Autologous And Cancer Stem Cell Gene Therapy

Stem cells provide for life-long cell replacement in tissues and organs, and have inherent homing abilities that are critical in therapeutic applications. Stem cells are also the driving force of cancer where genetic/epigenetic alterations culminate in tumorigenesis either in tissue stem cells or in some of their derivatives. As a rare subset of the tumor, cancer stem cells are the only drive of tumor initiation/propagation. Autologous and cancer stem cells are thus the key targets of 1) long-term and transient-regenerative/epigenetic gene therapy and 2) of recurrence-free anticancer therapy, respectively. While cancer stem cell gene therapy still needs time to accomplish, autologous stem cells have been instrumental in the first unequivocal successes for gene therapy whereby ex vivo retrovirally corrected hematopoietic stem cells have been returned to the patients. This timely book presents 1) the aforementioned stem cell gene therapy achievements that rely on random-integration of therapeutic transgenes into host chromosomes and 2) emerging experimental approaches aimed at eliminating random-integration oncogenic hazards through site-specific integration or gene targeting. Breakthrough endonuclease-boosted gene targeting for gene correction (inherited diseases) or targeted integration of therapeutic transgenes (other disorders) culminating in an efficiency compatible with clinical trials is one of the highlights of the book. Highlights also include the pioneering transplantation of adult pluripotent stem cells as a substitute for tissue-specific stem cells, thereby pinpointing the invaluable potential for stem cell gene therapy applications of autologous cells able to contribute to all three germ layers. Stem cell gene therapy is thus discussed in terms of 1) magnifying stem cell therapeutic homing through transient regenerative gene therapy and 2) of tackling most pathologies (including mitochondrial DNA diseases and ageing disorders) through stem cell repopulation dynamics into appropriate niches (long-term engraftment) and tissues (cell turn-over). Regarding cancer stem cell gene therapy, focus is on both the increasing number of identified tissue-specific cancer stem cells as the ultimate therapeutic targets and on the development of armed stem cells as tumor-homing vectors for targeted anticancer therapy. Sample Chapter(s). Introduction: Toward a Universal Platform for Autologous Stem Cell Gene Therapy (335 KB). Chapter 1: Stem Cell Gene Therapy for ADA-Deficiency without Myelopreparative Conditioning (318 KB). Contents: Introduction: Toward a Universal Platform for Autologous Stem Cell Gene Therapy; Long-term Stem Cell Gene Therapy: From Current Clinical Random-Integration Achievements to Stem Cell Gene Targeting/Cybridization: From Pioneering Clinical Retroviral Gene Therapy to Experimental Site-Specific Integrative Gene Therapy; Stem Cell Gene Targeting/Cybridization: Toward Endonuclease-Boosted Gene Repair/Alteration, Custom Site-Specific Integrative Gene Therapy and Transmitochondrial Therapy/Rejuvenation; Adult Pluripotent Stem Cells: Emerging Stem Cell/Gene Therapy Breakthrough; Cancer Stem Cell Gene Therapy: Cancer Stem Cells as Breakthrough Targets of Cancer Gene Therapy; Armed Stem Cells as Tumor-Homing Vectors for Cancer Gene Therapy. Readership: Clinicians, scientists, physicians, veterinarians, dentists, pharmacists/chemists, interested in an ongoing medical/veterinarian breakthrough that is expected to revolutionize the practice of Medicine/Dentistry and that relies on cell biology, molecular biology/genetics, developmental biology, biochemistry, molecular pathology and animal science.

Cell therapy is a rapidly developing area, drawing on cell biology, molecular biology, virology, immunology, cell quantitation techniques and biomedical engineering. It has potential in many clinical settings, in the treatment of cancer and other diseases. This volume in the series Cancer: Clinical Science in Practice examines the current state and future prospects of cell therapy, which seems likely to have an even more profound impact on health care than did the production of proteins by recombinant DNA technology. The coverage is broad, including the scientific principles of haematopoietic cell therapy, the technology of cell collection and preparation, current and likely future clinical applications of cell therapy, and the principles and practice of cellular immunotherapy. Up-to-date and authoritative, volumes in this series are intended for a wide audience of clinicians and researchers with an interest in the applications of biomedical science to the understanding and management of cancer.

The first unequivocal success for Gene Therapy was reported in April 2000 for X-SCID patients. Pioneering stem cell/gene therapy clinical trials are the focus of this book. Therapy successes such as the X-SCID trial and improved ADA-SCID ones are presented together with pioneering angio/vasculogenic clinical trials mediated either by transient gene

Much research has focused on the basic cellular and molecular biological aspects of stem cells. Much of this research has been fueled by their potential for use in regenerative medicine applications, which has in turn spurred growing numbers of translational and clinical studies. However, more work is needed if the potential is to be realized for improvement of the lives and well-being of patients with numerous diseases and conditions. This book series 'Cell Biology and Translational Medicine (CBTMED)' as part of SpringerNature’s longstanding and very successful Advances in Experimental Medicine and Biology book series, has the goal to accelerate advances by timely information exchange. Emerging areas of regenerative medicine and translational aspects of stem cells are covered in each volume. Outstanding researchers are recruited to highlight developments and remaining challenges in both the basic research and clinical arenas. This current book is the seventh volume of a continuing series. Chapter “Application of iPSC to Modelling of Respiratory Diseases” is available open access under a Creative Commons Attribution 4.0 International License via link.springer.com.

Among the many applications of stem cell research are nervous system diseases, diabetes, heart disease, auto-immune diseases as well as Parkinson's disease, end-stage kidney disease, liver failure, cancer, spinal cord injury, multiple sclerosis, Parkinson's disease, and Alzheimer's disease. Stem cells are self-renewing, unspecialised cells that can give rise to multiple types all of specialised cells of the body. Stem cell research also involves complex ethical and legal considerations since they involve adult, foetal tissue and embryonic sources. This new book brings together leading research from throughout the world in this frontier field.

Organ transplantation has been the most important therapeutic advance in the last third of the 20th century. Its development has revolutionized medicine, as demonstrated by the fact that a large number of researchers in this field have been awarded Nobel Prizes. In the beginning of this century, we are witnessing with great expectations the emergence of a new field of medicine related to the arrival of a new player on the scene: “stem cells” and their potential use in regenerative medicine. This volume aims to cover important aspects of the various facets of organ transplantation and regenerative medicine, with leading specialists in these fields setting out their vision. We try to rigorously explain current and novel scientific research in these fields—areas which arouse great interest from society in general, due to their potential use in modern medicine for the treatment of a great number of diseases.

Regenerative medicine – stem cell and gene-based therapy – offers a new approach for restoring function of damaged organs and tissues. This is the first book to cover the major new aspects and field of regenerative medicine. This title is therefore a timely addition to the literature. It brings together the major approaches to regenerative medicine in one text, which ensures that techniques learnt in one discipline are disseminated across other areas of medicine.