs from the phenotype of the NAb response to an AAV vector delivered to sero-negative nonhuman primates which is typically restricted to the administered AAV serotype. To better understand the mechanism by which natural AAV infections result in broad NAb responses, we conducted a longitudinal study spanning 10 years in which we evaluated serum-circulating AAV NAb levels in captive-housed chimpanzees...
June 1, 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27231756/design-of-a-phase-i-clinical-trial-to-evaluate-m032-a-genetically-engineered-hsv-1-expressing-il-12-in-patients-with-recurrent-progressive-glioblastoma-multiforme-anaplastic-astrocytoma-or-gliosarcoma
#19
Daxa Patel, Paul Foreman, Burt Nabors, Kristen Riley, Yancey Gillespie, James Markert
M032 is a second-generation oncolytic herpes simplex virus (oHSV) that selectively replicates in tumor cells. M032 kills tumor cells directly through oncolytic replication then proceeds to infect tumor cells in proximity, continuing the process of tumor destruction. In addition to this direct oncolytic activity, the virus carries a therapeutic payload-thus acting as a gene therapy vector-and causes the tumor cell to synthesize and secrete the immunity-stimulating protein interleukin-12 (IL-12) prior to cell death...
May 27, 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/26956923/safety-and-biodistribution-evaluation-in-cynomolgus-macaques-of-raav2tyf-pr1-7-hcngb3-a-recombinant-aav-vector-for-treatment-of-achromatopsia
#20
Guo-Jie Ye, Ewa Budzynski, Peter Sonnentag, T Michael Nork, Paul E Miller, Alok K Sharma, James N Ver Hoeve, Leia Smith, Tara Arndt, Roberto Calcedo, Chantelle Gaskin, Paulette Robinson, David R Knop, William W Hauswirth, Jeffrey David Chulay
AGTC is developing rAAV2tYF-PR1.7-hCNGB3, a recombinant adeno-associated virus (rAAV) vector expressing the human CNGB3 gene, for treatment of achromatopsia, an inherited retinal disorder characterized by markedly reduced visual acuity, extreme light sensitivity and absence of color discrimination. We report here results of a study evaluating the safety and biodistribution of rAAV2tYF-PR1.7-hCNGB3 in cynomolgus macaques. Three groups of animals (n=2 males and 2 females per group) received a subretinal injection in one eye of 300 µL containing either vehicle or rAAV2tYF-PR1...
March 8, 2016: Human Gene Therapy. Clinical Development
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