journal
MENU ▼
Read by QxMD icon Read
search

Human Gene Therapy. Clinical Development

journal
https://www.readbyqxmd.com/read/28106474/correction-to-hum-gene-ther-clin-devel-2016-27-4-123-126
#1
(no author information available yet)
No abstract text is available yet for this article.
January 20, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27983892/investor-outlook-solving-gene-therapy-pricing%C3%A2-with-a-cures-voucher
#2
Joshua Schimmer, Steven Breazzano
Gene therapy reimbursement continues to be an intense topic of discussion in the field given the unique and durable benefits from a single administration and generally small patient populations against a reimbursement framework that is not optimized for such "cures" or long-lived benefits. As more gene therapy programs enter the market and late-stage development, it is increasingly important for the field to define a reimbursement model that works for all stakeholders in order to encourage the next wave of innovation...
December 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27983891/user-friendly-technology-the-key-to-gene-editing-s-bloom-market-for-gene-editing-tools-estimated-at-608m-and-growing-as-new-applications-are-found
#3
Bruce Carlson
No abstract text is available yet for this article.
December 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27983890/gene-therapy-briefs
#4
Alex Philippidis
No abstract text is available yet for this article.
December 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27983889/interview-with-katherine-a-high-md
#5
James M Wilson
No abstract text is available yet for this article.
December 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27855487/transfer-of-therapeutic-genes-into-fetal-rhesus-monkeys-using-recombinant-adeno-associated-type-i-viral-vectors
#6
Thomas J Conlon, Cathryn S Mah, Christina A Pacak, Mary B Rucker Henninger, Kirsten E Erger, Marda L Jorgensen, C Chang I 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...
December 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
#7
Jason Gardner
A new type of medicine approved in Europe at the end of May represents the culmination of 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 (adenosine deaminase deficiency leading to severe combined immunodeficiency; 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, Italy, which then later partnered with GlaxoSmithKline (GSK, Brentford, UK)...
December 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
#8
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...
December 2016: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/27632772/gene-therapy-briefs
#9
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
#10
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
#11
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
#12
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
#13
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
#14
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/27575553/standing-on-the-shoulders-of-stem-cell-gene-therapists-history-hyperbole-and-hope-for-the-future
#15
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
#16
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
#17
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
#18
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
#19
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
#20
Cathy A Kodroff
No abstract text is available yet for this article.
June 2016: 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"