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Univ. of Pennsylvania, Philadelphia. Presents up-to-date summaries of recently completed and ongoing clinical trials, highlighting current regulatory issues, and the latest advances in the design and application of liposomes, replicating, adenoviral, adeno-associated viral vectors for study and treatment. ... Read more

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A complete introduction and guide to the latest developments in cancer gene therapy-from bench to bedside. The authors comprehensively review the anticancer genes and gene delivery methods currently available for cancer gene therapy, including the transfer of genetic material into the cancer cells, stimulation of the immune system to recognize and eliminate cancer cells, and the targeting of the nonmalignant stromal cells that support their growth. They also thoroughly examine the advantages and limitations of the different therapies and detail strategies to overcome obstacles to their clinical implementation. Topics of special interest include vector-targeting techniques, the lessons learned to date from clinical trials of cancer gene therapy, and the regulatory guidelines for future trials. Noninvasive techniques to monitor the extent of gene transfer and disease regression during the course of treatment are also discussed. ... Read more

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Every day, newspapers and television news programs present stories on the latest controversies over healthcare and medical advances, but they do not have the space to provide detailed background on the issues. Websites and weblogs provide information from activists and partisans intent on presenting their side of a story. But where can students - or even ordinary citizens - go to obtain unbiased, detailed background on the medical issues affecting their daily lives? This volume in the Health and Medical Issues Today series provides readers and researchers a balanced, in-depth introduction to the medical, scientific, legal, and cultural issues surrounding gene therapy and its import in today's world of healthcare.

Gene Therapy is organized to provide researchers with easy access to the information they need. Section 1 provides overview chapters on the background information needed to intelligently understand the issues and controversies surrounding gene therapy, such as the history of theories of the gene and recent developments in clinical trials. Section 2 offers capsule examinations of the contemporary issues and debates that provoke the most heated disagreements and misunderstandings, such as whether or not humans should tinker with genetic material and who pays for genetic therapies. Section 3 includes reference material on stem cells, including primary source documents from important players in the struggle over gene therapy, a timeline of important events, and an annotated bibliography of useful print and electronic resources. This volume in the Health and Medical Issues Today series provides everything a student requires to understand the issues involved in gene therapy and provides a springboard for further research into the issue.

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Animal Cell Technology: from Biopharmaceuticals to Gene Therapy provides a comprehensive insight into biological and engineering concepts related to mammalian and insect cell technology, as well as an overview of the applications of animal cell technology. Part 1 of the book covers the Fundamentals upon which this technology is based and covers the science underpinning the technology. Part 2 covers the Applications from the production of therapeutic proteins to gene therapy.  The authors of the chapters are internationally-recognized in the field of animal cell culture research and have extensive experience in the areas covered in their respective chapters.

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STEM CELL BIOLOGY AND GENE THERAPY

Edited by Peter J. Quesenberry, Gary S. Stein, Bernard Forget, and Sherman Weissman

Advances in molecular genetics and recombinant DNA technology have ushered in a new era in medical therapeutic research. New insights into the molecular basis of human disease and the role played by biological regulatory mechanisms have precipitated tremendous drug development efforts backed by intensive research into human gene therapy worldwide.

Stem Cell Biology and Gene Therapy is the first book to thoroughly cover major advances in the field and their applications to novel molecular therapies.

This self-contained volume integrates biological and clinical components of stem cell biology, examines some of the most difficult aspects of gene therapy, and provides a systematic review of advanced gene modification techniques. Twenty essays by leading researchers address some of the most compelling topics in contemporary medical research, including:
* Fundamental regulatory mechanisms that operate in stem cells
* Stem cells from a therapeutic perspective, including preparations of stem cells and their therapeutic potential as vehicles for gene therapy
* Delivery systems for therapeutic genes, including an overview of the most promising vectors
* Clinical applications for gene therapy, covering a broad range of diseases such as hemophilia, cancers, neurological disease, and more

Complete with illustrations and real-world examples of a variety of disorders, Stem Cell Biology and Gene Therapy is essential for researchers in gene therapy and members of the biotechnology industry who are developing human molecular therapies for commercial use. It is also an important reference for molecular biologists, cell biologists, immunologists, molecular geneticists, hematologists, cancer researchers, biochemists, and anyone working in internal medicine. ... Read more

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Researchers from academia and biotechnology describe proven molecular methods for the construction, development, and use of virus vectors for gene transfer and gene therapy. Offering detailed step-by-step instructions to ensure successful results, these experts detail the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Additional chapters demonstrate the use of virus vectors in the brain and central nervous system. Comprehensive and highly practical, Viral Vectors for Gene Therapy: Methods and Protocols provides not only researchers with the basic tools needed to design targeted gene delivery vectors, but also clinicians with an understanding of how to apply viral vectors to the treatment of genetic disorders. ... Read more

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Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight disease. Gene therapy is being studied in clinical trials for many different types of cancer and for numerous other diseases. This new book offers the latest research in a field bursting with new developments, hope and expectations. ... Read more

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The clinical applications of human gene therapy have been particularly fruitful in oncology, and in the past two decades there has been explosive growth in understanding of the genetic lesions leading to neoplasia.This volume in the series Cancer: Clinical Science in Practice reviews progress in the basic and clinical science of gene therapy in oncology, and looks toward future developments.It considers what has worked and what has not in the fast-evolving field of gene therapy, drawing on laboratory studies and clinical trials, including the ground-breaking work of the contributors themselves.Up to date and authoritative, the 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. ... Read more

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This book addresses the latest advances in viral and non-viral vectors, novel targets and methods for the treatment of autoimmune and inflammatory disease with a main focus in pre-clinical testing with up to date clinical trials. The first section of the book deals with current research approaches for treating rheumatoid arthritis, osteoarthritis, diabetes, Crohn's disease, multiple sclerosis, Sjögrens syndrome, cystic fibrosis, myositis and lupus. The second section is devoted to cell delivery and vector development including plasmids, regulated promoters, adenovirus gutted vectors and adeno-associated vectors. All chapters are summarised by leaders in these fields. A must read for post graduate students, clinical and non-clinical researchers in the disciplines of immunology, molecular medicine, pharmacology, gene and cell-mediated therapy.

Product Description
An Introduction to Molecular Medicine and Gene Therapy

Edited by Thomas F. Kresina, Ph.D. Gene therapy, or the use of genetic manipulation for disease treatment, is derived from advances in genetics, molecular biology, clinical medicine, and human genomics. Molecular medicine, the application of molecular biological techniques to disease treatment and diagnosis, is derived from the development of human organ transplantation, pharmacotherapy, and elucidation of the human genome. An Introduction to Molecular Medicine and Gene Therapy provides a basis for interpreting new clinical and basic research findings in the areas of cloning, gene transfer, and targeting; the applications of genetic medicine to clinical conditions; ethics and governmental regulations; and the burgeoning fields of genomics, biotechnology, and bioinformatics. By dividing the material into three sections - an introduction to basic science, a review of clinical applications, and a discussion of the evolving issues related to gene therapy and molecular medicine-this comprehensive manual describes the basic approaches to the broad range of actual and potential genetic-based therapies. In addition, An Introduction to Molecular Medicine and Gene Therapy:
* Covers new frontiers in gene therapy, animal models, vectors, gene targeting, and ethical/legal considerations
* Provides organ-based reviews of current studies in gene therapy for monogenetic, multifactoral or polygenic disorders, and infectious diseases
* Includes bold-faced terms, key concepts, summaries, and lists of helpful references by subject in each chapter
* Contains appendices on commercial implications and a review of the history of gene therapy

This textbook offers a clear, concise writing style, drawing upon the expertise of the authors, all renowned researchers in their respective specialties of molecular medicine. Researchers in genetics and molecular medicine will all find An Introduction to Molecular Medicine and Gene Therapy to be an essential guide to the rapidly evolving field of gene therapy and its applications in molecular medicine. ... Read more

Customer Reviews (3)

Excellent overview of a fascinating field
I did not have any special prior knowledge in biology or genetics. I needed to quickly get up to speed in this area for a professional reason. Parts of the book were a little difficult to understand at times, especially when it used many technical terms, but overall it provided me with an excellent overview of this fascinating and rapidly evolving field. I reduced one star because of not seeing enough illustrations and diagrams which in my view are essential in any text that includes complex concepts. With this book I also read "Genomes" to complement the basics of Genomics and together these two books did the trick!! Strongly reccomended to anyone who is interested in the field.

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The greatest difficulty with this field is that it is currently evolving at a rapid rate. Thus, books on the subject run the risk of being outdated before publication. However, Kresina and co-authors have done a solid job of reviewing existing methodologies, ethics and regulation of gene therapy as well as identifying some of the diseases for which this novel type of therapy could be applied. --Grant MacGregor, Emory University

An outstanding introduction
I am very impressed with the breadth of subjects that Dr. Kresina covered and how he was able to relate their associations with either molecular medicine and/or gene therapy.More importantly, he draws the two disciplines together in a logical and constructive manner.The book is not only an outstanding introduction to molecular medicine and gene therapy, but will be a valued reference guide for much of the information contained in the chapters. The topics are timely and most of the authors extremely well known and respected in their respective fields.While the second section of the book is primarily the clinical application, it draws very nicely from the more basic science introductory chapters.Chapters 13-15 and the Appendix provide a discussion of issues which undoubtedly will become significantly associated with advances in molecular medicine and gene therapy.The book is laid out in an easy-to-read format and provides ready access to a variety of different subjects.The index is inclusive and the chapters cover their individual areas in an up-to-date manner..."An Introduction..." should remain an outstanding introduction and reference text for a variety of students - from undergraduate to postdoctoral to all levels of faculty and investigators.--Clifford J. Steer, M.D., University of Minnesota Medical School ... Read more

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Despite various difficulties, the field of gene therapy, particularly with regard to cancer, has accumulated a tremendous amount of vital pre-clinical and clinical data. "Gene Therapy of Cancer: Methods and Protocols, Second Edition" fully updates the first edition with expert coverage of established and novel protocols involving both experimental and clinical approaches to cancer gene therapy. This state-of-the-art volume contains overviews of new concepts and strategies with chapters on regulatory and ethical issues, developments, problems and possible limitations of design and production of gene therapeutics as well as translational issues. Written in the highly successful Methods in Molecular Biology™ series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and notes on troubleshooting and avoiding known pitfalls.

Cutting edge and authoritative, "Gene Therapy of Cancer: Methods and Protocols, Second Edition" is an ideal guide for all those who wish to explore the fast-paced and critical study of nonviral, viral, experimental and clinical cancer gene therapy.

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Scientists are beginning to be able to treat disease by altering genes, units of inherited information that play a large part in determining how healthy a person will be. Many ethical as well as scientific questions must be answered, however, before the thrilling promise of gene therapy can be realized. (20020501) ... Read more

Product Description

Cell Therapy: cGMP Facilities and Manufacturing is the source for a complete discussion of facility design and operation with practical approaches to a variety of day-to-day activities, such as staff training and competency, cleaning procedures, and environmental monitoring. This in-depth book also includes detailed reviews of quality, the framework of regulations, and professional standards. It meets a previously unmet need for a thorough facility-focused resource, Cell Therapy: cGMP Facilities and Manufacturing will be an important addition to the cell therapy professional’s library.

Additional topics in Cell Therapy: cGMP Facilities and Manufacturing…Standard operating procedures - Supply management - Facility equipment - Product manufacturing, review, release and administration - Facility master file.

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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.

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. ... Read more

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We are standing at the threshold of a new and exciting medical era-an era of regeneration, rejuvenation, and renewal in which stems cells will set the stage for healing and in some cases, the restoration of injured, diseased, and debilitated tissues and organ. While stem cell therapy is still in its infancy, the field is rich with promise.The debate over the use of embryonic stem cells and the questionable effectiveness of adult stem cells have led many scientists and clinicians to concentrate their energies on umbilical-cord-derived stem cells from healthy newborn babies. While these cells are technically classifed as "adult stem cells," they appear to have greater restorative and regenerative potential than stem cells derived from adult tissues due to their young age.Human umbilical-cord stem cells (hUCSCs) have demonstrated great efficacy in promoting the healing of many conditions. In the last decade or so, pure cord-blood stem cells have been used by physcians to treat a mulitude of intractable diseases such as progressive multiple sclerosis, amyotrophic lateral sclerosis, certain degenerative eye disorders, stroke, diabetes, and various forms of heart disease. While certainly no cure all, umbilical-cord stem cell therapy appears to be amassing a respectable track record in terms of both safety and clinical utility.In "Umblical Cord Stem Cell Therapy: The Gift of Healing from Health Newborns", Drs. steenblock and Payne share some of the science that underlies stem cell therapy and put a human face on this field with accounts of people who have benefited from hUCSC treatments. And, in providing this information, they encourage readers to take that bold first step into this vast and wondrous medical frontier. ... Read more

Customer Reviews (14)

A true blessing!
This book was just the beginning of my journey to get umbilical cord stem cells for my son with brain injury.It helped me get educated to ask the right questions so I could make safe decisions for my son.I personally have met Dr. Steenblock.And my son did receive umbilical cord stem cells from Dr. Ramirez in Mexico.We have nothing but great things to say about them and the whole process.My son is definitely showning improvements and we are in the process of planning our next trip.Hope this helps. [...]

Thanks for bringing hope for healing
I read this book cover-to-cover in one sitting because I couldn't put it down.I happen to know that the embryonic stem cell "debate" is primarily a patenting and thus monetary issue, and not at all about what is best for mankind.If this book hadn't come about, most people would not now know about this "free" and fabulous source of regenerative medicine and healing.Drs Steenblock and Payne have done their research, their legwork, and their homework - even in the face of opposition (greed and ignorance have a tendency to cause that).I hope this is just the first in a series of books from Steenblock on regenerative therapies that lead to genuine healing for our very sick world. Judge this book for yourself, you'll be glad you did.

Well written and factual.
Stem Cells derived from Human Umbilical Cord blood are safe, ethical and have great promise to help with those disabilities, such as Cerebral Palsy, for which no cure exists today. Once I started reading this book, I couldn't put it down and am waiting for the sequel to be published!

A book I couldn't put down
I received this book as a gift and once I began reading it, could not put it down. I learned so much about stem cells and especially cord blood and cord blood stem cells, as well as how these cells are helping sick children and adults get better. It's been over 50 years since I graduated college and my science knowledge is definitely rusty, but I had no problem keeping up with everything in "Umbilical Cord Stem Cell Therapy". It is very, very well written and structured and gets to the point. Since I have an eye condition, I especially enjoyed reading how nurse Ruth got a turnaround in her diabetic retinopathy after a single cord stem cell treatment. I am now thinking of mailing a copy off to my representatives in Washington, DC along with a note urging them to put more tax dollars into research involving cord blood stem cells for macular degeneration, multiple sclerosis, cerebral palsy and traumatic brain injury (Ills covered in this fine book by Dr. Steenblock and Dr. Payne).

A joy to read
This is a smartly written book I enjoyed thoroughly. I hope the publishing house is considering putting out an expanded 2nd edition. ... Read more

Product Description
Human gene transfer is widely regarded as one of the most promising technologies for the treatment of a variety of disorders, but it presents practitioners with a variety of difficult ethical questions. Gene Transfer and the Ethics of First-in-Human Research examines the ethical and policy dimensions of testing interventions in human beings for the first time. The book discusses the difficult ethical challenges that arise from attempting to translate laboratory discoveries into clinical applications. These range from which available techniques to use, when to initiate human testing, questions of consent, expectation in public arenas, how to define acceptable risk, and the inclusion of vulnerable or disadvantaged subjects in early phase trials. This book is relevant to ethicists, legal practitioners, policy makers, geneticists and clinicians involved in clinical trials of new medical interventions. ... Read more

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The field of non-viral vector research has rapidly progressed since the publication of the first edition. This new edition is expanded to two separate volumes that contain in-depth discussions of different non-viral approaches, including cationic liposomes and polymers, naked DNA and various physical methods of delivery, as well as a comprehensive coverage of the molecular biological designs of the plasmid DNA for reduced toxicity, prolonged expression and tissue or disease specific genes. New developments such as the toxicity of the non-viral vectors and recent advances in nucleic acid therapeutics are fully covered in these volumes. ... Read more

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This book focuses on an emerging, central issue in molecular genetics and the development of eukaryotes: the control of gene expression by small species of RNA. As an exciting new field of endeavor, it is the first book by a single author to deal comprehensively with RNA silencing.

The book provides the historical background of the field preceding the seminal work by Fire and associates in 1998 on the impact of small double-stranded RNA on the expression of nematode genes, which is considered the beginning of RNA silencing research. RNA silencing is described in a wide range of plants and animals including protozoa, simple metazoa, insects, non-mammalian vertebrates, and mammals. In each case the experimental results are provided with the accompanying background and with illustrations. There is also an appendix on the prospective use of RNA silencing in gene therapy, which is intended as a guide for investigators wishing to explore this possibility. ... Read more

Customer Reviews (1)

Good literature survey
The discovery of RNA interference in molecular biology has resulted in an explosion of interest in recent years. This interest is both academic and practical, and shows promise in drug discovery as well as in gene therapy and genetic engineering. This book gives essentially a literature survey of the latest research in RNA interference or RNA silencing as it is called in the book. Readers will need a substantial background in molecular biology to follow the contents, but anyone, including non-experts (such as this reviewer) can gain much from the book if it is read carefully and with attention paid to detail. Since it is a literature survey, readers will need ready access to the references that are quoted in the book.This reviewer only read the first six chapters so only these will be subject of the commentary here.

The author begins the book by an historical summary and then in chapter 2 begins discussing RNA silencing in plants. He points to three viewpoints for studying gene silencing by nucleotide sequences, namely the silencing of invading pathogens by viruses, the co-suppression of an inserted transgene, and homology-dependent gene silencing. The chapter emphasizes the first viewpoint, wherein small RNA sequences are used to defend against invading viruses. One example discussed is the use of mutated replicase of potato virus X (PVX) to induce resistance to the wild-type virus. Other examples include the correlation of virus resistance with post transcriptional suppression of transgene mRNA accumulation in tobacco etch virus and potato virus Y. Also interesting in this chapter is the discussion on the mobility of the post transcriptional gene silencing (PTGS) signal in plants.

The author takes a look at gene silencing in fungi in chapter 3, with emphasis on the role of repeat-induced point (RIP) mutations and `quelling'. The latter is a posttranscriptional mechanism that was discovered in Neurospora crassa. The quelling involved taking part of a gene involved in carotene synthesis and transforming the fungus with the result that the transgene caused the silencing of the endogenous gene.

The nematode C. elegans is one of the most widely studied organisms in functional genomics and molecular biology and so it is not surprising to see that RNA interference was investigated in it. The author discusses this research in chapter 4 of the book. The phenomenon of RNA interference is well supported in C. elegans as a reading of this chapter will reveal. One fascinating fact that is brought out by the author regarding C. elegans is that one half of its 600 somatic cells are neurons. The author reveals that partial resistance to RNA interference occurs in the nervous system of C. elegans, particularly in its head which had full resistance to RNA interference. He reports that some researchers have found techniques to circumvent this resistance.

Chapter 5 considers RNA silencing in insects, namely in Drosophila melanogaster and in mosquitoes. The molecular biology and genetics of Drosophila is of course well known, and the author discusses in fair detail RNA interference in this organism. The double stranded RNA that represents a certain gene in Drosophila can silence this gene and in fact is more efficient than single stranded RNA. The author includes a detailed model for RNA interference in Drosophila with accompanying diagrams. For mosquitoes, the author argues that an ideal situation would be one where the double stranded RNA is used successfully namely to suppress their own pathogens but also to abolish the replication of human viruses. He does not report too many of the details of research in RNA interference in mosquitoes. The genetic engineering of mosquitoes is very exciting via the possibility of releasing transgenic mosquitoes in the wild

In chapter 6 the author discusses RNA silencing in protozoa, and reports the surprising fact that it does not occur in some protozoa. In particular there is no RNA silencing in some Trypanosoma species and species in the genus Leishmania. The lack of RNA silencing in these organisms is a counterexample to the belief that RNA silencing evolved as a mechanism for the defense against retrotransposons, argues the author. For those eukaryotes that do not have RNA silencing, it thus becomes imperative to find the mechanisms they use for coping with transposons. The discussion of RNA silencing in Trypanosomatids is interesting also in that it points to the existence of the `kinetoplast', which is a unique organelle in the mitochondrion consisting of a large DNA network embedded in which they are minicircles and macrocircles. The minicircles apparently play a role in RNA editing. Also discussed and very interesting is the phenomenon of `transsplicing' in some protozoa, and wherein alternative exons can be spliced in the same pre-mRNA. This results in different mRNA as final product. The author gives a thorough discussion of transsplicing along with accompanying diagrams. ... Read more

Product Description
Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight disease. Gene therapy is being studied in clinical trials for many different types of cancer and for numerous other diseases. The volume presents significant new research results in this promising field. ... Read more