Cell Therapy News Volume 19.31 | Sep 17 2018

TOP STORY

In Vivo CRISPR Editing with No Detectable Genome-Wide Off-Target Mutations

Investigators describe ‘verification of in vivo off-targets’, a highly sensitive strategy that can robustly identify the genome-wide off-target effects of CRISPR-Cas nucleases in vivo. [Nature] Abstract | Editorial

(Ranked by impact factor of the journal)

TRIM21 Mediates Antibody Inhibition of Adenovirus-Based Gene Delivery and Vaccination

Researchers showed that antibody prevention of adenoviral gene delivery in vivo was mediated by the cytosolic antibody receptor TRIM21. Genetic KO of TRIM21 or a single-antibody point mutation was sufficient to restore transgene expression to near-naïve immune levels. [Proc Natl Acad Sci USA] Full Article

MSC-Seeded Biomimetic Scaffolds as a Factory of Soluble RANKL in Rankl-Deficient Osteopetrosis

Magnesium-doped hydroxyapatite/collagen I-seeded murine MSCs showed improved properties, as compared to two-dimensional culture, in terms of proliferation and human soluble RANKL (hsRL) production, with respect to lentiviral transduction‐transduced cells. When implanted subcutaneously in Rankl^−/− mice, these cell constructs were well tolerated, colonized by host cells, and intensely vascularized. [Stem Cells Transl Med] Full Article

AAV Serotype 8-Mediated Liver Specific GNMT Expression Delays Progression of Hepatocellular Carcinoma and Prevents Carbon Tetrachloride-Induced Liver Damage

The authors investigated the activity of ectopic glycine N-methyltransferase (GNMT) delivered using recombinant adeno-associated virus gene therapy in mouse models of liver cirrhosis and hepatocellular carcinoma. [Sci Rep] Full Article

Therapeutic Effect of a Xeno-Free Three-Dimensional Stem Cell Mass in a Hind Limb Ischemia Model

Scientists describe an innovative method of culturing a three-dimensional stem cell mass (3DCM) in the xeno-free (XF) condition and demonstrate the therapeutic potential and safety of XF-3DCMs in ischemic mice and in SCID/BALB/c Slc-nu/nu nude mice, respectively. [Tissue Eng Part A] Abstract

Genome-Edited Skin Epidermal Stem Cells Protect Mice from Cocaine-Seeking Behavior and Cocaine Overdose

Researchers showed that the transplantation of skin cells into mice modified the expression of an enhanced form of butyrylcholinesterase—an enzyme that hydrolyses cocaine—that enabled the long-term release of the enzyme and efficiently protected the mice from cocaine-seeking behavior and cocaine overdose. [Nat Biomed Eng] Abstract | Editorial

In Vivo Conversion of Astrocytes into Oligodendrocyte Lineage Cells with Transcription Factor Sox10; Promise for Myelin Repair in Multiple Sclerosis

Investigators report that a single transcription factor Sox10 could reprogram astrocytes into oligodendrocyte-like cells, in vivo. For transdifferentiation, Sox10-GFP expressing viral particles were injected into cuprizone-induced demyelinated mice brains after which they assessed for the presence of specific oligodendrocyte lineage cell markers by immunohistofluorescence. [PLoS One] Full Article

Mesenchymal Stem Cell Treatment Attenuates Liver and Lung Inflammation after Ethanol Intoxication and Burn Injury

The authors determined whether intravenous infusion of exogenous bone marrow-derived mesenchymal stem cells could reduce post-burn and intoxication pulmonary, hepatic, and systemic inflammation. [Alcohol] Abstract

Transplanted Enteric Neural Stem Cells Integrate within the Developing Chick Spinal Cord: Implications for Spinal Cord Repair

Following enteric neural stem cells transplantation to an ablated region of chick embryo spinal cord (SC), donor neurons were found up to 12 days later. These neurons formed bridging connections within the SC injury zone, aligned along the anterior/posterior axis, and were immunopositive for TuJ1. [J Anat] Full Article

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Application of CRISPR/Cas9 Technologies Combined with iPSCs in the Study and Treatment of Retinal Degenerative Diseases

The authors compare genome-editing strategies and highlight the advantages and concerns of the CRISPR/Cas9 system. Moreover, the latest progress and applications of the CRISPR/Cas9 system and its combination with iPSCs for the treatment of retinal degenerative diseases are summarized. [Hum Genet] Abstract

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

CRISPR Therapeutics and ViaCyte Announce Strategic Collaboration to Develop Gene-Edited Stem Cell-Derived Therapy for Diabetes

CRISPR Therapeutics and ViaCyte, Inc. announced a collaboration focused on the discovery, development, and commercialization of gene-edited allogeneic stem cell therapies for the treatment of diabetes. [CRISPR Therapeutics] Press Release

Vineti Partners with World Courier to Provide State-of-the-Art Supply Chain Solution for Global Cell and Gene Therapy Ecosystem

Vineti, Inc. and World Courier announced a partnership to further extend the reach of cell and gene therapies world-wide. [Vineti, Inc. (PR Newswire Association LLC.)] Press Release

Stevenage Cell and Gene Therapy Catapult Center Gets Green Light to Make New Cell and Gene Therapies

New life-changing cell and gene therapies for use in health services around the world will be produced in Stevenage by innovative biotech firms as the Cell and Gene Therapy Catapult is granted new licences. [Cell and Gene Therapy Catapult] Press Release

Gilead Sciences and Precision BioSciences Announce Collaboration to Develop Therapies Against Hepatitis B Virus Using ARCUS Genome Editing

Gilead Sciences and Precision BioSciences announced that the companies have entered into a strategic collaboration to develop therapies targeting the in vivo elimination of hepatitis B virus with Precision’s proprietary genome editing platform, ARCUS. [Gilead Sciences] Press Release

Abeona Therapeutics Announces Authorization to Commence Phase I/II Gene Therapy Clinical Study for Patients with MPS IIIB in Spain

Abeona Therapeutics Inc. announced authorization to move forward with a Phase I/II clinical trial in Spain for the company’s gene therapy product ABO-101 for patients with Sanfilippo syndrome type B (MPS IIIB). [Abeona Therapeutics Inc.] Press Release

FACIT Invests in Three Top Ontario Entrepreneur-Teams from Near Record Pool of Applicants

FACIT has selected three novel cancer therapeutic discoveries to receive early-stage capital – biotechnology start-up Talon Pharmaceuticals, the Drug Discovery team at the Ontario Institute for Cancer Research and CCRM. [FACIT] Press Release

Engineered Natural Killer Cells May Be the Next Great Cancer Immunotherapy

The cancer fighters known as CAR T cells have proved their prowess in recent years. Three therapies using the altered T cells against lymphoma or leukemia have won FDA approval, and hundreds of trials are now unleashing them on other malignancies, including solid tumors. [ScienceInsider] Editorial

Boehringer Ingelheim Acquires All ViraTherapeutics Shares to Develop Next Generation Viral-Based Immuno-Oncology Therapies

Boehringer Ingelheim announced that it has acquired all shares of ViraTherapeutics, a biopharmaceutical company specializing in the development of oncolytic viral therapies. [Boehringer Ingelheim International GmbH] Press Release

Bone Therapeutics Announces Final Results from Phase I/IIA ALLOB Delayed-Union Fracture Study

Bone Therapeutics S.A. announced positive final results in the Phase I/IIA delayed-union study of its allogeneic bone cell therapy product ALLOB in 21 patients, supporting the future clinical development of the delayed union indication. [Bone Therapeutics S.A.] Press Release

NIH Launches Initiative to Accelerate Genetic Therapies to Cure Sickle Cell Disease

The National Institutes of Health (NIH) announced the launch of a new initiative to help speed the development of cures for sickle cell disease. The Cure Sickle Cell Initiative will take advantage of the latest genetic discoveries and technological advances to move the most promising genetic-based curative therapies safely into clinical trials within five to ten years. [National Institutes of Health] Press Release

Engineered Natural Killer Cells May Be the Next Great Cancer Immunotherapy

The cancer fighters known as CAR T cells have proved their prowess in recent years. Three therapies using the altered T cells against lymphoma or leukemia have won FDA approval, and hundreds of trials are now unleashing them on other malignancies, including solid tumors. [ScienceInsider] Editorial

Evidence-Based Medicine Group in Turmoil after Expulsion of Co-Founder

A bitter dispute with one of its co-founders has plunged Cochrane, an international network of scientists promoting evidence-based medicine, into a crisis on the eve of an international gathering that marks its 25^th anniversary. A narrow majority of the organization’s Governing Board apparently decided to end the Cochrane membership of Peter Gøtzsche, director of the Nordic Cochrane Centre in Copenhagen and a member of the board himself, for causing “disrepute” to the organization. Four other board members then resigned in protest. [ScienceInsider] Editorial

NIH Set to Strengthen Its Sexual-Harassment Policies

The US National Institutes of Health (NIH) is remaking how it handles allegations of harassment by its employees. The agency will soon introduce a centralized system for reporting harassment by NIH scientists, director Francis Collins said. [Nature News] Editorial

NIH

Prospective Grant of an Exclusive Patent License: Photoactivatable Liposomal Nanoparticle for the Delivery of an Immunotherapeutic or Immunotherapeutic-Enabling Agent (FR Doc. No:2018-19604) Notice

NEW LabRoots: 1^st Annual CRISPR Virtual Event
October 3, 2018
Online

Visit our events page to see a complete list of events in the community.

NEW Director – Clinical Development (California Institute for Biomedical Research)

NEW Postdoctoral Research Assistant – Gene Therapy (University of Oxford)

NEW Faculty Position – Adoptive T Cell Therapy (Dana-Farber Cancer Institute)

Professorship – Somatic Gene Therapy (University of Zurich)

Postdoctoral Staff Fellow – Biologist/Biomedical Engineer (Food and Drug Administration)

Associate Professor – Cancer Research (University of Maryland)

Senior Scientist – CAR-T Cell Research (CELLECTIS Inc.)

Postdoctoral Fellow – Immuno-Oncology (CELLECTIS Inc.)

Research Assistant or Associate – Cell Therapy (California Institute for Biomedical Research)

Postdoctoral Scientist – Novel T Cell Based Cancer Therapies (Netherlands Cancer Institute – Antoni van Leeuwenhoek)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

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