Gene therapy for ALS: A review
DA Amado, BL Davidson - Molecular Therapy, 2021 - cell.com
Amyotrophic lateral sclerosis (ALS) has historically posed unique challenges for gene-
therapy-based approaches, due to a paucity of therapeutic targets as well as the difficulty of …
therapy-based approaches, due to a paucity of therapeutic targets as well as the difficulty of …
Thirty years of Alzheimer's disease genetics: the implications of systematic meta-analyses
The genetic underpinnings of Alzheimer's disease (AD) remain largely elusive despite early
successes in identifying three genes that cause early-onset familial AD (those that encode …
successes in identifying three genes that cause early-onset familial AD (those that encode …
Cytoplasmic mislocalization of TDP-43 is toxic to neurons and enhanced by a mutation associated with familial amyotrophic lateral sclerosis
Mutations in the gene encoding TDP-43—the major protein component of neuronal
aggregates characteristic of amyotrophic lateral sclerosis (ALS) and frontotemporal lobar …
aggregates characteristic of amyotrophic lateral sclerosis (ALS) and frontotemporal lobar …
The heritability and genetics of frontotemporal lobar degeneration
Background: Frontotemporal lobar degeneration (FTLD) is a genetically and pathologically
heterogeneous neurodegenerative disorder. Methods: We collected blood samples from a …
heterogeneous neurodegenerative disorder. Methods: We collected blood samples from a …
FTD and ALS: a tale of two diseases
R Ferrari, D Kapogiannis, ED Huey… - Current Alzheimer …, 2011 - ingentaconnect.com
The first reports of disorders that in terms of cognitive and behavioral symptoms resemble
frontotemporal dementia (FTD) and in terms of motor symptoms resemble amyotrophic …
frontotemporal dementia (FTD) and in terms of motor symptoms resemble amyotrophic …
The two faces of protein misfolding: gain‐and loss‐of‐function in neurodegenerative diseases
KF Winklhofer, J Tatzelt, C Haass - The EMBO journal, 2008 - embopress.org
The etiologies of neurodegenerative diseases may be diverse; however, a common
pathological denominator is the formation of aberrant protein conformers and the occurrence …
pathological denominator is the formation of aberrant protein conformers and the occurrence …
Inflammation in Alzheimer's disease: amyloid-β oligomers trigger innate immunity defence via pattern recognition receptors
A Salminen, J Ojala, A Kauppinen, K Kaarniranta… - Progress in …, 2009 - Elsevier
The inflammatory process has a fundamental role in the pathogenesis of Alzheimer's
disease (AD). Recent studies indicate that inflammation is not merely a bystander in …
disease (AD). Recent studies indicate that inflammation is not merely a bystander in …
TMEM106B regulates progranulin levels and the penetrance of FTLD in GRN mutation carriers
N Finch, MM Carrasquillo, M Baker, NJ Rutherford… - Neurology, 2011 - AAN Enterprises
Objectives: To determine whether TMEM106B single nucleotide polymorphisms (SNPs) are
associated with frontotemporal lobar degeneration (FTLD) in patients with and without …
associated with frontotemporal lobar degeneration (FTLD) in patients with and without …
Profound downregulation of the RNA editing enzyme ADAR2 in ALS spinal motor neurons
T Hideyama, T Yamashita, H Aizawa, S Tsuji… - Neurobiology of …, 2012 - Elsevier
Amyotrophic lateral sclerosis (ALS) is the most common adult-onset fatal motor neuron
disease. In spinal motor neurons of patients with sporadic ALS, normal RNA editing of …
disease. In spinal motor neurons of patients with sporadic ALS, normal RNA editing of …
Functional mapping of the interaction between TDP-43 and hnRNP A2 in vivo
A D'Ambrogio, E Buratti, C Stuani… - Nucleic acids …, 2009 - academic.oup.com
Nuclear factor TDP-43 has been reported to play multiple roles in transcription, pre-mRNA
splicing, mRNA stability and mRNA transport. From a structural point of view, TDP-43 is a …
splicing, mRNA stability and mRNA transport. From a structural point of view, TDP-43 is a …