Developmental and epileptic encephalopathies: from genetic heterogeneity to phenotypic continuum

R Guerrini, V Conti, M Mantegazza… - Physiological …, 2023 - journals.physiology.org
Developmental and epileptic encephalopathies (DEEs) are a heterogeneous group of
disorders characterized by early-onset, often severe epileptic seizures and EEG …

[HTML][HTML] The role of insulin/IGF1 signalling in neurodevelopmental and neuropsychiatric disorders–evidence from human neuronal cell models

JM Réthelyi, K Vincze, D Schall, J Glennon… - … & Biobehavioral Reviews, 2023 - Elsevier
Insulin and insulin-like growth factor 1 (IGF1) signalling play a central role in the
development and maintenance of neurons in the brain, and in humans neurodevelopmental …

Opportunities and limitations for studying neuropsychiatric disorders using patient-derived induced pluripotent stem cells

Y Hong, Q Yang, H Song, G Ming - Molecular psychiatry, 2023 - nature.com
Neuropsychiatric disorders affect a large proportion of the global population and there is an
urgent need to understand the pathogenesis and to develop novel and improved treatments …

Toward the use of novel alternative methods in epilepsy modeling and drug discovery

C Miguel Sanz, M Martinez Navarro… - Frontiers in …, 2023 - frontiersin.org
Epilepsy is a chronic brain disease and, considering the amount of people affected of all
ages worldwide, one of the most common neurological disorders. Over 20 novel antiseizure …

Magnetic Nanoparticle‐Assisted Non‐Viral CRISPR‐Cas9 for Enhanced Genome Editing to Treat Rett Syndrome

HY Cho, M Yoo, T Pongkulapa, H Rabie… - Advanced …, 2024 - Wiley Online Library
The CRISPR‐Cas9 technology has the potential to revolutionize the treatment of various
diseases, including Rett syndrome, by enabling the correction of genes or mutations in …

Potential use of iPSCs for disease modeling, drug screening, and cell-based therapy for Alzheimer's disease

HE Marei, MUA Khan, A Hasan - Cellular & Molecular Biology Letters, 2023 - Springer
Alzheimer's disease (AD) is a chronic illness marked by increasing cognitive decline and
nervous system deterioration. At this time, there is no known medication that will stop the …

Development of therapeutic RNA manipulation for muscular dystrophy

Saifullah, N Motohashi, T Tsukahara… - Frontiers in Genome …, 2022 - frontiersin.org
Approval of therapeutic RNA molecules, including RNA vaccines, has paved the way for
next-generation treatment strategies for various diseases. Oligonucleotide-based …

Cell cluster sorting in automated differentiation of patient-specific induced pluripotent stem cells towards blood cells

Z Ma, MAS Toledo, P Wanek… - … in Bioengineering and …, 2022 - frontiersin.org
Induced pluripotent stem cells (iPS cells) represent a particularly versatile stem cell type for
a large array of applications in biology and medicine. Taking full advantage of iPS cell …

Human Brain Organoids in Migraine Research: Pathogenesis and Drug Development

P Gazerani - International Journal of Molecular Sciences, 2023 - mdpi.com
Human organoids are small, self-organized, three-dimensional (3D) tissue cultures that
have started to revolutionize medical science in terms of understanding disease, testing …

Bilirubin: A promising therapy for Parkinson's disease

S Jayanti, R Moretti, C Tiribelli, S Gazzin - International Journal of …, 2021 - mdpi.com
Following the increase in life expectancy, the prevalence of Parkinson's disease (PD) as the
most common movement disorder is expected to rise. Despite the incredibly huge efforts in …