Cell Therapy News 21.06 February 24, 2020 | |
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TOP STORYApart from steroid-responsive subretinal inflammation in patients at the higher doses, there were no notable safety concerns after subretinal delivery of an adeno-associated viral vector encoding codon-optimized human retinitis pigmentosa GTPase regulator (RPGR), meeting the pre-specified primary endpoint. [Nat Med] Abstract | Press Release | |
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PUBLICATIONS(Ranked by impact factor of the journal)Generation of Human Endothelium in Pig Embryos Deficient in ETV2 ETV2-null blastocysts were injected with human induced pluripotent stem cells overexpressing the antiapoptotic factor BCL2, transferred to synchronized gilts and analyzed between embryonic day 17 and embryonic day 18. In these embryos, all endothelial cells were of human origin. [Nat Biotechnol] Abstract Synaptotagmin 13 Is Neuroprotective across Motor Neuron Diseases Gene therapy with Syt13 prolonged the lifespan of amyotrophic lateral sclerosis mice by 14% and spinal muscular atrophy mice by 50% by preserving motor neurons and delaying muscle denervation. [Acta Neuropathol] Full Article Researchers investigated the effects of neural cell integration into the bioprinted skeletal muscle construct to accelerate functional muscle regeneration in vivo. The bioprinted constructs with neural cell integration facilitated rapid innervation and matured into organized muscle tissue that restored normal muscle weight and function in a rodent model of muscle defect injury. [Nat Commun] Full Article Engineering Light-Controllable CAR T Cells for Cancer Immunotherapy Researchers developed a light-inducible nuclear translocation and dimerization system for gene regulation to control CAR T activation. They demonstrated light-controllable gene expression and functional modulation in human embryonic kidney 293T and Jurkat T cell lines. [Sci Adv] Full Article | Editorial Investigators describe a dual-adeno-associated virus vector system that accomplished rapid short-term expression from a non-integrated minigene and long-term expression from the site-specific integration of this minigene without any selective growth advantage for ornithine transcarbamylase-positive cells in newborns. [Sci Adv] Full Article Scientists evaluated the efficacy of herpes simplex virus thymidine kinase suicide gene therapy for glioblastoma using neural stem/progenitor cells derived from human induced pluripotent stem cells (hiPSCs). Although transduction of hiPSCs is preferable for a safe and stable supply in the clinical setting, high-level and/or constitutive HSV-TK expression was highly cytotoxic to hiPSCs. [Hum Gene Ther] Abstract In an in vivo experiment, single-cell adipose-derived stem cells (ADSCs) and 3D-cultured ADSC spheroids were injected intra-articularly into the knees of rheumatoid arthritis model mice and histologically assessed. Marked improvement of synovial inflammation and articular cartilage regeneration was found in ADSC-treated mice. [Sci Rep] Full Article Scientists investigated the effect of Matrigel combined with neural stem cells (NSCs) in vitro and in vivo. They compared the effect of different types of scaffold on the survival and differentiation of brain-derived NSCs in an in vitro culture. [Sci Rep] Full Article Adipose Tissue-Derived Stem Cell Sheet Improves Glucose Metabolism in Obese Mice Researchers investigated the effects of adipose tissue-derived stem cell (ADSC) sheet transplantation into the subcutaneous sites on the diabetic state of mice fed high fat and high sucrose diet. ADSCs were isolated and propagated from subcutaneous adipose tissues of non-diabetic intact mice. They used the thermo-responsive designated cell culture dishes to fabricate ADSC cell sheets. [Stem Cells Dev] Abstract Researchers evaluated the utility of microminipigs for liver-targeted gene therapy. These pigs were intravenously inoculated with an adeno-associated virus (AAV)8 vector encoding the luciferase gene, and gene expression was assessed by an in vivo imaging system. Robust transgene expression was observed almost exclusively in the liver, even though the pig showed a low-titer of neutralizing antibody against the AAV8 capsid. [Gene Ther] Full Article The authors showed that β3 adrenergic agonists (β3AR) in combination with a CXCR4 antagonist, AMD3100/Plerixafor, could mobilize mesenchymal stromal cells into the blood in mice and rats. Mechanistically, they showed that reversal of the CXCL12 gradient across the bone marrow endothelium and local generation of endocannabinoids may both play a role in this process. [NPJ Regen Med] Full Article Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSEnvironmentally Responsive Hydrogels for Repair of Cardiovascular Tissue The authors review the occurrence and current treatment of cardiovascular disease, as well as the repair of cardiovascular injury by several environmental responsive hydrogels systems currently used, mainly focusing on the delivery of growth factors or the application of cell therapy to revascularization. They also discuss the enormous potential and personal perspectives of environmentally responsive hydrogels in cardiovascular repair. [Heart Fail Rev] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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INDUSTRY NEWSGenprex, Inc. announced that it signed an exclusive license agreement with the University of Pittsburgh for a diabetes gene therapy that may have the potential to cure Type 1 and Type 2 diabetes. [Genprex, Inc.] Press Release LogicBio Therapeutics, Inc. announced that the FDA has placed a clinical hold on the Investigational New Drug submission for LB-001 for the treatment of methylmalonic acidemia pending the resolution of certain clinical and nonclinical questions. [LogicBio Therapeutics, Inc.] Press Release BrainStorm Cell Therapeutics, Inc. announced that the company recently held a high level meeting with the FDA to discuss potential NurOwn® regulatory pathways for approval in ALS. [BrainStorm Cell Therapeutics, Inc.] Press Release Bristol-Myers Squibb Company announced that the FDA has accepted for Priority Review its BLA for lisocabtagene maraleucel, the company’s autologous anti-CD19 chimeric antigen receptor (CAR) T-cell immunotherapy with a defined composition of purified CD8+ and CD4+ CAR T cells for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after at least two prior therapies. [Bristol-Myers Squibb Company] Press Release | |
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POLICY NEWS‘The Disruption Is Enormous.’ Coronavirus Epidemic Snarls Science Worldwide Normal daily life has come to a virtual standstill in large parts of China as a result of the epidemic of COVID-19—and so has science. Universities across the country remain closed; access to labs is restricted, projects have been mothballed, field work interrupted, and travel severely curtailed. [ScienceInsider] Editorial White House Formally Invites Public Comment on Open-Access Policies The White House issued a notice seeking comment on its effort to enhance public access to federally funded research. It’s an old idea creating new controversy. White House Office of Science and Technology Policy Director Kelvin Droegemeier is pushing back against publishers that in December said the administration was quietly pursuing an executive order to require immediate free distribution of taxpayer funded research. [ScienceInsider] Editorial Has the UK Science Minister Been Demoted? A government reshuffle in the United Kingdom has born another new science minister – a position that has seen a revolving door of appointees, firings and resignations in recent years. The latest appointment also comes with an apparent political demotion for the role, which expert observers say is in conflict with a government that appears to have put research and innovation at the heart of its agenda. [Nature News] Editorial
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EVENTSNEW American Association for Cancer Research (AACR) Annual Meeting 2020 Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Research Associate – Stem Cell and Gene Therapy Program (Fred Hutchinson Cancer Research Center) Postdoctoral Researcher – Cell Therapies for Cancer (Technical University of Denmark) Postdoctoral Fellow – Inflammation and Cancer (University Hospital of Erlangen) Lecturer – Clinical Pharmacology (University of Bristol) Research Assistant – Organoid Modeling for Stem Cell Therapies (Boston Children’s Hospital) Postdoctoral Researcher – Drug Delivery (University of Minnesota Twin Cities) Postdoctoral Researchers – Cell Biology (Medicinal Bioconvergence Research Center) Postdoctoral Fellow – Cancer Research (BC Cancer) Research Assistant – Cancer Research (BC Cancer) Postdoctoral Fellow – Single Cell Genomics (BC Cancer) Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.
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