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Article
Cell & Tissue Engineering
Satoru Morimoto et al.
Summary: A phase 1/2a trial of ropinirole in ALS, based on iPSC-based drug discovery, showed that ropinirole could slow down disease progression and prolong disease-progression-free survival in ALS patients. The study also revealed the potential involvement of dopamine D2 receptor and SREBP2-cholesterol pathway in the therapeutic effects of ropinirole.
Article
Clinical Neurology
Ryoichi Nakamura et al.
Summary: In this study, three novel loci (FGF1, THSD7A, and LRP1) were identified to be associated with the survival of sporadic ALS patients. Decreased mRNA expression of FGF1 and THSD7A were also observed, which correlated with reduced viability of induced pluripotent stem cell-derived motor neurons from ALS patients.
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
(2023)
Review
Neurosciences
Elizabeth M. C. Fisher et al.
Summary: Amyotrophic lateral sclerosis (ALS) is a complex disorder with mostly unknown cause, but around 10% of cases are familial and caused by mutations in over 30 different genes. Mouse models exist for many genetic forms of ALS, but there is currently no model for the majority of ALS cases that are sporadic. The development of potential therapies has primarily relied on limited mouse models and has been tested on patients with different etiologies. The use of complex mouse models and patient stratification in clinical trials has proven successful in cancer research, and adopting a similar approach could lead to better understanding of ALS pathologies and faster translation of research findings into effective therapies.
MOLECULAR NEURODEGENERATION
(2023)
Review
Cell & Tissue Engineering
Elisa Giacomelli et al.
Summary: This article evaluates the status of using induced pluripotent stem cells (iPSCs) to model neurodegenerative diseases, focusing on amyotrophic lateral sclerosis (ALS) as an example. It discusses the methods and challenges associated with deriving and using disease-relevant neuronal and glial lineages.
Review
Cell & Tissue Engineering
Hideyuki Okano et al.
Summary: This review outlines the advances in iPSC modeling and therapeutic development for major neurodegenerative diseases, including amyotrophic lateral sclerosis, Parkinson's disease, and Alzheimer's disease.
Review
Pharmacology & Pharmacy
Stephen A. Johnson et al.
Summary: This article reviews existing ALS therapies and the current clinical drug development pipeline, highlighting the lack of effective treatments to stop or reverse disease progression. In addition to approved drugs, clinical trials are exploring small molecules and biologics such as stem cell transplantation.
Review
Medicine, General & Internal
Eva L. Feldman et al.
Summary: Amyotrophic lateral sclerosis, a fatal CNS neurodegenerative disease, lacks optimal management strategies. Improved understanding of clinical presentation, pathophysiology, and diagnostic criteria has not yet translated into direct patient benefit. However, the concept of amyotrophic lateral sclerosis as a complex syndrome is already influencing clinical practice.
Article
Medicine, General & Internal
Timothy M. Miller et al.
Summary: This study examined the use of tofersen in patients with SOD1 ALS and found that it can reduce protein concentrations in the cerebrospinal fluid and plasma, but does not improve clinical outcomes and is associated with adverse events. Further investigation is needed to evaluate the effects of early initiation of tofersen.
NEW ENGLAND JOURNAL OF MEDICINE
(2022)
