Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis

SE Mole, G Anderson, HA Band, SF Berkovic… - The Lancet …, 2019 - thelancet.com
Treatment of the neuronal ceroid lipofuscinoses, also known as Batten disease, is at the start
of a new era because of diagnostic and therapeutic advances relevant to this group of …

Ex vivo gene transfer and correction for cell-based therapies

L Naldini - Nature Reviews Genetics, 2011 - nature.com
Cell-based therapies are fast-growing forms of personalized medicine that make use of the
steady advances in stem cell manipulation and gene transfer technologies. In this Review, I …

Purification and characterization of human neural stem and progenitor cells

DD Liu, JQ He, R Sinha, AE Eastman, AM Toland… - Cell, 2023 - cell.com
The human brain undergoes rapid development at mid-gestation from a pool of neural stem
and progenitor cells (NSPCs) that give rise to the neurons, oligodendrocytes, and astrocytes …

Therapeutic landscape for Batten disease: current treatments and future prospects

TB Johnson, JT Cain, KA White… - Nature Reviews …, 2019 - nature.com
Batten disease (also known as neuronal ceroid lipofuscinoses) constitutes a family of
devastating lysosomal storage disorders that collectively represent the most common …

Glia disease and repair—remyelination

RJM Franklin, SA Goldman - Cold Spring Harbor …, 2015 - cshperspectives.cshlp.org
The inability of the mammalian central nervous system (CNS) to undergo spontaneous
regeneration has long been regarded as a central tenet of neurobiology. However, although …

Macrophages as mediators of tumor immunosurveillance

S Jaiswal, MP Chao, R Majeti, IL Weissman - Trends in immunology, 2010 - cell.com
Tumor immunosurveillance is a well-established mechanism for regulation of tumor growth.
In this regard, most studies have focused on the role of T-and NK-cells as the critical immune …

Human neural stem cells alleviate Alzheimer-like pathology in a mouse model

IS Lee, K Jung, IS Kim, H Lee, M Kim, S Yun… - Molecular …, 2015 - Springer
Background Alzheimer's disease (AD) is an inexorable neurodegenerative disease that
commonly occurs in the elderly. The cognitive impairment caused by AD is associated with …

The neuronal ceroid-lipofuscinoses (Batten disease)

SE Mole, A Schulz - Rosenberg's Molecular and Genetic Basis of …, 2025 - Elsevier
The neuronal ceroid-lipofuscinoses (NCLs), collectively also called Batten disease,
constitute one of the most common groups of inherited neurodegenerative disorders in …

Current and emerging treatment strategies for neuronal ceroid lipofuscinoses

A Kohlschütter, A Schulz, U Bartsch, S Storch - CNS drugs, 2019 - Springer
The neuronal ceroid lipofuscinoses comprise a group of neurodegenerative lysosomal
storage disorders caused by mutations in at least 13 different genes and primarily affect the …

Moving stem cells to the clinic: potential and limitations for brain repair

JA Steinbeck, L Studer - Neuron, 2015 - cell.com
Stem cell-based therapies hold considerable promise for many currently devastating
neurological disorders. Substantial progress has been made in the derivation of disease …