Cell Therapy News Volume 20.31 | Sep 16 2019

TOP STORY

CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia

Scientists transplanted CRISPR-edited CCR5-ablated hematopoietic stem and progenitor cells into a patient with HIV-1 infection and acute lymphoblastic leukemia. The acute lymphoblastic leukemia was in complete remission with full donor chimerism, and donor cells carrying the ablated CCR5 persisted for more than 19 months without gene editing-related adverse events. [N Engl J Med] Abstract | Editorial

(Ranked by impact factor of the journal)

Targeting Cardiac Fibrosis with Engineered T Cells

Adoptive transfer of T cells that expressed a chimeric antigen receptor against fibroblast activation protein resulted in a significant reduction in cardiac fibrosis and restoration of function after injury in mice. [Nature] Abstract | Press Release

In Vivo Epigenetic Editing of Sema6a Promoter Reverses Transcallosal Dysconnectivity Caused by C11orf46/Arl14ep Risk Gene

C11orf46 binding normalized SEMA6A expression and rescued transcallosal dysconnectivity via repressive chromatin remodeling by the SETDB1 repressor complex. [Nat Commun] Full Article

Doxycycline Dependent Self-Inactivation of CRISPR-SpCas9 to Temporally Regulate On- and Off-Target Editing

The authors describe a ‘self-inactivating CRISPR’ system consisting of a single guide RNA that deactivated the Streptococcus pyogenes Cas9 nuclease in a doxycycline dependent manner. This enabled defined, temporal control of Cas9 activity in any cell-type and also in vivo. [Mol Ther] Abstract | Graphical Abstract

A NeuroD1 AAV-Based Gene Therapy for Functional Brain Repair after Ischemic Injury through In Vivo Astrocyte-to-Neuron Conversion

Investigators demonstrated that NeuroD1-mediated in situ astrocyte-to-neuron conversion could regenerate a large number of functional new neurons after ischemic injury. [Mol Ther] Abstract | Press Release | Full Article | Graphical Abstract

A Muscle Hybrid Promoter as a Novel Tool for Gene Therapy

Combined with AAV2/9, the muscle hybrid promoter provided a high in vivo expression level in the skeletal muscles and heart after both intramuscular and systemic delivery. [Mol Ther Methods Clin Dev] Abstract | Full Article

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

Long-term engraftment of vector-transduced hematopoietic cells was achieved in irradiated Artemis-deficient mice following primary and secondary transplantation. Vector-treated mice displayed T and B lymphopoiesis, polyclonal T cells, had structured lymphoid tissues and produced immunoglobulins. [Mol Ther Methods Clin Dev] Abstract | Full Article

Efficacy of Pulmonary Transplantation of Engineered Macrophages Secreting IL-4 on Acute Lung Injury in C57BL/6J Mice

Macrophages secreting interleukin-4 (M-IL-4) were established and used to treat acute lung injury through pulmonary macrophage transplantation. The results showed that highly sustained levels of IL-4 and M2 macrophage markers were detected in mice lungs following pulmonary M-IL-4 transplantation. [Cell Death Dis] Full Article

Clot-Derived Contaminants in Transplanted Bone Marrow Mononuclear Cells Impair the Therapeutic Effect in Stroke

The authors showed that bone marrow mononuclear cells (BM-MNCs) separated by an automatic cell isolator, which did not have the ability to remove clots, did not attenuate brain atrophy after stroke. In contrast, manually isolated, clot-free BM-MNC exerted therapeutic effects. [Stroke] Abstract

Improving the Function and Engraftment of Transplanted Pancreatic Islets Using Pulsed Focused Ultrasound Therapy

Following syngeneic renal sub-capsule islet transplantation in C57/B6 mice, pulsed focused ultrasound improved the function of transplanted islets with diabetic animals rapidly re-establishing glycemic control. [Sci Rep] Full Article

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CRISPR Technologies for Stem Cell Engineering and Regenerative Medicine

Scientists introduce the principles and recent advances of these CRISPR technologies, their delivery vectors and review their applications in stem cell engineering and regenerative medicine. They focus on in vitro stem cell fate manipulation and in vivo applications such as prevention of retinal and muscular degeneration, neural regeneration, bone regeneration, cartilage tissue engineering, as well as treatment of diseases in blood, skin and liver. [Biotechnol Adv] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Be The Match BioTherapies^® and Celyad Announce Collaboration to Advance Development of Allogeneic CAR-T Therapies

Be The Match BioTherapies^® and Celyad announced a collaboration to advance the clinical trial of Celyad’s non-gene edited allogeneic CAR-T therapies. [Be The Match BioTherapies] Press Release

Gamida Cell and the CIBMTR Announce Collaboration to Advance Research for Life-Saving Cellular Therapy

Gamida Cell Ltd. announced the entry into a research agreement to collect and analyze health outcomes data in patients with hematologic malignancies who receive an allogeneic hematopoietic stem cell transplant from various donor sources. [Gamida Cell Ltd.] Press Release

Trucode Launched to Advance Next-Generation Gene Editing Platform

Trucode Gene Repair, Inc. announced that it is advancing its next-generation in vivo gene editing technology to correct mutations that cause sickle cell disease, cystic fibrosis, and other devastating genetic disorders. [Trucode Gene Repair, Inc.] Press Release

CBMG Hosts Meeting with Investigators to Launch AlloJoin^® Knee Osteoarthritis Phase II Clinical Trial

Cellular Biomedicine Group Inc. announced that the company held a meeting with investigators in Shanghai to launch the Phase II clinical trial for AlloJoin^®, an “off-the-shelf” allogenic human adipose-derived mesenchymal progenitor cell therapy product targeting knee osteoarthritis. [Cellular Biomedicine Group Inc.] Press Release

Momotaro-Gene Announces First Patient Dosed in Phase II Clinical Trial of MTG201 in Combination with Nivolumab in Patients with Relapsed Malignant Pleural Mesothelioma

Momotaro-Gene announced the dosing of the first patient in a Phase II clinical trial combining MTG201, the company’s lead therapeutic candidate, with the PD-1 inhibitor nivolumab in patients with relapsed malignant pleural mesothelioma. [Momotaro-Gene Inc. (GlobeNewswire, Inc.)] Press Release

DiscGenics’ US Clinical Study of Cell Therapy for Disc Degeneration Passes Halfway Mark

DiscGenics, Inc. announced that the first 30 subjects have been safely treated its first-in-human US clinical study of IDCT, an allogeneic, injectable disc cell therapy for mild to moderate degenerative disc disease. [DiscGenics, Inc.] Press Release

Prevail Therapeutics Provides Program Update on PR001 in Parkinson’s Disease with GBA1 Mutations and Neuronopathic Gaucher Disease

Prevail Therapeutics Inc. provided an update on the company’s clinical development of PR001, a potentially disease-modifying, single-dose gene therapy in development for Parkinson’s disease with GBA1 mutations and neuronopathic Gaucher disease. [Prevail Therapeutics Inc.] Press Release

Science under Maximum Pressure in Iran

From travel restrictions and publishing bans to currency collapse, the restoration of US sanctions has left researchers in Iran reeling. [The Scientist] Editorial

EU Research Commissioner Named, but Lacks ‘Research’ in Her Title

Mariya Gabriel, a conservative politician from Bulgaria, is slated to become the next European research chief- although her job title, unlike that of her predecessors, doesn’t include the words “research” or “science”. [ScienceInsider] Editorial

NEW 8^th Annual International Symposium on Regenerative Rehabilitation
October 24-26, 2019
Charlottesville, Virginia, United States

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NEW Research Assistant – Cancer Research (BC Cancer Agency)

Research Technologist (STEMCELL Technologies Inc.)

Scientific Communications Coordinator (STEMCELL Technologies Inc.)

Postdoctoral Fellow – Cancer Research (Baylor College of Medicine)

Postdoctoral Fellow – Molecular Genetic Basis of Behavioral Variation (University of California, San Francisco)

Postdoctoral Fellow – Cancer Therapy (Purdue University)

Postdoctoral Researcher – Cell Cycles in Stem Cells (University of California, Santa Cruz)

Postdoctoral Fellowship – Cancer Immunotherapy (McGill University)

Postdoctoral Position – Gene Therapy of Muscular Dystrophy (Ohio State University)

Postdoctoral Scholar – Gene and Cell Therapy (Stanford University)

Postdoctoral Scientist – HSV Vectors as a Therapy for Nervous System Tumors (Nationwide Children’s Hospital)

Research Scientist – Cellular Electrophysiology (Duke University)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center)

Tenure Track Faculty Position – Cancer Biology & Therapeutics (Geisel School of Medicine at Dartmouth)

Assistant/Associate or Professor – Cancer Immunotherapy/Tumor Immunology (University of California, Irvine)

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