Denali Therapeutics
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Advancing Gene-Targeted Therapies for Central Nervous System Disorders
Author: National Academies of Sciences, Engineering, and Medicine
language: en
Publisher: National Academies Press
Release Date: 2019-12-01
On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.
Receptor Tyrosine Kinases in Neurodegenerative and Psychiatric Disorders
Overexpression of receptor tyrosine kinases (RTKs) has been implicated in cancer. Increasing evidence suggests that dysregulation of RTKs is reported in many neurodegenerative and psychiatric disorders. Receptor Tyrosine Kinases in Neurodegenerative and Psychiatric Disorders provides a fundamental, pragmatic map of RTKs structure, activation, functions, mechanism of action, gene regulation, and signaling pathways in developing central nervous system (CNS), adult normal CNS, neurodegenerative and psychiatric disorders. Additionally, the structure, function, activation, and therapeutic potential of many growth factors have been covered. Here, international experts in the field offer a comprehensive discussion of results of pre-clinical and clinical studies of repositioning of anti-cancer receptor tyrosine kinase inhibitors (RTKIs) and other promising protein kinase inhibitors in various neurodegenerative disorders including, Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease, and multiple sclerosis, and psychiatric disorders including, anxiety, depression, alcohol use disorder, and schizophrenia. In addition, the book discusses the obstacles and opportunities for the potential repositioning of kinase inhibitors in neurodegenerative and psychiatric disorders. - Provides a thorough overview of RTKs biology and their role in health and disease progression and modulation - Highlights RTK families and the numerous receptors within each subfamily - Examines RTKIs and other protein kinase inhibitors in pre-clinical and clinical trials in both neurodegenerative and psychiatric disorders - Gives future directions of possible safe effective targeted RTKIs and other protein kinase inhibitors that may be repositioned in various neurodegenerative and psychiatric disorders - Includes chapter contributions from renowned experts in biology, pharmacology, neurology, psychiatry, and oncology
Microglia
Author: Marie-Ève Tremblay
language: en
Publisher: Springer Nature
Release Date: 2024-08-29
The past decade has witnessed a revolution in our understanding of microglia, especially since their roles in the healthy CNS have started to unravel. These cells were shown to actively maintain health, in concert with neurons and other types of CNS cells, providing further insight into their crucial involvement with diseases. Edited by Drs. Marie-Ève Tremblay and Alexei Verkhratsky, Microglia: Physiology, Pathophysiology and Therapeutic Potential shares with the scientific and medical community the latest discoveries in the microglial research field, with a truly comprehensive collection of chapters written by the top specialists across five continents. The book begins by explaining briefly what they are, from both historical and evolutionary points of view, and how they can be studied. The first section explains their physiological roles in the maturation, function, and plasticity of the CNS. The second section focuses on their general involvement in neuropathophysiology, and the third section on their critical implication in specific CNS diseases, including neurotrauma, neuropathic pain, ischemia and stroke, infectious diseases, autoimmune diseases, neurodevelopmental and neuropsychiatric disorders, substance use and addiction, sleep disorders, ageing, and neurodegenerative diseases. The fourth section presents their clinical potential as a targeted therapeutic tool for these CNS diseases.