Welcome to our book review site go-pdf.online!
You may have to Search all our reviewed books and magazines, click the sign up button below to create a free account.
Spinal Muscular Atrophy
Spinal Muscular Atrophy: Disease Mechanisms and Therapy provides the latest information on a condition that is characterized by motoneuron loss and muscle atrophy, and is the leading genetic cause of infant mortality. Since the identification of the gene responsible for SMA in 1995, there have been important advances in the basic understanding of disease mechanisms, and in therapeutic development. This book provides a comprehensive accounting of recent advances in basic and clinical research that covers SMA clinical features and standards of care, multifaceted aspects of SMN protein functions and SMA disease pathology, various animal models, and biomarkers, as well as current therapeutic dev...
Produzione di cellule FRT CFTR-W1282X per lo studio avanzato di molecole ad azione readthrough (TRIDs)
Negli ultimi 10 anni si sono registrati progressi molto significativi nello sviluppo di piccole molecole per il trattamento della fibrosi cistica (CF), molecole che sono correttori o potenziatori del canale CFTR. Tuttavia, queste molecole incrementano rispettivamente la quantità del canale nella membrana plasmatica e la probabilità di apertura del canale. Correttori e potenziatori per agire richiedono quindi la presenza della proteina Tuttavia, circa il 10% della popolazione affetta da CF mostra mutazioni non-senso che causano la formazione di un codone di terminazione prematura (PTC) associato a una proteina CFTR incompleta e non funzionale. In questo contesto genetico i correttori e i po...
Proceedings of FORM 2022
This book gathers the latest advances, innovations, and applications in the field of environmental and construction engineering, as presented by international researchers at the XXV International Scientific Conference "Construction: The Formation of Living Environment", held in Moscow, Russia on April 20-22, 2022. It covers highly diverse topics, including sustainable innovative development of the construction industry, building materials, reliability of buildings and constructions and safety in construction, modelling and mechanics of building structures, engineering and smart systems in construction, climate change and urban environment. The contributions, which were selected by means of a rigorous international peer-review process, highlight numerous exciting ideas that will spur novel research directions and foster multidisciplinary collaborations.
Journal of the Institution of Petroleum Technologists
Vols. 7- include "Abstracts" which, beginning with v. 9 form a separately paged section, and from v. 17 on, have separate title pages.
Phenotypic Drug Discovery
Phenotypic drug discovery has been highlighted in the past decade as an important strategy in the discovery of novel medical entities. This book aims to equip researchers with a thought-provoking guide to the application and development of contemporary phenotypic drug discovery for clinical success.
