Cell Therapy News 19.32 September 24, 2018 | |
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TOP STORYGenome Editing in Mitochondria Corrects a Pathogenic mtDNA Mutation In Vivo Through application of a programmable nuclease therapy approach, using systemically administered, mitochondrially targeted zinc-finger nucleases delivered by adeno-associated virus, scientists induced specific elimination of mutant mtDNA across the heart, coupled to a reversion of molecular and biochemical phenotypes. [Nat Med] Abstract | Press Release | |
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PUBLICATIONS(Ranked by impact factor of the journal)All-in-One Adeno-Associated Virus Delivery and Genome Editing by Neisseria meningitidis Cas9 In Vivo Using tail-vein hydrodynamic-based delivery of N. meningitidis Cas9 plasmid to target the Hpd gene, investigators successfully reprogrammed the tyrosine degradation pathway in Hereditary Tyrosinemia Type I mice. [Genome Biol] Full Article Investigators provide evidence that human CD19-CAR T cells can be generated directly in vivo using the lentiviral vector CD8-LV specifically targeting human CD8+ cells. [EMBO Mol Med] Full Article | Graphical Abstract Researchers demonstrated that an intratumoral injection of polymer-double-transfected mesenchymal stem cells (MSCs) along with prodrug ganciclovir injections can induce a significant synergistic therapeutic response both in vitro and in vivo compared to single plasmid transfections or untransfected MSCs. [Acta Biomater] Abstract | Graphical Abstract Interferon-γ-knockout mice with acute kidney injury (AKI) received pulsed focused ultrasound (pFUS)+mesenchymal stromal cell (MSC), but MSC‐derived IL‐10 expression and AKI were similar to using MSC alone. AKI in wild‐type mice receiving pFUS and IL‐10‐deficient MSC was also unimproved compared to administering IL‐10‐deficient MSC alone. [J Cell Mol Med] Abstract Scientists investigated strategies to increase myelination of regenerating axons by over-expression of platelet-derived growth factor-AA and noggin either alone or in combination in a mouse spinal cord injury model. [Biotechnol Bioen] Abstract Autologous transplantation of labeled muscle derived cells (MDCs), bone marrow mesenchymal stem/stromal cells (MSCs) or co-transplantation of MDC-MSC were performed in aged multiparous female goats. [Stem Cell Res Ther] Full Article Wharton’s jelly-derived mesenchymal stem cells (WJ-MSCs) have been introduced as a possible therapy in hypoxic-ischemic encephalopathy (HIE). The authors report a 16-year-old boy who was treated with WJ-MSCs in the course of HIE due to post-cardiopulmonary resuscitation. [Cell Transplant] Full Article Researchers evaluated the healing quality of implanted ultraporous β-tricalcium phosphate sown with expanded autologous mesenchymal stromal cells into femoral defects during revision hip arthroplasty. [Cell Transplant] Full Article Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSThe authors reviewed the mechanisms and applications of ultrasound-targeted microbubble destruction in terms of renal disease, including diabetic nephropathy, renal carcinoma, acute kidney injury, renal interstitial fibrosis, nephrotoxic nephritis, urinary stones and acute rejection. [Hum Gene Ther] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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INDUSTRY NEWSSt. Jude Children’s Research Hospital and Tessa Therapeutics Announce Strategic Collaboration St. Jude Children’s Research Hospital together with Tessa Therapeutics announced the establishment of a strategic collaboration focusing on the development of novel cellular immunotherapies that could lead to new treatment options for children with brain cancer. [St. Jude Children’s Research Hospital (PR Newswire Association LLC.)] Press Release Sarepta Therapeutics, Inc. announced that the FDA has lifted the clinical hold for the company’s Duchenne muscular dystrophy (DMD) micro-dystrophin gene therapy program. [Sarepta Therapeutics] Press Release Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the approval of voretigene neparvovec, a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. [Novartis AG] Press Release Two New Research Projects Funded to Explore Potential Treatments for Calpainopathy/LGMD2A Coalition to Cure Calpain 3 (C3) announced updates to its Gene Therapy Initiative. C3 launched the initiative in 2017 to accelerate the understanding of the potential of gene therapy to treat LGMD2A by funding research projects testing several approaches. [Coalition to Cure Calpain 3] Press Release Amicus Therapeutics announced the signing of a definitive agreement in which Amicus Therapeutics will receive worldwide development and commercial rights for ten gene therapy programs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. [Amicus Therapeutics] Press Release Fujifilm Plans iPS-Based Trials to Treat Complications from Bone Marrow Transplants Fujifilm Corp. aims to start clinical trials with artificially derived stem cells next year to prevent serious complications from bone marrow transplants used to treat leukemia, company officials say. [Fujifilm Corp. (The Japan Times Ltd.)] Editorial uniQure Provides Update on Dose-Confirmation Study of AMT-061 in Patients with Hemophilia B uniQure N.V. announced that the third patient has been treated in the Company’s Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. [uniQure N.V.] Press Release Mesoblast Limited announced continued strong survival outcomes through Day 180 in children with steroid refractory acute Graft Versus Host Disease treated with Mesoblast’s Phase III product candidate remestemcel-L, an allogeneic mesenchymal stem cell product candidate. [Mesoblast Limited] Download Press Release Adverum Biotechnologies, Inc. announced the FDA has granted Fast Track designation for ADVM-022. [Adverum Biotechnologies, Inc.] Press Release AIVITA Biomedical Announces Treatment of First Patient in Phase II Ovarian Cancer Trial AIVITA Biomedical announced that it has dosed its first of 10 patients currently enrolled as part of its Phase II trial in patients with advanced ovarian cancer. The trial is designed to investigate AIVITA Biomedical’s patient-specific cancer treatment consisting of autologous dendritic cells loaded with autologous antigens from the patient’s tumor-initiating cells. [AIVITA Biomedical] Press Release Cell Medica announced the treatment of the first patient world-wide to receive CMD-501, an autologous CAR-NKT therapy targeting pediatric neuroblastoma. [Cell Medica] Press Release Abzena, plc has signed an antibody humanisation agreement with Tmunity Therapeutics, a clinical stage T cell therapy company and a leader in the development of new chimeric antigen receptor T cell therapies for the treatment of solid and hematological cancers. [Abzena, plc] Press Release | |
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EVENTSNEW 10th International Symposium on Neuroprotection and Neurorepair Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Research Associate – Immunotherapy (Baylor College of Medicine) Director – Clinical Development (California Institute for Biomedical Research) Postdoctoral Research Assistant – Gene Therapy (University of Oxford) Faculty Position – Adoptive T Cell Therapy (Dana-Farber Cancer Institute) Professorship – Somatic Gene Therapy (University of Zurich) Postdoctoral Staff Fellow – Biologist/Biomedical Engineer (Food and Drug Administration) Associate Professor – Cancer Research (University of Maryland) Senior Scientist – CAR-T Cell Research (CELLECTIS Inc.) Postdoctoral Fellow – Immuno-Oncology (CELLECTIS Inc.) Research Assistant or Associate – Cell Therapy (California Institute for Biomedical Research) 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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