Diagnosis, prognosis, and treatment of leukodystrophies

MS van der Knaap, R Schiffmann, F Mochel… - The Lancet …, 2019 - thelancet.com
Leukodystrophies comprise a large group of rare genetic disorders primarily affecting CNS
white matter. Historically, the diagnostic process was slow and patient prognosis regarded …

[HTML][HTML] Krabbe disease: New hope for an old disease

AM Bradbury, ER Bongarzone, MS Sands - Neuroscience letters, 2021 - Elsevier
Krabbe disease (globoid cell leukodystrophy) is a lysosomal storage disease (LSD)
characterized by progressive and profound demyelination. Infantile, juvenile and adult-onset …

Defining recovery neurobiology of injured spinal cord by synthetic matrix-assisted hMSC implantation

AE Ropper, DK Thakor, IB Han, D Yu… - Proceedings of the …, 2017 - National Acad Sciences
Mesenchymal stromal stem cells (MSCs) isolated from adult tissues offer tangible potential
for regenerative medicine, given their feasibility for autologous transplantation. MSC …

Intraperitoneally infused human mesenchymal stem cells form aggregates with mouse immune cells and attach to peritoneal organs

N Bazhanov, JH Ylostalo, TJ Bartosh, A Tiblow… - Stem cell research & …, 2016 - Springer
Background Mesenchymal stem/progenitor cells (MSC) have shown beneficial effects in
many models of disease in part by modulating excessive inflammatory and immune …

Gene therapy for lysosomal storage disorders: a good start

A Biffi - Human molecular genetics, 2016 - academic.oup.com
Lysosomal storage disorders (LSDs) are a heterogeneous group of inherited diseases with
a collective frequency of∼ 1 in 7000 births, resulting from the deficiency in one or more …

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy

A Ricca, N Rufo, S Ungari, F Morena… - Human molecular …, 2015 - academic.oup.com
Globoid cell leukodystrophy (GLD) is a lysosomal storage disease caused by deficient
activity of β-galactocerebrosidase (GALC). The infantile forms manifest with rapid and …

Mesenchymal stem cell priming: fine-tuning adhesion and function

DPJ Kavanagh, J Robinson, N Kalia - Stem cell reviews and reports, 2014 - Springer
There is significant interest in the use of mesenchymal stem cells (MSCs) as a potential
therapeutic modality in disease and disorder, particularly those with an inflammation-based …

Clinical-grade human umbilical cord-derived mesenchymal stem cells reverse cognitive aging via improving synaptic plasticity and endogenous neurogenesis

N Cao, T Liao, J Liu, Z Fan, Q Zeng, J Zhou, H Pei… - Cell death & …, 2017 - nature.com
Cognitive aging is a leading public health concern with the increasing aging population, but
there is still lack of specific interventions directed against it. Recent studies have shown that …

Interleukin-1β pre-treated bone marrow stromal cells alleviate neuropathic pain through CCL7-mediated inhibition of microglial activation in the spinal cord

J Li, G Deng, H Wang, M Yang, R Yang, X Li… - Scientific Reports, 2017 - nature.com
Although neuropathic pain is one of the most intractable diseases, recent studies indicate
that systemic or local injection of bone marrow stromal cells (BMSCs) decreases pro …

Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy

A Lattanzi, C Salvagno, C Maderna… - Human Molecular …, 2014 - academic.oup.com
Globoid cell leukodystrophy (GLD) is an inherited lysosomal storage disease caused by β-
galactocerebrosidase (GALC) deficiency. Gene therapy (GT) should provide rapid, extensive …