Lentiviral vectors in gene therapy: their current status and future potential
The concept of gene therapy originated in the mid twentieth century and was perceived as a
revolutionary technology with the promise to cure almost any disease of which the molecular …
revolutionary technology with the promise to cure almost any disease of which the molecular …
Gene therapy tools for brain diseases
Neurological disorders affecting the central nervous system (CNS) are still incompletely
understood. Many of these disorders lack a cure and are seeking more specific and effective …
understood. Many of these disorders lack a cure and are seeking more specific and effective …
Direct conversion of human fibroblasts to dopaminergic neurons
Recent reports demonstrate that somatic mouse cells can be directly converted to other
mature cell types by using combined expression of defined factors. Here we show that the …
mature cell types by using combined expression of defined factors. Here we show that the …
A versatile tool for conditional gene expression and knockdown
Drug-inducible systems allowing the control of gene expression in mammalian cells are
invaluable tools for genetic research, and could also fulfill essential roles in gene-and cell …
invaluable tools for genetic research, and could also fulfill essential roles in gene-and cell …
[HTML][HTML] Age-related pathological impairments in directly reprogrammed dopaminergic neurons derived from patients with idiopathic Parkinson's disease
We have developed an efficient approach to generate functional induced dopaminergic (DA)
neurons from adult human dermal fibroblasts. When performing DA neuronal conversion of …
neurons from adult human dermal fibroblasts. When performing DA neuronal conversion of …
[HTML][HTML] Activation of neuronal genes via LINE-1 elements upon global DNA demethylation in human neural progenitors
ME Jönsson, P Ludvik Brattås, C Gustafsson… - Nature …, 2019 - nature.com
DNA methylation contributes to the maintenance of genomic integrity in somatic cells, in part
through the silencing of transposable elements. In this study, we use CRISPR-Cas9 …
through the silencing of transposable elements. In this study, we use CRISPR-Cas9 …
MicroRNA-124 is a subventricular zone neuronal fate determinant
M Åkerblom, R Sachdeva, I Barde, S Verp… - Journal of …, 2012 - Soc Neuroscience
New neurons are continuously generated from neural stem cells with astrocyte properties,
which reside in close proximity to the ventricle in the postnatal and adult brain. In this study …
which reside in close proximity to the ventricle in the postnatal and adult brain. In this study …
Immune-evasive gene switch enables regulated delivery of chondroitinase after spinal cord injury
Chondroitinase ABC is a promising preclinical therapy that promotes functional
neuroplasticity after CNS injury by degrading extracellular matrix inhibitors. Efficient delivery …
neuroplasticity after CNS injury by degrading extracellular matrix inhibitors. Efficient delivery …
Huntingtin aggregation impairs autophagy, leading to argonaute-2 accumulation and global MicroRNA dysregulation
Many neurodegenerative diseases are characterized by the presence of intracellular protein
aggregates, resulting in alterations in autophagy. However, the consequences of impaired …
aggregates, resulting in alterations in autophagy. However, the consequences of impaired …
GDNF-based therapies, GDNF-producing interneurons, and trophic support of the dopaminergic nigrostriatal pathway. Implications for Parkinson's disease
The glial cell line-derived neurotrophic factor (GDNF) is a well-established trophic agent for
dopaminergic (DA) neurons in vitro and in vivo. GDNF is necessary for maintenance of …
dopaminergic (DA) neurons in vitro and in vivo. GDNF is necessary for maintenance of …