Sarepta Therapeutics, Inc. announced positive results from the ongoing study of SRP-9003 the company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E.
[Sarepta Therapeutics, Inc.]
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Category Archives: Cell Therapy
Cell Therapy NewsCell Therapy News is an online publication and email newsletter dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.
Published weekly since 2002, Cell Therapy News is our longest running online publication. Our editorial team curates the top publications, reviews, and news on in vivo gene therapy, cell therapy, and immunotherapy research. With over 15,000 subscribers and followers across our website, email newsletter, and Twitter feed, we are proud to facilitate the communication of cutting-edge cell therapy innovations.
Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months After Administration
AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease
AVROBIO, Inc. announced that the European Commission has granted orphan drug designation for AVR-RD-02, the company’s investigational gene therapy for the treatment of Gaucher disease.
[AVROBIO, Inc.]
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A Defined N6-Methyladenosine (m6A) Profile Conferred by METTL3 Regulates Muscle Stem Cell/Myoblast State Transitions
RNAi studies showed that reducing levels of METTL3, the active m6A methyltransferase, reduced global m6A levels and forced C2C12 myoblasts to prematurely differentiate.
[Cell Death Discovery]
Gheller, B. J., Blum, J. E., Fong, E. H. H., Malysheva, O. V., Cosgrove, B. D., & Thalacker-Mercer, A. E. (2020). A defined N6-methyladenosine (m 6 A) profile conferred by METTL3 regulates muscle stem cell/myoblast state transitions. Cell Death Discovery, 6(1), 1–14. https://doi.org/10.1038/s41420-020-00328-5 Cite
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Vor Biopharma and Arbor Biotechnologies to Collaborate on Engineered Hematopoietic Stem-Cell Therapies
Vor Biopharma and Arbor Biotechnologies announced an agreement to use Arbor’s gene editing technologies to engineer hematopoietic stem cells, towards the goal of developing therapies for the treatment of blood cancers, such as acute myeloid leukemia.
[Vor Biopharma]
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AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease
AVROBIO, Inc. announced that the European Commission has granted orphan drug designation for AVR-RD-02, the company’s investigational gene therapy for the treatment of Gaucher disease.
[AVROBIO, Inc.]
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Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months After Administration
Sarepta Therapeutics, Inc. announced positive results from the ongoing study of SRP-9003 the company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E.
[Sarepta Therapeutics, Inc.]
0
Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months after Administration
Sarepta Therapeutics, Inc. announced positive results from the ongoing study of SRP-9003, the company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E.
[Sarepta Therapeutics, Inc. (GlobeNewswire, Inc.)]
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A Modified Human Myogenin Promoter That Is Highly Active in Alveolar Rhabdomyosarcoma
Investigators designed a suicide gene therapy by generating an ARMS-targeted promoter to drive the herpes simplex virus thymidine kinase suicide gene.
[Cancer Gene Therapy]
A modified human Myogenin promoter that is highly active in alveolar rhabdomyosarcoma | Cancer Gene Therapy. (n.d.). Retrieved September 25, 2020, from https://www.nature.com/articles/s41417-020-00225-0 Cite
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Reduced Dose of Posttransplant Cyclophosphamide in HLA-Haploidentical Peripheral Blood Stem Cell Transplantation
Scientists conducted two consecutive prospective multicenter phase II studies to evaluate the safety and efficacy of 80 mg/kg of PTCy in 137 patients who underwent HLA-haploidentical peripheral blood stem cell transplantation following reduced-intensity conditioning
[Bone Marrow Transplantation]
Sugita, J., Kamimura, T., Ishikawa, T., Ota, S., Eto, T., Kuroha, T., Miyazaki, Y., Kumagai, H., Matsuo, K., Akashi, K., Taniguchi, S., Harada, M., & Teshima, T. (2020). Reduced dose of posttransplant cyclophosphamide in HLA-haploidentical peripheral blood stem cell transplantation. Bone Marrow Transplantation, 1–9. https://doi.org/10.1038/s41409-020-01065-0 Cite
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BATF3 Programs CD8+ T Cell Memory
Investigators showed that BATF3 was expressed transiently within the first days after T cell priming and had long-lasting T cell–intrinsic effects.
[Nature Immunology]
Ataide, M. A., Komander, K., Knöpper, K., Peters, A. E., Wu, H., Eickhoff, S., Gogishvili, T., Weber, J., Grafen, A., Kallies, A., Garbi, N., Einsele, H., Hudecek, M., Gasteiger, G., Hölzel, M., Vaeth, M., & Kastenmüller, W. (2020). BATF3 programs CD8 + T cell memory. Nature Immunology, 1–11. https://doi.org/10.1038/s41590-020-0786-2 Cite
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Calreticulin del52 and ins5 Knock-In Mice Recapitulate Different Myeloproliferative Phenotypes Observed in Patients with MPN
Researchers generated heterozygous and homozygous conditional inducible knock-in mice expressing a chimeric murine CALR del52 or ins5 with the human mutated C-terminal tail to investigate their pathogenic effects on hematopoiesis.
[Nature Communications]
Benlabiod, C., Cacemiro, M. da C., Nédélec, A., Edmond, V., Muller, D., Rameau, P., Touchard, L., Gonin, P., Constantinescu, S. N., Raslova, H., Villeval, J.-L., Vainchenker, W., Plo, I., & Marty, C. (2020). Calreticulin del52 and ins5 knock-in mice recapitulate different myeloproliferative phenotypes observed in patients with MPN. Nature Communications, 11(1), 4886. https://doi.org/10.1038/s41467-020-18691-3 Cite
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