Volume 13.34 | Sep 3

Issue 13.34 September 3, 2012
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Gene Therapy Rescues Cilia Defects and Restores Olfactory Function in a Mammalian Ciliopathy Model
Researchers found that adenoviral-mediated expression of IFT88 in mature, fully differentiated olfactory sensory neurons of mice with a hypomorphic mutation in the intraflagellar transport protein IFT88 is sufficient to restore ciliary structures and rescue olfactory function. These studies are the first to use in vivo therapeutic treatment to reestablish cilia in a mammalian ciliopathy. [Nat Med] Abstract | Press Release

Learn more about the new smartphone app for human blood cell frequencies

PUBLICATIONS (Ranked by impact factor of the journal)

HLA-C-Dependent Prevention of Leukemia Relapse by Donor Activating KIR2DS1
Scientists assessed clinical data, HLA genotyping results, and donor cell lines or genomic DNA for 1277 patients with acute myeloid leukemia who had received hematopoietic stem-cell transplants from unrelated donors matched for HLA-A, B, C, DR, and DQ or with a single mismatch. They performed donor killer-cell immunoglobulin-like receptor (KIR) genotyping and evaluated the clinical effect of donor KIR genotype and donor and recipient HLA genotypes. [N Engl J Med] Abstract | Press Release

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression
Researchers describe RNAi by single-stranded siRNAs (ss-siRNAs). Intraventricular infusion of ss-siRNA produced selective silencing of the mutant huntingtin allele throughout the brain in a mouse Huntington disease model. [Cell] Abstract | Press Release | Graphical Abstract

Single-Stranded siRNAs Activate RNAi in Animals
Researchers describe single-stranded siRNAs (ss-siRNAs) that silence gene expression in animals absent lipid formulation. [Cell] Abstract | Press Release | Graphical Abstract

Lymphoid Priming in Human Bone Marrow Begins Before Expression of CD10 with Upregulation of L-Selectin
Investigators report a unique lymphoid-primed population in human bone marrow that was generated from hematopoietic stem cells before onset of the expression of CD10 and commitment to the B cell lineage. [Nat Immunol] Abstract | Press Release

Formation and Optogenetic Control of Engineered 3D Skeletal Muscle Bioactuators
Densely arrayed skeletal myotubes are activated individually and as a group using precise optical stimulation with high spatiotemporal resolution. Skeletal muscle myoblasts are genetically encoded to express light-activated cation channel, Channelrhodopsin-2, which allows for spatiotemporal coordination of the multitude of skeletal myotubes that contract in response to pulsed blue light. Furthermore, ensembles of mature functional 3D muscle microtissues have been formed from the optogenetically encoded myoblasts using a high-throughput device. [Lab Chip] Abstract | Press Release | Video

Strong and Sustained Effector Function of Memory versus Naïve Derived T Cells upon T-Cell Receptor RNA Transfer: Implications for Cellular Therapy
Scientists redirected T cells of cytomegalovirus-seronegative donors with a human genetically engineered T-cell receptor recognizing an HLA-A*0201-binding peptide epitope of CMVpp65. To facilitate clinical translation of this approach, they used a non-viral expression system based on in vitro transcribed RNA and electroporation. [Eur J Immunol] Abstract

Pre-Emptive Immunotherapy with Purified Natural Killer Cells after Haploidentical SCT: A Prospective Phase II Study in Two Centers
In a prospective phase II study in two centers, investigators administered purified natural killer cell products to high-risk patients treated with haploidentical T-cell-depleted SCT. [Bone Marrow Transplant] Abstract

Direct Differentiation of Human Pluripotent Stem Cells into Haploid Spermatogenic Cells
Researchers demonstrated that human embryonic stem cells and induced pluripotent stem cells differentiate directly into advanced male germ cell lineages, including postmeiotic, spermatid-like cells, in vitro without genetic manipulation. [Cell Rep]
Abstract | Press Release

Register for Training Course: Air-Liquid Interface Culture of Primary Human Bronchial Epithelial Cells

Based on your feedback, we’ve added a Reviews section to Cell Therapy News

Stem Cell Therapies for Muscle Disorders
This review focuses on stem cell-based therapies to treat skeletal muscle disorders, with a special emphasis on muscular dystrophies. [Curr Opin Neurol] Abstract

Cardiovascular Surgery for Realization of Regenerative Medicine
Researchers review the recent basic and clinical research associated with the realization of regenerative medicine in the field of cardiovascular surgery. [Gen Thorac Cardiovasc Surg] Abstract

PneumaCult™-ALI Medium for Bronchial Epithelial Cells: Watch the Video


Stem Cell-Powered Implant Set to Revolutionize Orthopedic Surgery
Scientists are working to harness the regenerative power of stem cells to improve orthopedic implant surgery. The team have found a reliable method to encourage bone cell growth around a new type of implant. [University of Glasgow] Press Release

Celladon Corporation Announces First Patient Dosed in CUPID Phase IIb Trial of MYDICAR® for Advanced Heart Failure
Celladon Corporation announced that it has dosed the first patient in a Phase IIb clinical trial of MYDICAR. The Phase IIb study titled “Calcium Up-Regulation by Percutaneous Administration of Gene Therapy In Cardiac Disease” (“CUPID Phase IIb Trial”) is a multinational, multicenter, double-blind, placebo-controlled, randomized study of a single intracoronary administration of 1 x 1013 DRP MYDICAR versus placebo added to an optimal heart failure regimen. [Celladon Corporation] Press Release

Cell Culture World Congress USA 2012


International Collaboration Key to Science and Engineering Globalization
International collaboration is a key aspect of the globalization of science and engineering. A recent report and data evaluation showed that one in six scientists and engineers in the United States reported working with individuals in other countries in a given week. [National Science Foundation, United States] Press Release

Republican Party Platform Has a Lot to Say About Science
Addressing climate change and reducing demand for fossil fuels are out, and more neuroscience research and space science missions are in. Those shifts from 2008 are evident in the 2012 Republican Party Platform approved at the GOP’s national convention in Tampa, Florida. [Republican Party, United States] Press Release

African Cooperation ‘Dropped from EU Research Calls’
African scientists may be more likely to be left out of lucrative collaborations with European Union (EU) researchers, according to some policy experts. A mandate for EU research groups to include African partners in projects was dropped from the Seventh Framework Programme 2013 calls for proposals for EU competitive research grant. [Seventh Framework Programme, European Union] Press Release


Biomedical Miracles: Prestigious Partnership Funds Five New Health Care Innovations by MU Scientists
University of Missouri (MU) was chosen as one of 15 universities in the nation to enter into a Coulter Translational Partnership to take biomedical engineering products from the lab to the market. Now, University of Missouri announced five projects getting a nudge toward commercialization thanks to $5.2 million in Coulter funds. [University of Missouri] Press Release

NIH Awards $7.5 Million, Designates URMC Orthopedics as Center of Research Translation
The University of Rochester Medical Center (URMC) was granted a $7.5 million Center of Research Translation award for programs designed to find new therapies for arthritis and bone healing. Scientists will primarily investigate the regulation of stem cells in joint tissue and bone, and how tissues respond after musculoskeletal injury to knees and hips. [University of Rochester Medical Center] Press Release

ThermoGenesis Announces Completion of CryoSeal® Product Line Transaction with Asahi
ThermoGenesis Corp. announced that it has received the $2 million cash payment from Asahi Kasei Medical Co., Ltd., associated with the sale of the CryoSeal Fibrin Sealant System to Asahi. [ThermoGenesis Corp.]
Press Release

America Stem Cell, Inc. Initiates a Phase I/II Trial of ASC-101 in Patients with Hematologic Malignancies Undergoing Dual-Cord Transplantation
America Stem Cell, Inc. announced the initiation of a single-center study at The University of Texas MD Anderson Cancer Center evaluating ASC-101 in dual-umbilical cord transplantation in patients with hematologic malignancies. [Business Wire] Press Release

Northwest Bio Receives Regulatory Approval to Proceed with its Phase III Trial in the UK
Northwest Biotherapeutics announced that it has received approval from the Medicines and Healthcare Products Regulatory Agency of the United Kingdom (UK), for the Company’s 300-patient Phase III clinical trial of DCVax®-L immune therapy for Glioblastoma multiforme brain cancer to proceed in the UK. [Northwest Biotherapeutics, Inc.] Press Release

Arrowhead Completes Enrollment in Phase Ib Trial of RNAi Drug Candidate CALAA-01
Arrowhead Research Corporation announced that, through its Calando Pharmaceuticals subsidiary, it has completed enrollment of a phase Ib clinical trial of CALAA-01, its RNAi therapeutic candidate targeting solid tumors. [Arrowhead Research Corporation] Press Release

Pluristem Files for Orphan Drug Status with U.S. FDA for Use of PLX Cells in Treatment of Aplastic Anemia
Pluristem Therapeutics, Inc. announced that it has filed the necessary documents requesting that the U.S. Food and Drug Administration (FDA) grant orphan drug status to its PLacental eXpanded (PLX) cells for the treatment of aplastic anemia. [Pluristem Therapeutics, Inc.] Press Release

Dr. Andrew L. Kung Named Director of Pediatric Hematology, Oncology and Stem Cell Transplantation
Dr. Andrew L. Kung has joined the Department of Pediatrics at New York-Presbyterian Morgan Stanley Children’s Hospital/Columbia University Medical Center as director of the Division of Pediatric Hematology Oncology and Stem Cell Transplantation. [NewYork-Presbyterian Hospital] Press Release


Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-21501)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-21253)

National Institute of Allergy and Infectious Diseases; Notice of Closed Meetings (FR Doc No: 2012-21252)


NEW Stem Cells 2012
October 10-12, 2012
Mumbai, India

NEW International Society of Differentiation Conference: Stem Cells, Development and Regulation
November 5-8, 2012
Amsterdam, Netherlands

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


Scientist – Mesenchymal Cell Research (STEMCELL Technologies, Inc.)

Scientific Communications & Publishing Coordinator (STEMCELL Technologies, Inc.)

Quality Control Operations Coordinator (STEMCELL Technologies, Inc.)

Research Technologist – Research and Development (STEMCELL Technologies, Inc.)

Research Technologist – Media Development (STEMCELL Technologies, Inc.)

Postdoctoral Position – Induced Pluripotent Stem Cell Research (University of Washington)

Chief Scientific and Medical Officer (Living Cell Technologies)

Postdoctoral Fellow – Preclinical Group (BN ImmunoTherapeutics)

Postdoctoral Positions – Immunity and Inflammation (CNRS & University of Toulouse, France)

Postdoctoral Position – Pathophysiology of Brain Tumors (UMR 6301 ISTCT)

Research Associate – Quality Assurance (University of California)

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