Cell Therapy News Volume 21.30 | Aug 24 2020

May 12, 2022

2020-08-24 | CTN 21.30

Cell Therapy News by STEMCELL Technologies

Vol. 21.30 – 24 August, 2020

TOP STORY

A
Gene Therapy for Inherited Blindness Using dCas9-VPR–Mediated Transcriptional Activation

Using dual adeno-associated viral vectors expressing split catalytically inactive dCas9 fused to transcriptional activators (dCas9-VPR), scientists showed efficient transcriptional activation and long-term expression of cone photoreceptor-specific M-opsin in a rhodopsin-deficient mouse model for retinitis pigmentosa.
[Science Advances]

Full Article

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PUBLICATIONSRanked by the impact factor of the journal

Curative In Vivo
Hematopoietic Stem Cell Gene Therapy of Murine Thalassemia Using Large Regulatory Elements

Investigators capitalized on the 35 kb insert capacity of HDAd5/35++ vectors to demonstrate that transcriptional regulatory regions of the β-globin locus with a total length of 29 kb could efficiently be transferred into hematopoietic stem/progenitor cells (HSPCs). The in vivo HSPC transduction resulted in stable γ-globin levels in erythroid cells that conferred a complete cure of murine thalassemia intermedia.
[JCI Insight]

Full Article Graphical Abstract

Gene Therapy for Alzheimer’s Disease Targeting
CD33 Reduces Amyloid Beta Accumulation and Neuroinflammation

Researchers tested whether a gene therapy strategy to reduce Siglec-3 (CD33) on microglia in Alzheimer’s disease could decrease amyloid beta plaque load.
[Human Molecular Genetics]

Abstract

A
New Islet Transplantation Method Combining Mesenchymal Stem Cells with Recombinant Peptide Pieces, Microencapsulated Islets, and Mesh Bags

Investigators performed islet transplantation in diabetic mice by combining three components—microencapsulated islets, mesenchymal stem cell-CellSaic, and a mesh bag that encapsulates them and enables their removal.
[Biomedicines]

Full Article

Comparison
of Immunosuppressant Regimens in Salvage Cord Blood Transplantation for Graft Failure after Allogeneic Hematopoietic Stem Cell Transplantation

Using nationwide registration data, scientists compared the transplant outcomes of patients who developed graft failure and underwent salvage cord blood transplanatation using immunosuppressants, including calcineurin (CNI) alone; CNI plus methotrexate; and CNI plus mycophenolate mofetil.
[Bone Marrow Transplantation]

Abstract

Comparison
of Two Different Rabbit Antithymocyte Globulin (r-ATG) Preparations:Thymocyte r-ATG versus T Lymphoblast Cell Line r-ATG in Allogeneic Hematopoietic Stem Cell Transplantation for Acquired Severe Aplastic Anemia: Propensity Score-Matched Analysis

Researchers retrospectively analyzed the outcomes of 214 severe aplastic anemia patients who underwent allogeneic hematopoietic stem cell transplantation with r-ATG or ATG-Fresenius.
[Biology of Blood and Marrow Transplantation]

Abstract

Chimeric
Antigen Receptor T Cell Therapy Targeting ICAM-1 in Gastric Cancer

Scientists demonstrated therapeutic potential of CAR T cells targeting ICAM-1 in preclinical models of systemic and intraperitoneal metastases of gastric cancer.
[Molecular Therapy-Oncolytics]

Abstract Full Article
Graphical Abstract

Intracoronary
Injection of Hematopoietic Precursor Cells Regenerates the Borders, but Not the Core, of Old Myocardial Scars

The authors studied function recovery at the scar zone and its impact in global left ventricular function after intracoronary injection of hematopoietic precursor cells.
[ESC Heart Failure]

Full Article

Development
of Electrophysiological and Morphological Properties of Human Embryonic Stem Cell-Derived GABAergic Interneurons at Different Times after Transplantation into the Mouse Hippocampus

Scientists transplanted human embryonic stem cell-derived human GABAergic interneuron progenitors into the mouse hippocampus, and then characterized their electrophysiological properties and dendritic arborizations after transplantation by means of ex vivo whole-cell patch clamp recording, followed by biocytin staining, confocal imaging and neuron reconstruction software.
[PLoS One]

Full Article

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REVIEWS

Active RNA Interference in Mitochondria

The authors report that transfected small interfering RNAs are not only able to enter the matrix of mitochondria, but also function there to specifically silence targeted mitochondrial transcripts.
[Cell Research]

Abstract

Optimization
of Electroporation and other Non‐Viral Gene Delivery Strategies for T Cells

Researchers focus on the current state of the most promising non‐viral gene delivery techniques, including electroporation and transfection with cationic polymers or lipids.
[Biotechnology Progress]

Abstract

INDUSTRY AND POLICY NEWS

In
a Shocking Decision, FDA Rejects BioMarin’s Gene Therapy for Hemophilia

The FDA has rejected a one-time gene therapy for hemophilia A, delaying what would have been a major medical milestone in a decision that upended the expectations of doctors, patients, and Wall Street.
[STAT News]

Editorial

Sentien Biotechnologies Announces Open IND in Phase I/II Trial of SBI-101 for Patients with COVID-19

Sentien Biotechnologies, Inc. announced that the FDA has approved the company’s Investigational New Drug (IND) application for the evaluation of Sentien’s lead asset, SBI-101, for the treatment of severe COVID-19.
[Sentien Biotechnologies, Inc.]

Press Release

Editas
Medicine Receives Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Sickle Cell Disease

Editas Medicine, Inc. announced that the FDA has granted Rare Pediatric Disease designation for EDIT-301, an experimental, autologous cell medicine, being developed as a medicine for sickle cell disease.
[Editas Medicine, Inc.]

Press Release

Expression Therapeutics Announces Success in Developing a Stem Cell Lentiviral Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH)

Primary HLH is a family of devastating primary immune deficiencies with limited treatment options and no gene therapies under clinical testing. Expression Therapeutics has developed a promising and potentially curative gene therapy candidate for familial HLH type 3.
[Expression Therapeutics (PR Newswire Association LLC.)]

Press Release

FEATURED EVENT

A
Gene Therapy for Inherited Blindness Using dCas9-VPR–Mediated Transcriptional Activation

Curative In Vivo
Hematopoietic Stem Cell Gene Therapy of Murine Thalassemia Using Large Regulatory Elements

Gene Therapy for Alzheimer’s Disease Targeting
CD33 Reduces Amyloid Beta Accumulation and Neuroinflammation

A
New Islet Transplantation Method Combining Mesenchymal Stem Cells with Recombinant Peptide Pieces, Microencapsulated Islets, and Mesh Bags

Comparison
of Immunosuppressant Regimens in Salvage Cord Blood Transplantation for Graft Failure after Allogeneic Hematopoietic Stem Cell Transplantation

Comparison
of Two Different Rabbit Antithymocyte Globulin (r-ATG) Preparations:Thymocyte r-ATG versus T Lymphoblast Cell Line r-ATG in Allogeneic Hematopoietic Stem Cell Transplantation for Acquired Severe Aplastic Anemia: Propensity Score-Matched Analysis

Chimeric
Antigen Receptor T Cell Therapy Targeting ICAM-1 in Gastric Cancer

Intracoronary
Injection of Hematopoietic Precursor Cells Regenerates the Borders, but Not the Core, of Old Myocardial Scars

Development
of Electrophysiological and Morphological Properties of Human Embryonic Stem Cell-Derived GABAergic Interneurons at Different Times after Transplantation into the Mouse Hippocampus

Active RNA Interference in Mitochondria

Optimization
of Electroporation and other Non‐Viral Gene Delivery Strategies for T Cells

In
a Shocking Decision, FDA Rejects BioMarin’s Gene Therapy for Hemophilia

Sentien Biotechnologies Announces Open IND in Phase I/II Trial of SBI-101 for Patients with COVID-19

Editas
Medicine Receives Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Sickle Cell Disease

Expression Therapeutics Announces Success in Developing a Stem Cell Lentiviral Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH)

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