Volume 14.00 | Jan 7

Multiplex Genome Engineering Using CRISPR/Cas Systems
Researchers engineered two different type II CRISPR (clustered regularly interspaced short palindromic repeats) systems and demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells. [Science] Abstract | Press Release

Regeneration of Human Tumor Antigen-Specific T Cells from iPSCs Derived from Mature CD8⁺ T Cells
Researchers established iPSCs from mature cytotoxic T cells specific for the melanoma epitope MART-1. When cocultured with OP9/DLL1 cells, these iPSCs efficiently generated TCRβ⁺CD4⁺CD8⁺ double positive cells expressing a T cell receptor specific for the MART-1 epitope. Stimulation of the CD8⁺ T cells with MART-1 antigen-presenting cells led to the secretion of IFNγ, demonstrating their specific reactivity. [Cell Stem Cell] Abstract | Graphical Abstract | Press Release

Generation of Rejuvenated Antigen-Specific T Cells by Reprogramming to Pluripotency and Redifferentiation
Investigators reprogrammed clonally expanded antigen-specific CD8⁺ T cells from an HIV-1-infected patient to pluripotency. The T cell-derived induced pluripotent stem cells were then redifferentiated into CD8⁺ T cells that had a high proliferative capacity and elongated telomeres. These rejuvenated cells possessed antigen-specific killing activity and exhibited T cell receptor gene-rearrangement patterns identical to those of the original T cell clone from the patient. [Cell Stem Cell] Abstract | Graphical Abstract | Press Release

Multimodal Actions of Neural Stem Cells in a Mouse Model of ALS: A Meta-Analysis
Investigators tested the hypothesis that transplanted, undifferentiated multipotent migratory neural stem cells may help to ameliorate an array of pathological mechanisms in the SOD1^G93A transgenic mouse model of amyotrophic lateral sclerosis (ALS). [Sci Transl Med] Abstract | Press Release

Umbilical Cord Blood Therapy Potentiated with Erythropoietin for Children with Cerebral Palsy: A Double-Blind, Randomized, Placebo-Controlled Trial
Investigators assessed the safety and efficacy of allogeneic umbilical cord blood potentiated with recombinant human erythropoietin in children with cerebral palsy. [Stem Cells] Abstract | Full Article

Bone Marrow Transplantation in Hindlimb Muscles of Motoneuron Degenerative Mice Reduces Neuronal Death and Improves Motor Function
Scientists transplanted bone marrow cells into a mouse model of motoneuron degeneration, with the particularity of placing the cells in the hindlimb muscles rather than in the spinal cord where neuronal degeneration occurs. To this end, they analyzed the possibility for the transplanted cells to increase the survival rate of the spinal cord motoneurons by axonal-guided retrograde neurotrophism. As a result, the mice significantly improved their motor functions. [Stem Cells Dev] Abstract

Stem Cell Therapy Remediates Reconstruction of the Craniofacial Skeleton after Radiation Therapy
This study utilized transplanted bone marrow stromal cells (BMSCs) as a cellular replacement therapy to remedy radiation induced injury and restore impaired new bone formation during distraction osteogenesis (DO). BMSC therapy brought about the successful generation of new bone and significantly improved both the rate and quality of bony union of irradiated, distracted murine mandibles to the level of non-irradiated DO animals. [Stem Cells Dev] Abstract

PEP-1-CAT-Transduced Mesenchymal Stem Cells Acquire an Enhanced Viability and Promote Ischemia-Induced Angiogenesis
Researchers investigated the role of PEP-1-CAT transduction in mesenchymal stem cells’ survival and its effect on ischemia-induced angiogenesis. [PLoS One] Full Article

Cell Therapy with Bone Marrow Stromal Cells after Intracerebral Hemorrhage: Impact of Platelet-Rich Plasma Scaffolds
Cell therapy using bone marrow stromal cells (BMSCs) has been considered a promising strategy for neurologic sequelae after intracerebral hemorrhage. Investigators demonstrated neurologic improvement after BMSC transplantation and significantly better functional improvement for the group of animals that received BMSCs in the platelet-rich plasma-derived scaffold compared with the group that received BMSCs in saline. [Cytotherapy] Abstract | Full Article

Injection of Vessel-Derived Stem Cells Prevents Dilated Cardiomyopathy and Promotes Angiogenesis and Endogenous Cardiac Stem Cell Proliferation in mdx/utrn^−/− but Not Aged mdx Mouse Models for Duchenne Muscular Dystrophy
The authors hypothesized that mesoangioblast stem cells (aorta-derived mesoangioblasts) would restore dystrophin and alleviate or prevent dilated cardiomyopathy in animal models of Duchenne muscular dystrophy. [Stem Cells Transl Med] Abstract

Therapeutic Opportunities for Manipulating T_Reg Cells in Autoimmunity and Cancer
In this review, the authors discuss insights into the generation of regulatory T (T_Reg) cells, their involvement in disease and the molecular basis of the dominant tolerance exerted by FOXP3⁺ T_Reg cells that could permit their safe and specific manipulation in humans. [Nat Rev Drug Discov] Abstract

Gene Therapy for the Prevention of Vein Graft Disease
Researchers review the pathogenesis of vein graft disease and discuss vector delivery strategies and potential molecular targets for its prevention. They summarize the preclinical and clinical literature on gene therapy in vein grafting and discuss additional considerations for future therapies to prevent vein graft disease. [Transl Res] Abstract

First in Michigan: Stem Cells to Preserve Heart Function
Physicians at Henry Ford Hospital are the first in Michigan in a new national research study to investigate treating a recent heart attack with a patient’s own stem cells to preserve heart muscle function. [Henry Ford Health System] Press Release

Alnylam Files Clinical Trial Application to Initiate a Phase I Study for ALN-TTRsc, a Subcutaneously Administered RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR)
Alnylam Pharmaceuticals, Inc. announced that it has filed a Clinical Trial Application with the U.K. Medicines and Healthcare products Regulatory Agency to initiate a Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting TTR for the treatment of ATTR. [Alnylam Pharmaceuticals, Inc.] Press Release

Stem Cell Lawsuit Finally Over
The Supreme Court rejected a request to ban U.S.-funded research on human embryonic stem cells (hESCs). The decision brings to an end a long legal battle that has cast a shadow over hESC studies for over three years. [Supreme Court, United States] Press Release

India Unveils Ambitious Science Policy
The Indian government adopted a new science, technology, and innovation policy that calls for doubling the investment in science in the next five years and establishing India among the top five nations in output of scientific publications by the end of the decade. [Indian Government, India] Press Release

US Fiscal Deal Leaves Science Vulnerable
Law-makers in Washington DC greeted the new year with a frantic deal meant to avert a fiscal crisis. But the bill that passed the Senate and the House keeps researchers on tenterhooks for at least another two months by delaying mandatory spending cuts that could threaten science funding. [US Government, United States]
Press Release

BioTime Signs Definitive Agreement with Geron Regarding Stem Cell Assets
BioTime, Inc. and its recently formed subsidiary BioTime Acquisition Corporation jointly announced that they have entered into a definitive Asset Contribution Agreement with Geron Corporation to acquire the intellectual property, including patents and patent applications, and other assets related to Geron’s human embryonic stem cell programs consistent with the financial terms outlined in the letter of intent announced on November 15, 2012. [BioTime, Inc.] Press Release

Ethris Announces Research Based Alliance on SNIM^® Modified RNA with Shire
Ethris GmbH and Shire announced the initiation of a research based alliance focused on the development and commercialization of novel RNA-based therapeutics. [Ethris GmbH] Press Release

Sanford-Burnham Medical Research Institute and Intrexon Corporation Establish Collaboration to Accelerate Stem Cell Research
Sanford-Burnham Medical Research Institute and Intrexon Corporation announced a new collaboration to accelerate stem cell research. [Intrexon Corporation] Press Release

Evotec and Apeiron Biologics Announce Collaboration on Cancer Immunotherapy
Evotec AG announced that Evotec and Apeiron Biologics have entered into a research collaboration with the objective of developing immunomodulatory lead compounds for the treatment of cancer. [Evotec AG]
Press Release

Emory/Georgia Tech Regenerative Engineering and Medicine Center Awards 11 Collaborative Seed Grants
The Emory/Georgia Tech Regenerative Engineering and Medicine Center recently awarded 11 seed grants, totaling $630,000, for promising new research in regenerative medicine. The seed grants focus on how the body – including bone, muscle, nerves, blood vessels and tissues – can harness its own potential to heal or regenerate following trauma or disease. [Georgia Institute of Technology] Press Release

Solstice Biologics Secures $18 Million Series A; Licenses RNAi Delivery Technology from UC San Diego
Solstice Biologics LLC announced the completion of a $18 million Series A financing led by venBio and joined by Aeris Capital AG. In addition, Solstice has acquired an exclusive license to University of California (UC), San Diego intellectual property covering technology that enables RNA molecules to cross membranes of multiple cell types, a perplexing problem that has been a bottleneck in delivering on the therapeutic potential of RNA interference (RNAi) and microRNA. [PR Newswire Association LLC] Press Release

Soft Tissue Regeneration Receives FDA Clearance to Market Breakthrough Rotator Cuff Repair Device
Soft Tissue Regeneration announced that it has received FDA clearance to market its STR GRAFT, a biodegradable scaffold used for soft tissue augmentation and rotator cuff repair. Developed by Cato T. Laurencin, the STR GRAFT is a three-dimensional braided engineered matrix that Laurencin likens to a patch. [PR Newswire Association LLC] Press Release

ThermoGenesis Announces Sale of ThermoLine Product Line to Helmer Scientific
ThermoGenesis Corp. announced it has sold its ThermoLine plasma freezer and thawer product line to Helmer Scientific for an undisclosed sum of cash. The acquisition is part of Helmer’s growth strategy and a continuing commitment to providing high quality products to the medical and blood bank community. [ThermoGenesis Corp.] Press Release

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-31074)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-31451)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-31630)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-31739)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-31740)

Clinical Center; Notice of Meeting (FR Doc No: 2012-31741)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2012-31456)

Tissue Reference Group

November 15, 2012: Vaccines and Related Biological Products Advisory Committee Meeting Transcripts

November 14, 2012: Vaccines and Related Biological Products Advisory Committee Meeting Transcripts

November 14, 2012: Vaccines and Related Biological Products Advisory Committee Meeting Summary Minutes

Food and Drug Administration (United States)

Advisory Committees; Tentative Schedule of Meetings for 2013 (FR Doc No: 2012-31475)

Guidance for Industry and Food and Drug Administration Staff; Acceptance and Filing Reviews for Premarket Approval Applications; Availability (FR Doc No: 2012-31476)


Therapeutic Goods Administration (Australia)

Documents Released under Section 11C of the Freedom of Information Act 1982

NEW The Jackson Laboratory: How to Select and Maintain Mouse Models of Type 1 Diabetes
January 10, 1:00PM (ET in US)
Webinar

NEW Cell Culture World Congress 2013
February 26-28, 2013
Munich, Germany

NEW REGEN 2013 – Clinical Trials & Reimbursement
March 19-21, 2013
Boston, United States

NEW World Stem Cells & Regenerative Medicine Congress 2013
May 21-23, 2013
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.

Scientist or Engineer – hPSC Bioengineer (STEMCELL Technologies, Inc.)

MSc – Future Opportunities (STEMCELL Technologies, Inc.)

PhD – Future Opportunities (STEMCELL Technologies, Inc.)

Postdoctoral Positions in Cell Biology, Biophysics and Neurobiology (University of Missouri – Kansas City)

Senior QC Analyst/Assay Development Associate (StemCells, Inc.)

Postdoctoral Position (Sandra Rotman Centre for Global Health)

Postdoctoral Fellow – Molecular Biology (Cleveland Clinic Foundation)

Faculty Position – Stem Cell, Vascular and Developmental Biology in Ophthalmology (Zhongshan Ophthalmic Center, Sun Yat-Sen University)

Postdoctoral Position – Cardiovascular Regenerative Therapies (University of California, Los Angeles)

Cellular Therapy Laboratory Assistant Supervisor (Seattle Cancer Care Alliance)

Postdoctoral Researcher (The University of Texas Health Science Center at San Antonio)


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