Cell Therapy News Volume 21.09 | Mar 16 2020

TOP STORY

Therapeutic Base Editing of Human Hematopoietic Stem Cells

The authors demonstrated the potential of ribonucleoprotein base editing of human hematopoietic stem and progenitor cells (HSPCs) as a feasible alternative to nuclease editing for HSC-targeted therapeutic genome modification. [Nat Med] Abstract

(Ranked by impact factor of the journal)

An Engineered Cell-Laden Adhesive Hydrogel Promotes Craniofacial Bone Tissue Regeneration in Rats

In a well-established rat peri-implantitis model, application of the adhesive hydrogel encapsulating gingival mesenchymal stem cells resulted in complete bone regeneration around ailing dental implants with peri-implant bone loss. [Sci Transl Med] Abstract

CRISPR/Cas9 Mediated miR-29b Editing as a Treatment of Different Types of Muscle Atrophy in Mice

In vivo, CRISPR-based treatment prevented the muscle atrophy induced by angiotensin II (AngII), immobilization and denervation via activation of AKT-FOXO3A-mTOR signaling pathway and protected against AngII-induced myocyte apoptosis in mice, leading to significantly increased exercise capacity. [Mol Ther] Abstract | Graphical Abstract | Full Article

Glycine Enhances Satellite Cell Proliferation, Cell Transplantation and Oligonucleotide Efficacy in Dystrophic Muscle

Researchers demonstrated that co-administered glycine improved phosphorodiamidate morpholino oligomer (PMO) potency in mdx mice with marked functional improvement and up to 50-fold increase of dystrophin in abdominal muscles compared to PMO in saline. [Mol Ther] Abstract | Graphical Abstract

Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH

A family of hepatotropic Clade F adeno-associated viruses was isolated from human CD34⁺ hematopoietic stem cells and one was utilized to deliver a vector to correct the phenylketonuria phenotype in Pah^enu2 mice. [Mol Ther Methods Clin Dev] Abstract | Full Article

Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A

Researchers investigated the suitability of human placental cells as delivery vehicles for FVIII, and determined an optimal FVIII transgene to produce/secrete therapeutic FVIII levels from these cells. [Mol Ther Methods Clin Dev] Abstract | Graphical Abstract | Full Article

Therapeutic Mechanism of Cord Blood Mononuclear Cells via the IL-8-Mediated Angiogenic Pathway in Neonatal Hypoxic-Ischemic Brain Injury

Human umbilical cord blood mononuclear cell administration restored the sequential downstream signaling axis of p-p38/p-MAPKAPK2, NFκB, and angiogenic factors, which were downregulated by hypoxic-ischemic brain injury. [Sci Rep] Full Article

Syngeneic Mesenchymal Stem Cells Reduce Immune Rejection after Induced Pluripotent Stem Cell-Derived Allogeneic Cardiomyocyte Transplantation

To determine whether mesenchymal stem cell (MSC) co-transplantation could reduce immune rejection after allogeneic induced pluripotent stem cell-derived cardiomyocyte transplantation, the latter cell type, harboring a luciferase transgene, was subcutaneously transplanted alone or together with syngeneic MSCs into BALB/c mice. [Sci Rep] Full Article

Cardiopoietic Stem Cell Therapy Restores Infarction-Altered Cardiac Proteome

Cell therapy non-stochastically reversed a majority of infarction-provoked changes, remediating 85% of disease-affected protein clusters. Pathway and network analysis decoded functional reorganization, distinguished by prioritization of vasculogenesis, cardiac development, organ regeneration, and differentiation. [NPJ Regen Med] Full Article | Press Release

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Anti-CRISPR Protein Applications: Natural Brakes for CRISPR-Cas Technologies

Scientists discuss applications of anti-CRISPR proteins for post-translational control of CRISPR-Cas systems in prokaryotic and mammalian cells, organisms and ecosystems. [Nat Methods] Abstract

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

Mesoblast to Evaluate Anti-Inflammatory Cell Therapy Remestemcel-L for Treatment of COVID-19 Lung Disease

Mesoblast Limited announced that it plans to evaluate its allogeneic MSC product candidate remestemcel-L in patients with acute respiratory distress syndrome caused by coronavirus in the United States, Australia, China and Europe. [Mesoblast Limited] Press Release

Kuur Therapeutics Launches to Develop and Commercialize “Off-the-Shelf” CAR-NKT Cell Therapies Targeting Hematological and Solid Tumors

Kuur Therapeutics announced the launch of its new business in partnership with Baylor College of Medicine and Baylor’s Center for Cell and Gene Therapy. Kuur Therapeutics will advance the work of its predecessor, Cell Medica, to develop anti-cancer therapies using its innovative chimeric antigen receptor natural killer T cell (CAR-NKT) therapy platform. [Kuur Therapeutics] Press Release

GeneTx and Ultragenyx Announce First Patient Dosed in Phase I/II Clinical Trial of GTX-102 in Patients with Angelman Syndrome

GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. announced that it has dosed the first patient in its Knockdown of UBE3A-antisense in Kids with Angelman Syndrome study of GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome. [GeneTx Biotherapeutics LLC (GlobeNewswire, Inc.)] Press Release

UK Government to Boost R&D Spending

The British government has announced a 15 percent increase in public funding for research and development as part of its 2020 budget in a move that was generally welcomed by scientists. [The Scientist] Editorial

Universities Are Forging Ties with the FBI as US Cracks Down on Foreign Influence

US universities are forging closer ties with FBI agents, encouraging scientists to disclose foreign sources of research funding, and tightening restrictions on researchers’ travel. [Nature News] Editorial

NEW Cambridge International Stem Cell Symposium 2020
September 27-29, 2020
Cambridge, United Kingdom

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

NEW Scientist – Cancer Immunotherapy (Lund University)

Postdoctoral Position – Cancer Immunotherapy (The University of Texas MD Anderson Cancer Center)

Sr Associate Scientist – Autoimmunity and Tumor Immunotherapy (Amgen)

Research Associate – Stem Cell and Gene Therapy Program (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – Inflammation and Cancer (University Hospital of Erlangen)

Lecturer – Clinical Pharmacology (University of Bristol)

Research Assistant – Organoid Modeling for Stem Cell Therapies (Boston Children’s Hospital)

Postdoctoral Fellow – Cancer Research (BC Cancer)

Research Assistant – Cancer Research (BC Cancer)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer)

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