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Pulmonary Cell News| Cell Therapy News 20.08 March 11, 2019 | |
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TOP STORYResearchers developed CRISPR-Cas9 nanocomplexes and showed they were effective in the adult mouse brain, with minimal off-target effects. [Nat Neurosci] Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)CD8+ T cells expressing CD19-targeted chimeric antigen receptors (CARs) persistently depleted CD19+ B cells, eliminated autoantibody production, reversed disease manifestations in target organs, and extended life spans well beyond normal in (NZB × NZW) F1 and MRLfas/fas mouse models of lupus. [Sci Transl Med] Full Article | Press Release CRISPR-Cas9 Corrects Duchenne Muscular Dystrophy Exon 44 Deletion Mutations in Mice and Human Cells Scientists present a simple and efficient strategy for correction of exon 44 deletion mutations by CRISPR-Cas9 gene editing in cardiomyocytes obtained from patient-derived induced pluripotent stem cells and in a new mouse model harboring the same deletion mutation. [Sci Adv] Full Article | Press Release MRI‐Guided Phase I Trial of Putaminal AADC Gene Therapy for Parkinson’s Disease Novel intraoperative monitoring of administration facilitated targeted delivery of VY‐AADC01 in this Phase I study, which was well tolerated. [Ann Neurol] Abstract AAV cis-Regulatory Sequences Are Correlated with Ocular Toxicity Even in areas of immune privilege such as the eye, adeno-associated viral (AAV) vectors are capable of eliciting host-cell responses. To investigate the effects of such responses on several ocular cell types, the authors tested multiple AAV genome structures and capsid types using subretinal injections in mice. [Proc Natl Acad Sci USA] Full Article To compare the gene transfer efficiency and inflammatory response of NeuRet vs. HiRet vectors, each vector was injected into the striatum in macaque monkeys, common marmosets, and rats. [Sci Rep] Full Article Lipopolysaccharide induced neuroinflammation and apoptosis, and reduced the number of mature oligodendrocytes. Mesenchymal stem cells (MSCs) reduced astrogliosis, but umbilical cord blood (UCB) did not have the same effect. UCB significantly decreased cerebral apoptosis and protected mature myelinating oligodendrocytes, but MSCs did not. [Pediatr Res] Abstract Transient Receptor Potential Vanilloid 1-Based Gene Therapy Alleviates Orthodontic Pain in Rats The role of transient receptor potential vanilloid 1 (TRPV1) in modulating tooth-movement pain was examined by injecting a TRPV1 antagonist into the trigeminal ganglia of rats. [Int J Oral Sci] Full Article Researchers evaluated the influence of rat bone marrow-mesenchymal stem cell treatment on liver histology, biochemical liver function tests, systemic immunoregulatory state and natural killer cell distribution in liver and peripheral blood in rat model of common bile duct ligation and compared the results with the control group. [Mol Biol Rep] Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSGene Therapy Targeting Hematopoietic Stem Cells for Inherited Diseases: Progress and Challenges Several challenges, such as effective reconstitution during inflammation, remain if gene therapy is to be extended to more complex diseases in which hematopoietic stem and progenitor cells can be altered by the disease environment. [Nat Rev Drug Discov] Abstract Mechanisms of Resistance to CAR T Cell Therapy The authors discuss the factors that can preclude durable remissions following CAR T cell therapy, with a primary focus on the resistance mechanisms that underlie disease relapse. [Nat Rev Clin Oncol] Abstract Can glucose levels in people with diabetes be normalized with artificial pancreas systems or via cell replacement approaches? Investigators review the road ahead, including the challenges and opportunities of both approaches. [Cell Metab] Full Article Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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INDUSTRY NEWSSTEMCELL Technologies Wins Deloitte Best Managed Companies Award STEMCELL Technologies announced that the company has been selected as a winner of Canada’s Best Managed Companies program for 2019. Canada’s Best Managed Companies is the country’s leading business awards program, recognizing excellence in the management abilities and practices of Canadian owned and managed companies with revenues over $15 million. [STEMCELL Technologies Inc.] Press Release Rocket Pharmaceuticals Sponsors Clinical Trial at University of California, Los Angeles (UCLA) Rocket Pharmaceuticals, Inc. announced a research agreement to support the clinical development of Rocket’s lentiviral vector-based gene therapy programs in leukocyte adhesion deficiency-I and infantile malignant osteopetrosis towards registrational trials. [Rocket Pharmaceuticals, Inc.] Press Release BioTime, Inc. announced the closing of its previously reported acquisition of Asterias Biotherapeutics, Inc., whereby BioTime has acquired through a merger, all of the remaining outstanding common stock of Asterias which was not previously owned by BioTime. [BioTime, Inc.] Press Release Alnylam Pharmaceuticals, Inc. announced that the ENVISION Phase III study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 in development for the treatment of acute hepatic porphyria, met its primary efficacy endpoint and the majority of secondary endpoints. [Alnylam Pharmaceuticals, Inc.] Press Release Cellect Biotechnology Ltd. announced positive preliminary results from its collaboration with Cell2in, a privately-held South Korean company focused on improving the quality of stem cells. [Cellect Biotechnology Ltd. (PR Newswire Association LLC.)] Press Release Arrowhead Pharmaceuticals Inc. announced that it has dosed the first subjects in a Phase I clinical study of ARO-APOC3, an RNAi-based investigational medicine targeting Apolipoprotein C-III being developed for the treatment of hypertriglyceridemia. [Arrowhead Pharmaceuticals Inc.] Press Release SpinalCyte Announces New Chondrocyte Patents SpinalCyte, LLC announced the issuance of new patents in the U.S. and Japan. The company’s patent portfolio now includes 39 U.S. and international patents issued with 100+ patents pending focused on the clinical use of fibroblasts. [SpinalCyte, LLC (Business Wire, Inc.)] Press Release | |
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POLICY NEWSTrump Once Again Requests Deep Cuts in U.S. Science Spending For the third year in a row, President Donald Trump’s administration has unveiled a budget request to Congress that calls for deep spending cuts at many federal science agencies, including a 15% cut for the National Institutes of Health and a 12% cut for the National Science Foundation, while providing hefty increases for the military. [ScienceInsider] Editorial Head of US Food and Drug Agency Resigns Scott Gottlieb, the head of the US Food and Drug Administration (FDA), resigned on 5 March and will depart the agency in one month. [Nature News] Editorial | Opinion China to Tighten Rules on Gene Editing in Humans China’s health ministry has issued draft regulations that will restrict the use of gene editing in humans, just three months after Chinese researcher He Jiankui announced that twin girls had been born with edited genomes. The proposal includes severe penalties for those who break the rules. If approved, scientists say the policy could have gains and drawbacks for research. [Nature News] Editorial
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REGULATORYTGAConsultation: Proposed Medical Device Classification for Human Cells, Tissues and Organs Storage Solutions and IVF Media Notice
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EVENTSNEW 2019 Tissue Engineering and Regenerative Medicine International Society (TERMIS) World Congress Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Cell Biologist – Regenerative Medicine (Hepatx Corporation) Research Technologist – Immunology (STEMCELL Technologies Inc.) Research Associate – Bioengineering (STEMCELL Technologies Inc.) Stem Cell Podcast Co-Host (Stem Cell Podcast) Associate Scientist – Immuno-Oncology (HMH Center for Discovery & Innovation) Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma) Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center) Cell Manufacturing Scientist (CSL Behring) Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University) Faculty Positions – Hematology and Oncology (University of California, Davis) 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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