Gene and Cell Therapy - Therapeutic Mechanisms and Strategies

This book presents extensive background and basic information, state-of-the-art technologies, important achievements, and lingering challenges in the fields of gene and cell therapies. This edition also offers more material on the contributors' research efforts, current technologies, disease targets, and clinical applications.

List of contents

Lentiviral Vectors: Design and Applications. Adenovirus-Vector-Mediated Gene Delivery: Barriers and Chemical Vector Modifications. Helper-Dependent Adenoviral Vectors for Gene Therapy. Vaccinia Viral Vectors for Vaccines and Oncolytic Virotherapy. Oncolytic Measles Virus-Based Delivery Systems. Baculovirus: A High-Capacity Vector for Versatile Gene Transfer. Redox-Responsive Polymer-Based Gene Delivery Systems. Dendrimers, Dendrigrafts, and Their Conjugations as Delivery Vectors in Gene Therapy. Optimization of Nonviral Gene Therapeutics Using Bilamellar Invaginated Vesicles. Magnetofection: Using Magnetic Particles and Magnetic Force to Enhance and Target Nucleic Acid Delivery. Pressurized Transvenous-Retrograde Extremity Perfusion. Oncolytic Viruses for Treatment of Cancer. Baculovirus as a Gene Delivery Vector for Bone and Cartilage Tissue Engineering. Bacteria as Gene Therapy Vectors for Cancer. New Advances in Cell-Based Drug and Gene Delivery Systems. Antisense Oligonucleotide-Based Therapeutics. Development of RNAi Therapeutics. Therapeutic Potential of microRNAs. Deliberate Regulation of Therapeutic Transgenes: Recent Advances in System Development and Uses. DNA Vaccines. FANG: bi-shRNAifurin and GMCSF DNA-Augmented Autologous Tumor Cell Vaccine: Clinical Results. Biology and Immunology of Recombinant Adeno-Associated Viral Vectors in Gene Therapy. Alphavirus Vectors in Gene Therapy and Neuroscience. Gene Electrotransfer. Evolution of Polyplexes: Refining Structural Motifs Required for Nucleic Acid Delivery. Therapeutic Applications of Morpholino Oligonucleotides. Nonviral Genome Modification Strategies for Gene Therapy: Transposon, Integrase, and Nuclease Systems. Complex In Vitro Models and Their Application in Gene Editing Technologies. Gene Therapy for Hematopoietic Disorders. Gene Therapy for Vision Disorders. Molecular Therapy for Type 1 and Type 2 Diabetes. Alipogene Tiparvovec: Gene Therapy for Familial Lipoprotein Lipase Deficiency. Gene Therapy for Neurological Diseases. Gene Therapy for Alzheimer's Disease. Gene Therapy for the Treatment of Cancer: From Laboratory to Bedside. Gene Therapy for the Treatment of Lung Cancer. RNA Interference-Based Gene Therapy Strategies for the Treatment of HIV Infection. Pluripotent Stem Cells and Their Therapeutic Applications. Directing Pluripotent Human Embryonic Stem Cells toward Lineage-Specific Cell Therapy Derivatives for Regenerative Medicine. Therapies with Adipose Tissue-Derived Stem/Stromal Cells. Placenta-Derived Stem Cells and Their Therapeutic Applications. Cell Based Therapies from Umbilical Cord Blood. Cell Therapies for the Treatment of Inborn Metabolic Errors. Cell Therapies for Lung Disease. Cell Therapies for Gastro-Intestinal Disease. Cell Therapies for Genitourinary Disorders. Cell Therapy for the Creation of Cartilage and Related Clinical Trials (online only). Translating Stem Cell-Derived Extracellular Vesicle Therapy for Brain Disorders. CARtalysts of Change: CAR T Cells at the Intersection of Engineering and Clinical Practice. Gene and Cell Therapies for Muscular Dystrophies. Prenatal Cell and Gene Therapy for Genetic Disorders. Cell and Gene Therapies for Cardiovascular Disease. Cell and Gene Therapies for the Treatment of Hearing Disorders. Cutaneous Cell- and Gene-Based Therapies for Inherited and Acquired Skin Disorders. Monitoring Gene and Cell Therapies in Living Subjects with Molecular Imaging Technologies.

About the author

Dr. Graça Almeida-Porada earned her MD and her PhD from School of Medicine and Biomedical Sciences at the University of Porto, Portugal. She is currently a Professor and the Director of the Fetal Research and Therapy Program at the Wake Forest Institute for Regenerative Medicine. She is also a Professor in the Department of Internal Medicine, Section of Hematology and Oncology, and School of Biomedical Engineering and Sciences, Wake Forest School of Medicine.
Dr. Almeida-Porada's research focuses on the development of cellular and gene delivery platforms to treat genetic and immune-mediated diseases. She is particularly interested in improving the outcome of stem cell transplantation and gene therapy in fetal and neonatal patients with genetic disorders, and in developing therapies for children with immune-mediated diseases.
She is the co-founder of the International Fetal Transplantation and Immunology Society, whose mission is to accelerate clinical applications of stem cell transplantation and gene therapy approaches to treat fetuses with congenital disorders.
Dr. Almeida-Porada serves on numerous review panels, study sections, and professional and scientific committees, and she is a reviewer for many peer-reviewed journals. She has been a member of several NIH study sections, and she serves as an Editor, or on the Editorial Boards, of multiple scientific journals and book series. Dr. Almeida-Porada also serves on the scientific advisory boards for several ongoing prenatal clinical trials, both in the US and in Europe.
Dr. Almeida-Porada holds several patents, and she has authored more than 300 scientific works including papers, abstracts, and book chapters.
Dr. Anthony Atala is a practicing surgeon and the G. Link Professor and Director of the Wake Forest Institute for Regenerative Medicine. His work focuses on bioprinting and growing human cells, tissues and organs. Seventeen applications of technologies developed in Dr. Atala's laboratory have been used clinically. Dr. Atala was elected to the National Academy of Medicine, the National Academy of Inventors, and the American Institute for Medical and Biological Engineering. He is a recipient of the US Congress funded Christopher Columbus Foundation Award, bestowed on a living American who is currently working on a discovery that will significantly affect society, the World Technology Award in Health and Medicine, the Edison Science/Medical Award, the R&D Innovator of the Year Award, and the Smithsonian Ingenuity Award. Dr. Atala's work was listed twice as Time Magazine's top 10 medical breakthroughs of the year, as one of 5 discoveries that will change the future of organ transplants and was ranked by the Project Management Institute as one of the top 10 most impactful biotech projects from the past 50 years. Dr. Atala was named by Scientific American as one of the world's most influential people in biotechnology, by U.S. News & World Report as one of 14 Pioneers of Medical Progress in the 21st Century, by Life Sciences Intellectual Property Review as one of 50 key influencers in the life sciences intellectual property arena, and by the journal Nature Biotechnology as one of the top 10 translational researchers in the world. Dr. Atala has led or served several national professional and government committees, including the National Institutes of Health working group on Cells and Developmental Biology, the National Institutes of Health Bioengineering Consortium, and the National Cancer Institute's Advisory Board. He was a Founder of the Tissue Engineering Society, the Regenerative Medicine Society, the Regenerative Medicine Foundation, the Alliance for Regenerative Medicine, the Regenerative Medicine Development Organization, the Regenerative Medicine Manufacturing Society, and the Regenerative Medicine Manufacturing Consortium. Dr. Atala is editor of 25 books and 2 journals, has published more than 900 journal articles, and has applied for or received over 300 national and international patents.

Product details

• Authors: Graca Atala Almeida-Porada
• Assisted by: Almeida-Porada Graca (Editor), Atala Anthony (Editor)
• Publisher: Taylor & Francis Ltd.
• Languages: English
• Product format: Hardback
• Release: 12.09.2025
• EAN: 9780367771799
• ISBN: 978-0-367-77179-9
• No. of pages: 1360
• Subjects: Natural sciences, medicine, IT, technology > Medicine > Non-clinical medicine; SCIENCE / Life Sciences / General, SCIENCE / Life Sciences / Molecular Biology, SCIENCE / Life Sciences / Cell Biology, SCIENCE / Life Sciences / Genetics & Genomics, molecular biology, Cellular biology (cytology), Genetics (non-medical)