Cell Therapy News Volume 21.06 | Feb 24 2020

TOP STORY

Initial Results from a First-in-Human Gene Therapy Trial on X-Linked Retinitis Pigmentosa Caused by Mutations in RPGR

Apart from steroid-responsive subretinal inflammation in patients at the higher doses, there were no notable safety concerns after subretinal delivery of an adeno-associated viral vector encoding codon-optimized human retinitis pigmentosa GTPase regulator (RPGR), meeting the pre-specified primary endpoint. [Nat Med] Abstract | Press Release

(Ranked by impact factor of the journal)

Generation of Human Endothelium in Pig Embryos Deficient in ETV2

ETV2-null blastocysts were injected with human induced pluripotent stem cells overexpressing the antiapoptotic factor BCL2, transferred to synchronized gilts and analyzed between embryonic day 17 and embryonic day 18. In these embryos, all endothelial cells were of human origin. [Nat Biotechnol] Abstract

Synaptotagmin 13 Is Neuroprotective across Motor Neuron Diseases

Gene therapy with Syt13 prolonged the lifespan of amyotrophic lateral sclerosis mice by 14% and spinal muscular atrophy mice by 50% by preserving motor neurons and delaying muscle denervation. [Acta Neuropathol] Full Article

Neural Cell Integration into 3D Bioprinted Skeletal Muscle Constructs Accelerates Restoration of Muscle Function

Researchers investigated the effects of neural cell integration into the bioprinted skeletal muscle construct to accelerate functional muscle regeneration in vivo. The bioprinted constructs with neural cell integration facilitated rapid innervation and matured into organized muscle tissue that restored normal muscle weight and function in a rodent model of muscle defect injury. [Nat Commun] Full Article

Engineering Light-Controllable CAR T Cells for Cancer Immunotherapy

Researchers developed a light-inducible nuclear translocation and dimerization system for gene regulation to control CAR T activation. They demonstrated light-controllable gene expression and functional modulation in human embryonic kidney 293T and Jurkat T cell lines. [Sci Adv] Full Article | Editorial

A Mutation-Independent CRISPR-Cas9-Mediated Gene Targeting Approach to Treat a Murine Model of Ornithine Transcarbamylase Deficiency

Investigators describe a dual-adeno-associated virus vector system that accomplished rapid short-term expression from a non-integrated minigene and long-term expression from the site-specific integration of this minigene without any selective growth advantage for ornithine transcarbamylase-positive cells in newborns. [Sci Adv] Full Article

Gene Therapy Using Neural Stem/Progenitor Cells Derived from Human Induced Pluripotent Stem Cells: Visualization of Migration and Bystander Killing Effect

Scientists evaluated the efficacy of herpes simplex virus thymidine kinase suicide gene therapy for glioblastoma using neural stem/progenitor cells derived from human induced pluripotent stem cells (hiPSCs). Although transduction of hiPSCs is preferable for a safe and stable supply in the clinical setting, high-level and/or constitutive HSV-TK expression was highly cytotoxic to hiPSCs. [Hum Gene Ther] Abstract

Local Transplantation of Adipose-Derived Stem Cells Has a Significant Therapeutic Effect in a Mouse Model of Rheumatoid Arthritis

In an in vivo experiment, single-cell adipose-derived stem cells (ADSCs) and 3D-cultured ADSC spheroids were injected intra-articularly into the knees of rheumatoid arthritis model mice and histologically assessed. Marked improvement of synovial inflammation and articular cartilage regeneration was found in ADSC-treated mice. [Sci Rep] Full Article

The Effect of Matrigel as Scaffold Material for Neural Stem Cell Transplantation for Treating Spinal Cord Injury

Scientists investigated the effect of Matrigel combined with neural stem cells (NSCs) in vitro and in vivo. They compared the effect of different types of scaffold on the survival and differentiation of brain-derived NSCs in an in vitro culture. [Sci Rep] Full Article

Adipose Tissue-Derived Stem Cell Sheet Improves Glucose Metabolism in Obese Mice

Researchers investigated the effects of adipose tissue-derived stem cell (ADSC) sheet transplantation into the subcutaneous sites on the diabetic state of mice fed high fat and high sucrose diet. ADSCs were isolated and propagated from subcutaneous adipose tissues of non-diabetic intact mice. They used the thermo-responsive designated cell culture dishes to fabricate ADSC cell sheets. [Stem Cells Dev] Abstract

Utility of Microminipigs for Evaluating Liver-Mediated Gene Expression in the Presence of Neutralizing Antibody against Vector Capsid

Researchers evaluated the utility of microminipigs for liver-targeted gene therapy. These pigs were intravenously inoculated with an adeno-associated virus (AAV)8 vector encoding the luciferase gene, and gene expression was assessed by an in vivo imaging system. Robust transgene expression was observed almost exclusively in the liver, even though the pig showed a low-titer of neutralizing antibody against the AAV8 capsid. [Gene Ther] Full Article

Pharmacological Tools to Mobilize Mesenchymal Stromal Cells into the Blood Promote Bone Formation after Surgery

The authors showed that β3 adrenergic agonists (β3AR) in combination with a CXCR4 antagonist, AMD3100/Plerixafor, could mobilize mesenchymal stromal cells into the blood in mice and rats. Mechanistically, they showed that reversal of the CXCL12 gradient across the bone marrow endothelium and local generation of endocannabinoids may both play a role in this process. [NPJ Regen Med] Full Article

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Environmentally Responsive Hydrogels for Repair of Cardiovascular Tissue

The authors review the occurrence and current treatment of cardiovascular disease, as well as the repair of cardiovascular injury by several environmental responsive hydrogels systems currently used, mainly focusing on the delivery of growth factors or the application of cell therapy to revascularization. They also discuss the enormous potential and personal perspectives of environmentally responsive hydrogels in cardiovascular repair. [Heart Fail Rev] Abstract

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

Genprex and University of Pittsburgh Sign Exclusive License Agreement for Potentially Curative Gene Therapy Candidate for Diabetes

Genprex, Inc. announced that it signed an exclusive license agreement with the University of Pittsburgh for a diabetes gene therapy that may have the potential to cure Type 1 and Type 2 diabetes. [Genprex, Inc.] Press Release

LogicBio Therapeutics Provides Update on FDA Review of Investigational New Drug Application for LB-001 for Methylmalonic Acidemia

LogicBio Therapeutics, Inc. announced that the FDA has placed a clinical hold on the Investigational New Drug submission for LB-001 for the treatment of methylmalonic acidemia pending the resolution of certain clinical and nonclinical questions. [LogicBio Therapeutics, Inc.] Press Release

BrainStorm Cell Therapeutics and FDA Agree to Potential NurOwn^® Regulatory Pathway for Approval in ALS

BrainStorm Cell Therapeutics, Inc. announced that the company recently held a high level meeting with the FDA to discuss potential NurOwn^® regulatory pathways for approval in ALS. [BrainStorm Cell Therapeutics, Inc.] Press Release

FDA Accepts for Priority Review Bristol-Myers Squibb’s Biologics License Application (BLA) for Lisocabtagene Maraleucel (liso-cel) for Adult Patients with Relapsed or Refractory Large B-Cell Lymphoma

Bristol-Myers Squibb Company announced that the FDA has accepted for Priority Review its BLA for lisocabtagene maraleucel, the company’s autologous anti-CD19 chimeric antigen receptor (CAR) T-cell immunotherapy with a defined composition of purified CD8⁺ and CD4⁺ CAR T cells for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after at least two prior therapies. [Bristol-Myers Squibb Company] Press Release

‘The Disruption Is Enormous.’ Coronavirus Epidemic Snarls Science Worldwide

Normal daily life has come to a virtual standstill in large parts of China as a result of the epidemic of COVID-19—and so has science. Universities across the country remain closed; access to labs is restricted, projects have been mothballed, field work interrupted, and travel severely curtailed. [ScienceInsider] Editorial

White House Formally Invites Public Comment on Open-Access Policies

The White House issued a notice seeking comment on its effort to enhance public access to federally funded research. It’s an old idea creating new controversy. White House Office of Science and Technology Policy Director Kelvin Droegemeier is pushing back against publishers that in December said the administration was quietly pursuing an executive order to require immediate free distribution of taxpayer funded research. [ScienceInsider] Editorial

Has the UK Science Minister Been Demoted?

A government reshuffle in the United Kingdom has born another new science minister – a position that has seen a revolving door of appointees, firings and resignations in recent years. The latest appointment also comes with an apparent political demotion for the role, which expert observers say is in conflict with a government that appears to have put research and innovation at the heart of its agenda. [Nature News] Editorial

NEW American Association for Cancer Research (AACR) Annual Meeting 2020
April 24-29, 2020
San Diego, California, United States

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

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

Postdoctoral Researcher – Cell Therapies for Cancer (Technical University of Denmark)

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 Researcher – Drug Delivery (University of Minnesota Twin Cities)

Postdoctoral Researchers – Cell Biology (Medicinal Bioconvergence Research Center)

Postdoctoral Fellow – Cancer Research (BC Cancer)

Research Assistant – Cancer Research (BC Cancer)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer)

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