Volume 17.06 | Feb 15

Issue 17.06 February 15, 2016
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Deriving Human ENS Lineages for Cell Therapy and Drug Discovery in Hirschsprung Disease
Researchers demonstrated the efficient derivation and isolation of enteric nervous system (ENS) progenitors from human pluripotent stem (PS) cells, and their further differentiation into functional enteric neurons. The in vivo engraftment and migration of human PS-cell-derived ENS precursors rescued disease-related mortality in Hirschsprung disease mice, although the mechanism of action remains unclear. [Nature] Abstract
Free Wallchart from Nature Reviews Neuroscience on Modeling Neurological Disease with iPS Cells
PUBLICATIONS (Ranked by impact factor of the journal)
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells
The authors describe a CRISPR/Cas9 platform applicable to 60% of Duchenne muscular dystrophy (DMD) patient mutations. Internally deleted dystrophin was functional as demonstrated by improved membrane integrity and restoration of the dystrophin glycoprotein complex in vitro and in vivo. [Cell Stem Cell]
Abstract | Graphical Abstract | Press Release

Liraglutide Compromises Pancreatic β Cell Function in a Humanized Mouse Model
Researchers showed that prolonged daily liraglutide treatment of >200 days in humanized mice, transplanted with human pancreatic islets in the anterior chamber of the eye, is associated with compromised release of human insulin and deranged overall glucose homeostasis. [Cell Metab] Abstract | Graphical Abstract | Press Release

Endothelial Dicer Promotes Atherosclerosis and Vascular Inflammation by miRNA-103-Mediated Suppression of KLF4
Investigators showed that reduced endothelial expression of the RNAse Dicer, which generates almost all mature miRNAs, decreases monocyte adhesion, endothelial C–X–C motif chemokine 1 expression, atherosclerosis and the lesional macrophage content in apolipoprotein E knockout mice after exposure to a high-fat diet. [Nat Commun] Full Article | Press Release

Promoting Axon Regeneration in the Adult CNS by Modulation of the Melanopsin/GPCR Signaling
Researchers found that subtypes of intrinsically photosensitive retinal ganglion cells (ipRGCs) in mice maintained high mammalian target of rapamycin (mTOR) levels after axotomy and that the light-sensitive G protein-coupled receptor (GPCR) melanopsin mediated this sustained expression. Melanopsin overexpression in the RGCs stimulated axonal regeneration after optic nerve crush by up-regulating mTOR complex 1. [Proc Natl Acad Sci USA] Abstract | Full Article | Press Release

IGF-1 C Domain–Modified Hydrogel Enhances Cell Therapy for AKI
Scientists synthesized a bioactive hydrogel by immobilizing the C domain peptide of IGF-1 (IGF-1C) on chitosan, and hypothesized that this hydrogel could provide a favorable niche for adipose-derived mesenchymal stem cells and thereby enhance cell survival in an AKI model. [J Am Soc Nephrol] Abstract

AAV-Mediated Gene Therapy Halts Retinal Degeneration in PDE6β-Deficient Dogs
Scientists provide a detailed assessment of the long-term effects of gene therapy on the spatiotemporal pattern of retinal degeneration in rod-cone dysplasia type 1 dogs treated at 20 days of age. Whereas no rods were observed outside the bleb or in untreated eyes, gene transfer halted rod degeneration in all vector-exposed regions. [Mol Ther] Abstract

Basic FGF or VEGF Gene Therapy Corrects Insufficiency in the Intrinsic Healing Capacity of Tendons
By basic fibroblast growth factor (bFGF) or vascular endothelial growth factor (VEGF) gene therapy via adeno-associated viral type-2 vector to produce supernormal amounts of bFGF or VEGF intrinsically in the tendon, researchers effectively corrected the insufficiency of the tendon healing capacity. [Sci Rep] Full Article

3D Printing Surgical Implants at the Clinic: A Experimental Study on Anterior Cruciate Ligament Reconstruction
A well-defined, orthogonal, porous PLA screw-like scaffold was printed, then coated with hydroxyapatite to improve its osteoconductivity. As an internal fixation as well as an ideal cell delivery system, the osteogenic scaffold loaded with mesenchymal stem cells were evaluated through both in vitro and in vivo tests to observe bone-ligament healing via cell therapy. [Sci Rep] Full Article

Transplantation of Neural Progenitor Cells in Chronic Spinal Cord Injury
Investigators tested whether transplanted neural progenitor cells can also improve functional recovery after chronic spinal cord injury alone or in combination with the reduction of glial scar and neurotrophic support. [Neuroscience] Abstract

Mifepristone-Inducible Transgene Expression in Neural Progenitor Cells In Vitro and In Vivo
The authors used a lentivirus-based vector platform for the ex vivo production of mifepristone-inducible murine neural progenitor cells that express transgenes of interest. Inducible cells were then transplanted into the brains of rodents, where they exhibited appropriate mifepristone-inducible expression. [Gene Ther] Abstract

Effects of FVIII Immunity on Hepatocyte and Hematopoietic Stem Cell–Directed Gene Therapy of Murine Hemophilia A
As clinical immune responses to coagulation factors VIII (FVIII) and IX differ, scientists investigated the ability of liver-directed adeno-associated viral-FVIII gene therapy to affect FVIII immunity in hemophilia A mice. [Mol Ther Methods Clin Dev] Full Article

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Gene Insertion into Genomic Safe Harbors for Human Gene Therapy
The authors review their formerly proposed genomic safe harbor (GSH) criteria and discuss additional considerations on extending these criteria, on strategies for the identification and validation of GSHs, as well as future prospects on GSH targeting for therapeutic applications. [Mol Ther] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research fields.

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Researchers Create ‘Mini-Brains’ in Lab to Study Neurological Diseases
Researchers have developed tiny “mini-brains” made up of many of the neurons and cells of the human brain – and even some of its functionality – and which can be replicated on a large scale. [Press release from Johns Hopkins Bloomberg School of Public Health discussing research presented at the American Association for the Advancement of Science (AAAS) Annual Meeting, Washington, DC] Press Release

Stan Riddell, Fred Hutch Cancer Immunotherapy Innovator, to Present at AAAS Annual Meeting
Dr. Stanley Riddell, an immunotherapy researcher and oncologist, presented an update on new adoptive T-cell strategies for cancer. [Press release from Fred Hutchinson Cancer Research Center (EurekAlert!) discussing research presented at the American Association for the Advancement of Science (AAAS) Annual Meeting, Washington, DC] Press Release

Progress in Fighting Cancer and Infections with T Cell Therapy
Scientists have worked on providing cell products that will upon transfer to patients expand to large numbers and stay active for a long time, potentially life-long. [Press release from the Technical University of Munich discussing research presented at the American Association for the Advancement of Science (AAAS) Annual Meeting, Washington, DC] Press Release

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Karolinska Institute Vice-Chancellor Resigns in Wake of Macchiarini Scandal
In the wake of an ever-widening scandal surrounding surgeon Paolo Macchiarini, the vice-chancellor of the Karolinska Institute (KI) in Stockholm has resigned. Anders Hamsten was in charge of an investigation last year into Macchiarini’s work at KI. [ScienceInsider] Editorial

Obama Makes Risky Bid to Increase Science Spending
With less than a year before he leaves office, US President Barack Obama is making a strong push to increase spending on scientific research. His fiscal year 2017 budget plan calls for a 4% bump in research and development funding across the federal government. [Nature News] Editorial

American College of Rheumatology Encourages Safe Adoption of Biosimilars During FDA Public Hearing on CT-P13, a Proposed Biosimilar for Infliximab (Remicade)
During a public meeting held by the Food and Drug Administration (FDA)’s Arthritis Advisory Committee to review the license application of CT-P13, the American College of Rheumatology provided recommendations for policy guidelines to implement as the committee decides whether to license additional biosimilars for sale in the U.S. [American College of Rheumatology] Editorial
PDS Biotechnology Signs Agreement with National Cancer Institute
PDS Biotechnology announced that the company has signed a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI). Under the CRADA, PDS and NCI aim to co-develop several immunotherapies through Phase II clinical trials utilizing combinations of Versamune® with NCI- and PDS-sourced tumor-related proteins or their fragments recognized by the immune system in prostate, breast, and HPV-related cancers. [PDS Biotechnology Corporation] Press Release

UI Strikes Agreement with Pfizer Inc. to Develop Potential Cystic Fibrosis Gene Therapy
The University of Iowa (UI) Research Foundation finalized a license and sponsored research agreement with Pfizer Inc. to support the development of potential gene therapies for cystic fibrosis. [The University of Iowa] Press Release

Regeneron Joins Human Vaccines Project to Improve Global Disease Prevention and Treatment
Regeneron Pharmaceuticals Inc. announced that it has joined the Human Vaccines Project, a public-private partnership to help discover and develop new vaccines and immunotherapies for infectious diseases and cancer. [Regeneron Pharmaceuticals Inc.] Press Release

Another £1 Million for Research Projects
Pancreatic Cancer Research Fund is funding six new research projects with a total of £1 million – bringing the charity’s support for research into the UK’s most lethal cancer to over £8 million. [Pancreatic Cancer Research Fund] Press Release

SITC Offering Fellowships Nearing $300,000 to Support Next Generation of Cancer Immunotherapy Researchers
The Society for Immunotherapy of Cancer (SITC) is dedicated to cultivating young scientists who are instrumental to cancer immunotherapy research and, in that commitment, is presenting the next generation of cancer immunotherapy researchers four, one-year fellowship opportunities with total award nearing $300,000. [Society for Immunotherapy of Cancer] Press Release

Clinical Trial Offers New Drug Combo to Fight Lung Cancer
The Phase III clinical trial, called “Neptune,” compares a combination of two immune drugs with standard chemotherapy. It is one of the first clinical trials to test immune drugs as a first line treatment for lung cancer. [University of New Mexico Comprehensive Cancer Center] Press Release

Immune Design Announces Positive Topline Data from Three Phase I Clinical Trials of Immuno-Oncology Product Candidates
Immune Design reported positive topline data from three ongoing Phase I oncology studies that support continued development of its two primary in vivo immuno-oncology product candidates, CMB305 and G100. [Immune Design] Press Release

Myasthenia Gravis: CuraVac Launches Phase Ib Study in Patients
CuraVac Inc. announced that it has launched a Phase Ib study of its lead program, CVMG01. CV-MG01 is an immunotherapy developed for the treatment and potential cure of myasthenia gravis, a debilitating autoimmune disease. [CuraVac Inc.] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2016-02972)

National Institute of General Medical Sciences; Notice of Closed Meeting (FR Doc. No: 2016-02853)
Food and Drug Administration (United States)

Request for Nominations for Individuals and Consumer Organizations for Advisory Committees (FR Doc. No: 2016-03010)

Food and Drug Administration Clinical Trial Requirements, Regulations, Compliance, and Good Clinical Practices; Public Workshop (FR Doc. No: 2016-02965)
NEW Tumor Microenvironment and Signaling
April 3-6, 2016
Heidelberg, Germany

NEW American Society of Gene & Cell Therapy (ASGCT) 19th Annual Meeting 2016
May 4-7, 2016
Washington, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Lead Scientist – Translational Therapy (NHS Blood and Transplant)

NEW PhD Position – Leukemogenesis and Treatment Resistance (Institute for Tumor Biology and Experimental Therapy)

Postdoctoral Positions – Gene Regulation and Translational Cancer Research (University of Texas Health Science Center)

Postdoctoral Position – Hematology/Oncology (Penn State College of Medicine)

Group Leader – Cell Therapy (Miltenyi Biotec)

Director of Manufacturing – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Professorship – Transfusion Medicine and Cell Therapeutics (Ludwig-Maximilians-Universität München)

Postdoctoral Fellow/Research Associate – Cancer Therapy (Memorial Sloan Kettering Cancer Center)

Translational Research Scientist – Hematology/Oncology (University of Chicago)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Postdoctoral Position – CAR T Cell Engineering & Cancer Immunotherapy (University Hospital Würzburg)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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