Dr. Miccio holds patents related to viral gene delivery vectors. The Topic Editors acknowledge the use of image material from kindpng.com and from Crystal and Annie Spratt on unsplash.com.

This new volume of Methods in Enzymology continues the legacy of this premier serial with quality chapters authored by leaders in the field. This volume covers recent research and methods development for changing the DNA sequence within the genomes of cells and organisms. Focusing on enzymes that generate double-strand breaks in DNA, the chapters describe use of molecular tools to introduce or delete genetic information at specific sites in the genomes of animal, plant and bacterial cells. - Continues the legacy of this premier serial with quality chapters authored by leaders in the field - Covers research methods in biomineralization science - Contains sections on such topics as genome editing, genome engineering, CRISPR, Cas9, TALEN and zinc finger nuclease

In this issue of Hematology/Oncology Clinics, guest editors Drs. Sung-Yun Pai and Nirali N. Shah bring their considerable expertise to the topic of Gene-Based Therapies for Pediatric Blood Diseases. Top experts in the field cover key topics such as CAR T-cell therapy: current status; engineered T cells; NK-cell therapy; hemoglobinopathies: beta-thalassemia, sickle cell disease; hemophilia A/B; primary immunodeficiencies; and more. - Contains 14 relevant, practice-oriented topics including the evolution of gene therapy; viral vectors in hematopoietic stem cell gene therapy; gene editing in hematopoietic stem cells; nonintegrating vectors and engineered capsids; regulatory aspects of gene therapy; and more. - Provides in-depth clinical reviews on gene-based therapies for pediatric blood diseases, offering actionable insights for clinical practice. - Presents the latest information on this timely, focused topic under the leadership of experienced editors in the field. Authors synthesize and distill the latest research and practice guidelines to create clinically significant, topic-based reviews.

Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy—the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome. - Provides a comprehensive view of gene therapy and genome editing technologies, including epigenetic editing - Describes the state-of-the-art and future directions for the treatment of genetic diseases, also considering economical aspects - Presents chapters that each give a thorough review of a specific disease, target organ or visionary approach, including ethical considerations

The book “STEM CELL THERAPY FOR ORGAN FAILURES” edited by Dr. S. Indumathi demonstrates the In Vitro and In vivo therapeutic strategies and applications of pre- and post-natal stem cells for treating the failures of various organ systems of our body in a wide perspective. It explores the past, present and the futuristic approach of the exciting field of stem cells and its intriguing properties involved in tissue repair and regeneration. The prime focus of this volume is to unravel the basic, advanced, therapeutic and translational approaches put-forth so far in the field of stem cells and regenerative medicine at research, pre-clinical and clinical levels. Stem cells has ushered in widespread interest and exciting possibilities for cell based therapies, albeit failures do prevail and small uncontrolled phase I/II studies are only signals generating, rather than definite proof of concept thereby limiting its applicability in curative therapeutics. Despite certain initiatives and meticulous untiring efforts, bringing this basic bench side research into advanced transitional bedside remained a challenge. Thus, this book embarked upon the expanding researches in these areas that seem decisive in improvising regenerative medical therapeutics, thereby leading to further path-breaking studies that cure all health challenges facing mankind. Overall, this book reveals the imperativeness of various stem cell sources and its utility in curative therapeutics.

CRISPR-Cas-Based Genome Editing for Treating Human Diseases-Part A, Volume 208 represents CRISPR-Cas systems for genome editing. Currently, CRISPR-Cas systems are proven a key technology for targeted genome editing, which is acting as a simple, rapid, and cost-effective solution. CRISPR-Cas9 system is being used in microbial genome editing, mammalian genome editing, disease models, and more. It has shown potential in human disease treatment. However, it is not easy to find CRISPR-Cas systems genome editing in a single source.This volume offers CRISPR-Cas systems for human diseases, bacterial disease, gut microbiome editing, viral disease, protozoan disease, fungal disease, stem cell therapy, CRISPR in epigenetics, CRISPR in cancer, autoimmune and blood disorders. In addition, it highlights a number of aspects of the CRISPR-Cas systems that can help the basic understanding of students, researchers, clinicians, entrepreneurs, and stakeholders to perform their research with great interest. - Updares on CRISPR-Cas systems for human diseases, bacterial disease, gut microbiome editing, viral disease, protozoan disease and fungal disease - Discusses stem cell therapy, CRISPR in epigenetics, CRISPR in cancer, autoimmune and blood disorders - Includes commentary on CRISPR in autoimmune and blood disorders

This Research Topic honors the memory of Prof. Antonius “Ton” G. Rolink (April 19, 1953–August 06, 2017), our colleague, mentor and friend in immunology. It is now over a year since Ton left us. This article collection, authored by many of Ton’s friends and colleagues, reflects the huge contribution to cellular and molecular immunology that work emanating directly from Ton’s own hands and laboratory have made to the understanding of lymphocyte development. Ton’s hard work, expertise, generosity, passion for science and infectious humor were legendary and for all of those lucky enough to have been his colleague, he ensured that science was fun. We take this opportunity of thanking all contributors for submitting their manuscripts; we are sure that Ton would have enjoyed reading and making his own insightful comments on them. In the form of original research and review articles, these papers cover many of Ton’s scientific interests in different aspects of lymphocyte development in mouse and man. In the first section, Development of hematopoietic cells and lymphocytes, Klein et al. describe the accumulation of multipotent hematopoietic progenitors in peripheral lymphoid organs of IL-7xFlt3L double transgenic mice and Pang et al. the role of the transcription factor PU.1 on the development of Common Lymphoid Progenitors. In Early B cell development, Winkler and Mårtensson review the role of the Pre-B cell receptor in B cell development and papers by Hobeika et al. and Brennecke et al. describe models of inducible B cell development. For B cell selection, survival and tolerance, Smulski and Eibel review the role of BAFF and Kowalczyk-Quintans et al. analyse the role of membrane-bound BAFF. The impact of BIM on B cell homeostasis is discussed by Liu et al. The role of the MEK-ERK pathway in B cell tolerance is discussed by Greaves et al. and the transcriptional regulation of germinal center development is reviewed by Song and Matthias. For Hematological diseases, Ghia reviews how studies of B cell development help the understanding of Leukemia development, Kim and Schaniel review how iPS technology helps the understanding of hematological diseases and Hellmann et al. describe development of new therapeutic antibody drug conjugates. Finally, in T cell development, homeostasis and graft vs. host disease, Heiler et al. describe the therapeutic effects of IL-2/anti-IL-2 immune complexes in GvHD, Calvo-Asensio et al. describe the DNA damage response of thymocyte progenitors and Mori and Pieters review the role of Coronin 1 in T cell survival.

Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy, presents the collation of chapters written by eminent scientists worldwide. CRISPR-Cas9 is a key technology for targeted genome editing and regulation in a number of organisms including mammalian cells. It is a rapid, simple, and cost-effective solution. CRISPR-Cas system has recently gained much scientific and public attention. This volume covers CRISPR-Cas9 based mammalian genome editing, creating disease models, cancer therapy, neurological, heredity, blood disorders, defective gene correction, stem cells therapy, epigenetic modifications, patents, ethics, biosafety and regulatory issues challenges and opportunities. This book is a key source of information on mammalian genome editing available in a single volume. This book will be useful for beginners in mammalian genome editing and also students, researchers, scientists, policymakers, clinicians and stakeholders interested in genome editing in several areas. - Offers basic understanding and a clear picture of mammalian genome editing through CRISPR-Cas systems - Discusses how to create mammalian disease models, stem cell modification, epigenetic modifications, correction of defective gene in blood disorders, heredity, neurological disorders and many more - Discusses the application of CRISPR-Cas9 systems in basic sciences, biomedicine, molecular biology, translational sciences, neurobiology, neurology, cancer, stem cells, and many more

For more than 95 years, Goldman-Cecil Medicine has been the authoritative source for internal medicine and the care of adult patients. Every chapter is written by acclaimed experts who, with the oversight of our editors, provide definitive, unbiased advice on the diagnosis and treatment of thousands of common and uncommon conditions, always guided by an understanding of the epidemiology and pathobiology, as well as the latest medical literature. But Goldman-Cecil Medicine is not just a textbook. It is designed to optimize electronic searches that will rapidly take you to exactly the information you are seeking. Throughout the lifetime of each edition, periodic updates continually include the newest information from a wide range of journals. Furthermore, Goldman-Cecil Medicine is available for all users of ClinicalKey, Elsevier's full library of subspecialty textbooks that can be accessed by readers who may want even more in-depth information. - More than 400 chapters authored by a veritable "Who's Who" of modern medicine - A practical, templated organization with an emphasis on up-to-date, evidence-based references - New chapters on Population Health, Effects of Climate Change on Health, Bradycardias, Transgender Medicine, Whipple Disease, COVID-19 Virology and Pathobiology, COVID-19 Epidemiology/Clinical Manifestations/Diagnosis/Community Prevention, COVID-19 Treatment and Vaccination, Polyomaviruses, and more - Thousands of algorithms, figures, and tables that make its information readily accessible - Over 100 supplementary videos, heart sounds, and key references - Available in print and on a variety of electronic devices - Continuously updated by Lee Goldman, MD - An eBook version is included with purchase. The eBook allows you to access all of the text, figures, and references, with the ability to search, customize your content, make notes and highlights, and have content read aloud.

About 7 million people worldwide are suffering from various inherited neuromuscular diseases. Gene therapy brings the hope of treating these diseases at their genetic roots. Muscle Gene Therapy is the only book dedicated to this topic. The first edition was published in 2010 when the field was just about to enter its prime time. The progress made since then has been unprecedented. The number of diseases that have been targeted by gene therapy has increased tremendously. The gene therapy toolbox is expanded greatly with many creative novel strategies (such as genome editing and therapy with disease-modifying genes). Most importantly, clinical benefits have begun to emerge in human patients. To reflect rapid advances in the field, we have compiled the second edition of Muscle Gene Therapy with contributions from experts that have conducted gene therapy studies either in animal models and/or in human patients. The new edition offers a much needed, up-to-date overview and perspective on the foundation and current status of neuromuscular disease gene therapy. It provides a framework to the development and regulatory approval of muscle gene therapy drugs in the upcoming years. This book is a must-have for anyone who is interested in neuromuscular disease gene therapy including those in the research arena (established investigators and trainees in the fields of clinical practice, veterinary medicine and basic biomedical sciences), funding and regulatory agencies, and patient community.