Category REGENERATIVE MEDICINE

Phase I/II Study of Safety and Preliminary Efficacy of Intravenous Allogeneic Mesenchymal Stem Cells in Chronic Stroke

Background and Purpose— Stroke is a leading cause of long-term disability. Limited treatment options exist for patients with chronic stroke and substantial functional deficits. The current study examined safety and preliminary efficacy estimates of intravenous allogeneic mesenchymal stem cells in this population. Methods— Entry criteria included ischemic stroke >6 months prior and substantial impairment (National

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The use of validated and nonvalidated surrogate endpoints in two European Medicines Agency expedited approval pathways: A cross-sectional study of products authorised 2011–2018

The majority of marketing authorizations granted through two expedited assessment pathways in Europe are based on non-validated surrogate endpoints rather than clinical outcomes, according to a study published September 10 in the open-access journal PLOS Medicine by Patricia McGettigan of Queen Mary University of London, and colleagues. The approval of new pharmaceutical products addressing an unmet need

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Study shows Mesenchymal Stromal Cells importance of tissue origin in predicting function

A new study released today in STEM CELLS demonstrates that mesenchymal stromal cells (MSCs) expanded from adipose tissue (ASC) outperform those expanded from bone marrow (BM-MSCs). A new study released today in STEM CELLS demonstrates that mesenchymal stromal cells (MSCs) expanded from adipose tissue (ASC) outperform those expanded from bone marrow (BM-MSCs). Importantly, while several

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Exosomes from Adipose-Derived Stem Cells (ADSCs) Overexpressing miR-21 Promote Vascularization of Endothelial Cells

miR-21 has been indicated to be an important regulator of angiogenesis, involving in regulating the proliferation and migration of vascular cells, like endothelial cells. Direct evidences have shown that downregulation of miR-21 expression signifcantly reduces the proliferation and migration of HUVECs, and conversely, miR-21 overexpression signifcantly enhances HUVECs proliferation and migration, indicating the importance of

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Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies

Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their

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Success of gene therapy for a form of inherited blindness depends on timing

An FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved patients’ sight. But new research underscores the importance of further investigation to halt the progression of the disorder. Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was

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