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In den letzten zehn Jahren hat sich die Gentherapie als eine bahnbrechende neue Behandlungsoption entwickelt, insbesondere durch die Zulassung von Produkten für die Hämophilie A und B. Diese Therapieform verspricht eine weitgehende Unabhängigkeit von regelmäßiger Substitutionstherapie bei niedrigen Blutungsraten, dank der Verwendung von adeno-assoziierten Viren (AAV) als Vektoren. Das vorliegende Buch vermittelt die aktuellen Kenntnisse zum Potenzial der neuen Gentherapeutika in der Behandlung von Hämophilie A und B. Es deckt dabei ein breites Spektrum ab: von den Grundlagen der Hämophilie über die verschiedenen gentherapeutischen Ansätze (AAV, Lentivirus, Gene Editing) bis hin zu spezifischen Aspekten wie der Auswahl geeigneter Patienten für die Gentherapie, den Auswirkungen auf Gerinnungsfaktorwerte, leberbezogenen Fragestellungen und der Immunantwort auf AAV-vermittelte Gentherapie. Es erläutert außerdem die Notwendigkeit einer interdisziplinären Zusammenarbeit und diskutiert das innovative “Hub and Spoke”-Modell. Dieses Buch soll als Brücke dienen, die die Lücke zwischen dem aktuellen Wissenstand und zukünftigen Entwicklungen schließt, und es zielt darauf ab, eine umfassende Informationsquelle für Fachleute in der klinischen Praxis und Forschung zu sein.

Treatment Landscape of Targeted Therapies in Oncology: Challenges and Opportunities provides up-to-date knowledge on antitumor-targeted therapies and immunotherapy. The book's chapters are written by researchers dynamically working/focusing on cancer treatment. The content is designed to help those who are new to the field (beginners) and various specialized scientists and researchers involved in cancer research. For decades, the hallmark of cancer treatment has been conventional chemotherapy. But with the rapid increase in our understanding of the immune system, more and more small molecules, peptides, recombinant antibodies, vaccines and cellular therapeutic modalities are being applied to manipulate the immune response for cancer treatment. - Covers basic concepts and updated knowledge of the latest anticancer drugs - Brings comprehensive understanding of the treatment of cancer in context of immunotherapy and targeted therapy - Introduces the latest drugs and advancements in the treatment of cancer - Includes detailed diagrams to give insight to the mechanisms of action

Post-transcriptional Gene Regulation in Human Disease, a new volume in the Translational Epigenetics book series, offers a thorough overview and discussion of post-transcriptional genetic control mechanisms and their roles across various pathologies and human developmental outcomes, along with regulatory mechanisms targeted for therapeutic approaches. The book is broadly divided in two parts: early chapters describe the basics of post-transcriptional gene regulation, associated epigenetic mechanisms, the role of RNA binding proteins, the evolution of post-transcriptional gene regulation, and methods to study these mechanisms. The second half of the book includes deeper discussion of post-transcriptional gene regulation across specific diseases and therapeutics targets. Various post-transcriptional events, including alternative splicing and polyadenylation, mRNA stability, and miRNAs and their involvement in the disease progression, are examined in detail. - Includes full-color imagery illustrating key concepts and post-transcriptional disease processes, as well as descriptions of methods for studying post-transcriptional gene regulation - Presents fundamental knowledge, molecular and biochemical mechanisms, and recent findings in concise and easily understandable formats - Features a summary and conclusion at the end of each chapter