Volume 14.01 | Jan 14

Issue 14.01 January 14, 2013
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Researchers Regenerate Sensory Hair Cells, Restore Hearing to Noise-Damaged Ears
Researchers demonstrated for the first time that hair cells can be regenerated in an adult mammalian ear by using a drug to stimulate resident cells to become new hair cells, resulting in partial recovery of hearing in mouse ears damaged by noise trauma. [Press release from Massachusetts Eye and Ear discussing online prepublication in Neuron] Press Release | Abstract | Video Abstract

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PUBLICATIONS (Ranked by impact factor of the journal)

RNA-Guided Human Genome Engineering via Cas9
Researchers engineered the type II bacterial clustered regularly interspaced short palindromic repeats system to function with custom guide RNA in human cells. Results establish an RNA-guided editing tool for facile, robust, and multiplexable human genome engineering. [Science] Abstract | Press Release

Dynamic Chromatin Remodeling Mediated by Polycomb Proteins Orchestrates Pancreatic Differentiation of Human Embryonic Stem Cells
Scientists profiled the transcriptome and select chromatin modifications at defined stages during pancreatic endocrine differentiation of human embryonic stem cells. In vivo– but not in vitro-differentiated endocrine cells exhibit close similarity to primary human islets in regard to transcriptome and chromatin structure. [Cell Stem Cell] Abstract | Graphical Abstract | Press Release

Molecular Imaging of the Paracrine Proangiogenic Effects of Progenitor Cell Therapy in Limb Ischemia
Using molecular imaging, the authors tested the hypothesis that treatment of limb ischemia with multipotential adult progenitor cells promotes recovery of blood flow through the recruitment of pro-angiogenic monocytes. [Circulation] Abstract

Intrathymic Progenitor Cell Transplantation across Histocompatibility Barriers Results in the Persistence of Early Thymic Progenitors and T Cell Differentiation
Researchers previously showed that the forced intrathymic (IT) administration of histocompatible hematopoietic stem cells can sustain long-term thymopoiesis in ZAP-70-immunodeficient mice. In the absence of conditioning, long term thymopoiesis by semiallogeneic progenitors was detected in IT, but not IV, transplanted mice. In IT-transplanted mice, ongoing thymopoiesis was associated with a ten-fold higher level of early thymic progenitors. [Blood] Abstract

Fetal Membrane Cells for Treatment of Steroid-Refractory Acute Graft-versus-Host Disease
Investigators tested fetal membrane cells for treatment of steroid-refractory acute graft-versus-host disease. [Stem Cells] Abstract

Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in Spinal Muscular Atrophy Mice
Researchers investigated whether gene delivery to motor neurons (MNs) could also be achieved via intramuscular self-complementary (sc) AAV9 injection and subsequent retrograde transport along the MNs axons. Unexpectedly, they found that a single injection of scAAV9 into the adult mouse gastrocnemius mediated widespread MN transduction along the whole spinal cord, without limitation to the MNs connected to the injected muscle. [Mol Ther] Abstract

Endothelial Reconstitution by CD34+ Progenitors Derived from Baboon Embryonic Stem Cells
Researchers used a large non-human primate model, the baboon, to establish a step-wise protocol to generate CD34+ endothelial progenitor cells from embryonic stem cells and to demonstrate their reparative effects. [J Cell Mol Med] Abstract | Full Article | Press Release

Sickle Erythrocytes Target Cytotoxics to Hypoxic Tumor Microvessels and Potentiate a Tumoricidal Response
Investigators exploited a hitherto unrecognized ability of sickled erythrocytes (SSRBCs) but not normal RBCs (NLRBCs) to selectively target hypoxic tumor vascular microenvironment and induce diffuse vaso-occlusion. Within minutes after injection SSRBCs, but not NLRBCs, home and adhere to hypoxic 4T1 tumor vasculature with hemoglobin saturation levels at or below 10% that are distributed over 70% of the tumor space. [PLoS One]
Full Article | Press Release

Biomimetic Three-Dimensional Anisotropic Geometries by Uniaxial Stretch of Poly(ɛ-Caprolactone) Films for Mesenchymal Stem Cell Proliferation, Alignment, and Myogenic Differentiation
Researchers present a novel simple, solvent-free, and reproducible method via uniaxial stretching for incorporating anisotropic topographies on bioresorbable films with ambitions to realize stem cell alignment control. Compared to unstretched films, human mesenchymal stem cells on uniaxial-stretched poly(ɛ-caprolactone) had larger increase in cellular alignment and elongation, without indication of cytotoxicity or reduction in cellular proliferation. [Tissue Eng Part C Methods] Abstract | Press Release

Combining T-Cell Immunotherapy and Anti-Androgen Therapy for Prostate Cancer
Investigators demonstrated that T cells can be genetically engineered to express a chimeric antigen receptor (CAR) targeting the tumor-associated antigen Muc1. CAR-Muc1 T cells were able to selectively kill Muc1-expressing human prostate cancer cells. [Prostate Cancer Prostatic Dis] Abstract

New StemSpan(TM) SFEM II - for optimal CD34+ cell expansion of bone marrow and cord blood cells


IL-27 in Tumor Immunity and Immunotherapy
In this review, the authors argue that by virtue of its rate-limiting functions in innate and adaptive immune responses, modulating interleukin (IL)-27 holds considerable promise for future cancer immunotherapy. [Trends Mol Med] Abstract

Recent Developments in Oncolytic Adenovirus-Based Immunotherapeutic Agents for Use against Metastatic Cancers
This review focuses on the most recent updates in strategies to develop potent oncolytic adenovirus-based immunotherapeutics for use in cancer gene therapy. [Cancer Gene Ther] Abstract

New: Directed Differentiation of Pluripotent Stem Cells. A Cell Stem Cell Poster


Stem Cells May Hold Promise for Lou Gehrig’s Disease
Apparent stem cell transplant success in mice may hold promise for people with amyotrophic lateral sclerosis, or Lou Gehrig’s disease. The study found that stem cell transplantation significantly extended the lifespan of the mice by 20 days and improved their neuromuscular function by 15 percent. [Press release from the American Academy of Neurology discussing research to be presented at the American Academy of Neurology’s 65th Annual Meeting, San Diego] Press Release

Mesoblast’s NeoFuse Stem Cell Product Shows Positive Results in Phase II Lumbar Spinal Fusion Trial
Mesoblast Limited announced that its Phase II clinical trial for lumbar spinal fusion had successfully met its safety and efficacy endpoints. The results suggest that Mesoblast’s NeoFuse product comprising allogeneic Mesenchymal Precursor Cells is as effective for interbody lumbar fusion as the gold standard, bone autograft, without the need for a second surgical procedure and its attendant morbidity risks. [Mesoblast Limited]
Press Release

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Japan Stimulus to Boost Science
Japan’s government approved a plan to spend $116 billion to jump-start the economy and set the stage for long-term growth. Sources in the Japanese press are hinting that research on renewable energy and on stem cells could land a significant chunk of the new cash. [Japanese Government, Japan] Press Release

Surprise Choices Mark New Leadership on U.S. House Science Panel
High turnover is not unusual within the Committee on Science, Space, and Technology of the House of Representatives, which ranks low on the totem pole of powerful committees. Even so, Representative Lamar Smith, the new chair of the full 40-member committee, has made some surprising decisions in selecting the chairpersons of the panel’s six subcommittees. [House of Representatives, United States] Press Release


NeoStem’s Subsidiary, Progenitor Cell Therapy, and Hackensack University Medical Center Enter into a Cell Processing and Storage Services Agreement
NeoStem, Inc. and its subsidiary, Progenitor Cell Therapy, LLC (PCT) and Hackensack University Medical Center (HackensackUMC) announced that HackensackUMC has renewed its engagement of PCT’s services for processing and storage of a variety of cell types used therapeutically by the hospital. HackensackUMC will use the services of PCT for processing and storage services for peripheral blood progenitor cells, donor leukocytes, bone marrow and cord blood, as well as for further assays, storage, retrieval and transportation. [NeoStem, Inc.] Press Release

New Company Applies Regenerative Medicine to Corneal Transplantation
Ocular Systems, Inc., Wake Forest Baptist Medical Center and the North Carolina Eye Bank have formed a new company based on a promising new technology aimed at engineering replacement corneas in the lab for transplantation. The new approach, not yet tested in patients, involves isolating cells from “banked” donor corneas to grow replacement corneal tissue in the lab. [Wake Forest Baptist Medical Center] Press Release

George Huang of the University of Tennessee Health Science Center Continues Research for Root Canal Treatment with $1.8 Million Grant Funding
The concept of trying to regenerate growth of a tooth by inserting stem cell tissue has been around since the 60s, but little progress has been made. If Dr. Huang’s research is successful, the canals that have been cleared would be injected with stem cell tissue. Not only would this technique avoid the need for the rubber-like filling, it could actually stimulate healthy tissue growth, essentially rebuilding the tooth. [The University of Tennessee Health Science Center] Press Release

America Stem Cell, Inc. Awarded a Phase I SBIR to Explore the Therapeutic Potential of Its Platform Technology (ASC-101) with Neural Stem Cells in Multiple Sclerosis Model
America Stem Cell, Inc. (ASC) announced that it has been awarded an Advanced Technology Small Business Innovation Research (SBIR) grant from the National Heart Lung and Blood Institute at the National Institutes of Health. The studies outlined within the presently approved grant will examine the effect of ASC’s lead technology, ASC-101, in combination with neural stem cells in an experimental mouse model of multiple sclerosis. [Business Wire] Press Release

U.S.-Based Gene Therapy Study Begins Treating Participants
Cooley’s Anemia Foundation has learned that the first U.S.-based clinical trial in gene therapy for beta-thalassemia has begun treating trial participants. The Phase I study, the official title of which is “ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene,” is being conducted at Memorial Sloan-Kettering Cancer Center in Manhattan. [Cooley’s Anemia Foundation, Inc.] Press Release

BioTime Submits Protocol for Initiation of Human Clinical Trials of Renevia™ in Europe
BioTime, Inc. announced it has submitted a Clinical Investigation Protocol to European regulatory authorities for approval to initiate studies for its Renevia™ stem cell delivery platform. [BioTime, Inc.] Press Release

Regenerative Medicine: Clinical Trials Launched for the Treatment of Ununited Fractures
The REBORNE project (Regenerating Bone Defects using New biomedical Engineering approaches), funded by the European Commission and coordinated by Inserm, has just received the green light from the French Medicinal Agency ANSM to begin orthopedic surgery clinical trials that aim to repair bones using adult stem cells combined with biomaterials. [INSERM] Press Release

Genea Stem Cells (GSC): 25 New Disease Specific Pluripotent Stem Cell Lines Placed on the NIH Registry
Genea Stem Cells Pty Ltd announced that 25 of its disease specific embryonic pluripotent stem cell lines have been placed on the USA National Institutes of Health (NIH) human stem cell registry. These embryonic stem cell lines are now all available commercially for use in medical research. [Business Wire] Press Release


Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-00282)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-00173)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-00088)

Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc No: 2013-00089)


January 15, 2013: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Agenda

January 15, 2013: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Draft Roster

2012 Biological Device Application Approvals


Food and Drug Administration (United States)

Blood Products Advisory Committee; Notice of Meeting (FR Doc No: 2013-00491)

Therapeutic Goods Administration (Australia)

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

Products Regulated as Biologicals


NEW The Jackson Laboratory: Modeling Type 2 Diabetes Complications
January 17, 1:00PM (ET in US)

NEW The Jackson Laboratory: Beyond Leptin – Emerging Mouse Models of Type 2 Diabetes
January 24, 1:00PM (ET in US)

NEW The 3rd International Congress on Controversies in Cryopreservation of Stem Cells, Reproductive Cells, Tissue and Organs (CRYO)
March 21-23, 2013
Berlin, Germany

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.)

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)

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

Postdoctoral Fellow – Transcriptional Regulation in CD8 T lymphocytes (Université Libre de Bruxelles – Institute for Medical Immunology)

Postdoctoral Researcher Position in Bioinformatics (University of Oulu)

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