1,865 research outputs found
Hematopoietic stem cell gene therapy to halt neurodegeneration
No full textMicroglia play fundamental roles in multiple pathological primary and secondary processes affecting the central nervous system that ultimately result in neurodegeneration and for this reason they are considered as a key therapeutic target in several neurodegenerative diseases. Microglia-targeted therapies are directed at either restoring or modulating microglia function, to redirect their functional features toward neuroprotection. Among these strategies, hematopoietic stem cell gene therapy have proven to be endowed with a unique potential for replacing diseased microglia with engineered, transplant progeny cells that can integrate and exert relevant beneficial effects in the central nervous system of patients affected by inherited and acquired neurodegenerative conditions
Gene therapy goes the distance in Wiskott-Aldrich syndrome
No full textLong-term follow-up data reinforce the curative potential of hematopoietic stem-cell gene therapy for this rare primary immunodeficiency disorder
Clinical Translation of TALENS: Treating SCID-X1 by Gene Editing in iPSCs
No full textMutations causing X-linked severe combined immunodeficiency (SCID-X1) reduce immune cell populations and function and may be amenable to targeted gene correction strategies. Now in Cell Stem Cell, Menon et al. (2015) correct SCID-X1-related blood differentiation defects by TALEN-mediated genome editing in patient-derived iPSCs, suggesting a possible strategy for autologous cell therapy of SCID-X1
Selective Biochemical Manipulation of Twin Neuronal Networks on Microelectrode Arrays
No full textComparability of responses to neuroactive compounds and spatially and temporally resolved delivery of soluble factors are two major key features for pharmacological assays. Here, we describe the fabrication and the use of a device for long-term growth of twin neuronal networks and for their controlled biochemicalstimulation. The device is formed by a PDMS microfl uidic chamber coupled to a fl at Microelectrode Array (MEA), which provides the electrophysiological readout of the pharmacological stimulation. A partial physical barrier divides the chamber in two sub-compartments, where two functionally independent but fl uidically connected neuronal networks can be grown. This platform improves biological comparability between cultures and allows to perform selective and temporally controlled stimulations to neurons, running parallel pharmacological tests on the same device
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