Volume 16.12 | Mar 30

Issue 16.12 March 30, 2015
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MDC Researchers Greatly Increase Precision of New Genome Editing Tool
CRISPR-Cas9 is a powerful new tool for editing the genome. Using a molecular trick, researchers have found a solution to considerably increase the efficiency of precise genetic modifications by up to eightfold. [Press release from Max Delbrück Center for Molecular Medicine Berlin-Buch discussing online publication in Nature Biotechnology] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
NRF2 Promotes Neuronal Survival in Neurodegeneration and Acute Nerve Damage
The authors created several adeno-associated virus vectors to deliver genes that combat oxidation. They tested the effectiveness of this approach in three models of photoreceptor degeneration and in a nerve crush model. [J Clin Invest] Full Article | Press Release | Video

Oncogenesis Driven by the Ras/Raf Pathway Requires the SUMO E2 Ligase Ubc9
Scientists showed that the small ubiquitin-like modifier (SUMO) pathway is required for KRAS-driven transformation. RNAi depletion of the SUMO E2 ligase Ubc9 suppresses 3D growth of KRAS mutant colorectal cancer cells in vitro and attenuates tumor growth in vivo. [Proc Natl Acad Sci USA] Abstract

TGF-β3 Encapsulated PLCL Scaffold by a Supercritical CO2-HFIP Co-Solvent System for Cartilage Tissue Engineering
Researchers investigated the tissue regeneration efficiency of the TGF-β3 encapsulated poly(lactide-co-caprolacton) (PLCL) scaffold using human adipose-derived stem cells in vitro and in vivo. [J Control Release] Abstract

Tau Immunotherapy Modulates Both Pathological Tau and Upstream Amyloid Pathology in an Alzheimer’s Disease Mouse Model
Investigators evaluated the specific removal of pathological tau oligomers in aged Tg2576 mice by passive immunotherapy using tau oligomer-specific monoclonal antibody. [J Neurosci] Abstract | Press Release

Optogenetic Control of Insulin Secretion by Pancreatic β-Cells In Vitro and In Vivo
Scientists used optogenetics to investigate whether insulin secretion and blood glucose homeostasis could be controlled by regulating intracellular calcium ion concentrations in a mouse pancreatic β-cell line transfected with the optogenetic protein channelrhodopsin-2. [Gene Ther] Abstract

Tissue-Engineered Regeneration of Completely Transected Spinal Cord Using Induced Neural Stem Cells and Gelatin-Electrospun Poly(Lactide-co-Glycolide)/Polyethylene Glycol Scaffolds
Researchers investigated induced mouse embryonic fibroblasts directly reprogrammed into neural stem cells (iNSCs), as a cell source. Three-dimensional electrospun poly(lactide-co-glycolide)/polyethylene glycol nanofiber scaffolds were used for iNSCs adhesion and growth. [PLoS One] Full Article

Adoptive T-Cell Immunotherapy for Ganciclovir-Resistant CMV Disease after Lung Transplantation
Scientists demonstrated successful expansion and adoptive transfer of autologous cytomegalovirus (CMV)-specific T cells from a seronegative recipient of a seropositive lung allograft with ganciclovir-resistant CMV disease, resulting in the long-term reconstitution of protective anti-viral immunity, CMV infection, disease-free survival and no allograft rejection. [Clin Transl Immunol] Full Article

Immune Therapy of Metastatic Melanoma Developing after Allogeneic Bone Marrow Transplant
Researchers report two patients with metastatic melanoma who received treatment with immunotherapy after allogeneic stem cell transplantation that did not result in graft versus host disease. [J Immunother Cancer] Full Article

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Advances and Challenges in Immunotherapy for Solid Organ and Hematopoietic Stem Cell Transplantation
The authors outline the current immunological limitations for hematopoietic stem cell and solid organ transplantation and discuss new immune-modulating therapies in preclinical development and in clinical trials that may allow these obstacles to be overcome. [Sci Transl Med] Abstract

Advances in CRISPR-Cas9 Genome Engineering: Lessons Learned from RNA Interference
The authors examine how the history of RNA interference can inform today’s challenges in clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 genome engineering such as efficiency, specificity, high-throughput screening and delivery for in vivo and therapeutic applications. [Nucleic Acids Res] Full Article

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

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Cleveland Clinic Researchers First to Demonstrate Significant Blocking of Opioid Tolerance with Mesenchymal Stem Cell Transplant
Mesenchymal stem cell transplantation reduced opioid tolerance and opioid-induced hyperalgesia caused by daily morphine injections in rats, according to new research. [Press release from American Academy of Pain Medicine (AAPM) discussing research presented at the 31st Annual Meeting of the AAPM, Washington D.C.] Press Release

Advaxis’s Lm-LLO Immunotherapy Platform to be Featured in Three Poster Presentations
Advaxis, Inc. announced that three abstracts revealing new data for Advaxis’s proprietary Lm-LLO cancer immunotherapy platform have been selected for poster presentations. [Press release from Advaxis, Inc. discussing research to be presented at the 2015 American Association for Cancer Research (AACR) Annual Meeting, Philadelphia] Press Release

Stem Cells May Significantly Improve Tendon Healing, Reduce Retear Risk in Rotator Cuff Surgery
An injection of a patient’s bone marrow stem cells during rotator cuff surgery significantly improved healing and tendon durability. Each year in the U.S., more than 2 million people have rotator cuff surgery to re-attach their shoulder tendon to the head of the humerus. [Press release from American Academy of Orthopaedic Surgeons (AAOS) discussing research presented at the 2015 Annual Meeting of the AAOS, Las Vegas] Press Release

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U.K. Government Scientists Hit with Media Restrictions
Advocates for science communication in the United Kingdom have expressed “deep concern” about a change to the Civil Service Code for public workers, including researchers at government agencies. [ScienceInsider] Editorial

Max Planck Society Unveils €50 Million Support Plan for Young Scientists
The Max Planck Society will improve its support for junior scientists and do away with a stipend system used mostly for foreign Ph.D. students and postdocs that many had decried as unfair because it doesn’t include basic social security benefits. [ScienceInsider] Editorial
Taconic Biosciences and Cellaria Biosciences Agree to Scientific Collaboration to Facilitate Xenograft Use in Oncology Research
Taconic Biosciences and Cellaria Biosciences announced that the two companies have entered into a scientific collaboration designed to facilitate and improve the utility of patient-derived xenografts in animal models for oncology and immuno-oncology research. [Taconic Biosciences, Inc.] Press Release

Novasep and Celladon Execute MYDICAR API Supply Agreement
Novasep and Celladon Corporation announced that they have signed a Development, Manufacturing and Supply Agreement pursuant to which, if supported by upcoming MYDICAR clinical data, Novasep would manufacture MYDICAR drug substance through 2018 with extension options through 2020. [Novasep] Press Release

RXi Pharmaceuticals and MirImmune LLC Enter into an Exclusive Licensing Agreement for RXi’s Self-Delivering RNAi (sd-rxRNA) Technology to Develop Cell-Based Immunotherapies to Treat Cancer
RXi Pharmaceuticals Corporation and privately-held MirImmune LLC, announced that they have entered into an exclusive license agreement to RXi’s novel and proprietary sd-rxRNA® technology for use in developing innovative cell-based cancer immunotherapies. [RXi Pharmaceuticals Corporation] Press Release

Accellta Grants Exclusive Rights for Micro-Carrier Free Stem Cells Suspension Culture to STEMCELL Technologies
The stem cell company Accellta announced that it has granted exclusive worldwide rights to STEMCELL Technologies Inc. for manufacturing and distribution of medium for culturing pluripotent stem cells in suspension under feeder-free, non-adherent conditions. [Accellta Ltd.] Press Release

apceth Initiates Phase II Clinical Trial for Pioneering Engineered Cell Therapy to Treat Gastrointestinal Cancer
apceth announced the successful completion of the Phase I and initiation of the Phase II part of its ongoing monocentric Phase I/II clinical trial TREAT-ME 1 with the engineered cell therapeutic product Agenmestencel-T. [apceth GmbH & Co. KG] Press Release

Medigene Starts Phase I/II Study with DC Vaccine to Treat Acute Myeloid Leukemia (AML)
Medigene AG announced that the company’s Phase I/II clinical trial with its dendritic cell vaccine for the treatment of AML has started. [Medigene AG] Press Release

Immunovaccine Initiates Phase II Clinical Trial of DPX-Survivac Immunotherapy in Recurrent Lymphoma
Immunovaccine Inc. announced that it has treated the first patient with diffuse large B cell lymphoma in a Phase II clinical study of its lead cancer immunotherapy DPX-Survivac. [Immunovaccine Inc.] Press Release

UC Researchers Awarded Stem Cell Grants
University of California researchers from two campuses received three grants totaling more than $12 million in funding from the state’s stem cell agency to develop stem cell treatments for Huntington’s disease, spina bifida and chronic diabetic wounds. [UC Health] Press Release

City of Hope Launches Stem Cell Therapy Clinic to Fight Incurable Disease in New Ways
The City of Hope officially opened the Alpha Clinic for Cell Therapy and Innovation for patients battling cancer and other life-threatening diseases. The Alpha Clinic is funded by an $8 million, five-year grant from the California Institute for Regenerative Medicine that will be supplemented by matching funds from City of Hope. [City of Hope] Press Release

Researchers Aim to Broaden Understanding of How Toxins Affect the Body
University of Wisconsin-Madison researchers received $6 million from the U.S. Environmental Protection Agency to develop models and screening tools that will rapidly advance knowledge of the health effects of an ever-growing assortment of environmental toxins. Awarded through the EPA Science to Achieve Results program, the grant will create the Human Models for Analysis of Pathways Center at UW-Madison. [University of Wisconsin-Madison] Press Release
National Institute of Neurological Disorders and Stroke; Notice of Closed Meeting (FR Doc. No: 2015-06841)
User Fee Billable Biologic Products and Potencies Approved Under Section 351 of the PHS Act
Food and Drug Administration (United States)

Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines, and Related Recombinant Viral or Microbial Products; Guidance for Industry: Availability (FR Doc. No: 2015-06686)

Agency Information Collection Activities; Proposed Collection; Comment Request; Registration of Producers of Drugs and Listing of Drugs in Commercial Distribution (FR Doc. No: 2015-06497)
NEW Stem Cells: From Basic Research to Bioprocessing
June 9-11, 2015
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Lecturer – Gene Therapy (University College London)

NEW Postdoctoral Position – Cancer Imaging (King’s College London)

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Scientist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Research Associate – Cell Culture Specialist (Tisch MS Research Center of New York)

Manufacturing Manager – Cell Therapy (Opexa Therapeutics)

Postdoctoral Positions – Developmental Biology (Joslin Diabetes Center)

Cell and Gene Therapy Formulation Scientist (Stelfox)

Research Scientist – Cell Signaling in Normal and Leukemic Hematopoietic Stem and Progenitor Cells (Indiana University)

Postdoctoral Position – Gene Therapy of Lysosomal Storage Diseases (Cincinnati Children’s Hospital Medical Center)

Associate Director, Center for Gene Therapy (City of Hope)

Assistant Professor – Neuroscience (Georgia State University)

Research Technician in AAV Gene Therapy (Icahn School of Medicine at Mount Sinai)

Assistant/Associate Professor – Gastroenterology (North Carolina State University College of Veterinary Medicine)

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