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Polyymer Based Gene Therapy For Lung Cystic Fibrosis Disease
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Book Synopsis Gene Therapy for Diseases of the Lung by : Kenneth Brigham
Download or read book Gene Therapy for Diseases of the Lung written by Kenneth Brigham and published by CRC Press. This book was released on 2020-10-14 with total page 460 pages. Available in PDF, EPUB and Kindle. Book excerpt: This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems.
Book Synopsis Gene Therapy in Lung Disease by : Steven M. Albeda
Download or read book Gene Therapy in Lung Disease written by Steven M. Albeda and published by CRC Press. This book was released on 2002-09-20 with total page 582 pages. Available in PDF, EPUB and Kindle. Book excerpt: Presents up-to-date summaries of recently completed and ongoing clinical trials. With writings from more than 35 internationally renowned experts, Gene Therapy in Lung Disease unlocks the biological mysteries of infection immunity cytokine behavior fibrosis and illustrates the use of gene the
Book Synopsis Polyymer-based Gene Therapy for Lung Cystic Fibrosis Disease by : Bei Qiu
Download or read book Polyymer-based Gene Therapy for Lung Cystic Fibrosis Disease written by Bei Qiu and published by . This book was released on 2024 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Molecular Basis and Gene Therapies of Cystic Fibrosis by : John Engelhardt
Download or read book Molecular Basis and Gene Therapies of Cystic Fibrosis written by John Engelhardt and published by MDPI. This book was released on 2020-12-11 with total page 208 pages. Available in PDF, EPUB and Kindle. Book excerpt: Summary of Genes. Thirty years ago, the gene responsible for cystic fibrosis (CF), a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator gene, was identified. This progress has considerably changed our understanding of the pathophysiology of CF and has paved the way for the development of novel and specific therapies for the disease. The CFTR gene contains 27 exons and is characterized by a frequent three base pair deletion of the p.Phe508del. As a result of collaborative work, today more than 2000 mutations have been reported in the gene, and their impact on protein function is now more evident and useful in designing new strategies to correct the gene defect. The field of gene therapy, as illustrated by Ziying Yan in this book, has worked on identifying an efficient vector system for the delivery of the wild-type CFTR gene to the lung. At the same time, animal models have been developed in mice, rats, rabbits, zebrafish, ferrets, and pigs to establish the efficacity of gene delivery. These animals are also of the utmost importance in testing new molecules as modulators or correctors to improve the CFTR lung function. During the last three decades, the epidemiology of CF has dramatically changed, as today cystic fibrosis is now a chronic adult pulmonary disease.
Book Synopsis Cystic Fibrosis Gene Therapy by : Karlea Lee Kremer
Download or read book Cystic Fibrosis Gene Therapy written by Karlea Lee Kremer and published by . This book was released on 2010 with total page 544 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy potentially holds the key for the treatment and cure of many genetic diseases, including cystic fibrosis. A number of delivery methods have been developed for the integration of a functional gene into the host genome, one of which is the use of a HIV-1 derived lentivirus, as is used in this thesis. However, a large number of issues need to be addressed before an effective gene therapy protocol can be developed, and some of these are described further in this thesis. One such issue is that the response initiated by cells to the gene transfer vector need to be addressed, as organelles such as the proteasome and lysosome that break down foreign peptides and proteins may be involved in the degradation of our gene transfer vector, ultimately limiting the amount of gene transfer vector that is able to successfully integrate into the genome. Therefore, the potential use of proteasome and lysosome inhibitors for facilitating higher levels of gene transduction in vivo was investigated. As this project uses a HIV-1 derived lentivirus for gene transfer, the use of an inhibitor of the IN1/PML innate antiretroviral response (Leptomycin B) was also assessed, again with the aim of increasing the efficiency, and hence level, of gene transfer obtained. Using a robust animal model of disease is essential for testing lentivirus constructs containing the therapeutic gene and analysing phenotypic changes in disease. A mouse model of cystic fibrosis without gastrointestinal disease was bred to obtain a robust colony of mice that efficiently produce affected mice (CFTR knockout). Visual analysis of therapeutic gene transfer in cystic fibrosis is often difficult due to the lack of antibodies available. Short DNA molecules that adopt a specific 3-D shape known as aptamers hold much potential as agents that can be developed to bind to the CFTR gene product. These can then be labelled and used in the same way as antibodies to probe tissues excised from animals treated with the therapeutic CFTR gene. Essential to gene therapy is the development of methods for the consistent determination of lentivirus titre. As the production of lentivirus becomes more sophisticated with the use of multiple transgenes in a single virus preparation, the need for multiple assays to determine the titres of each individual virus component are required. Real time PCR assays were developed for each individual transgene for titre determination. A real time PCR assay for use with CHOK-1 cells was also developed for comparison of real time PCR titre of a LacZ virus to the titre obtained using the traditional LacZ titre achieved via a staining assay in CHOK-1 cells. The use of a standard real time PCR assay for the determination of titre for all viruses- containing any transgene is essential to allow comparison by titre. Determination of the level of gene expression required to achieve a therapeutic outcome in a cystic fibrosis mouse model is an important factor to consider, as high level expression in all cells may not achieve the best outcomes, and low level, ciliated-cell specific expression may in fact achieve a superior result. A range of different strength and cell targeted promoters (EF1[alpha], pgk and K18) were tested for their effect on phenotypic correction of the cystic fibrosis knockout mouse model. Lastly, targeting therapeutics to the disease affected areas is essential in achieving the best patient outcomes. As the main target organ in cystic fibrosis are the conducting airways of the lungs, delivery of our gene transfer vectors to the lungs as an aerosol is a necessary step towards moving this gene therapy protocol to the clinic. Aerosolisation of the treatment protocol was investigated in the rat lung for evidence of whether this is a viable means of lentiviral mediated gene delivery to the airways.
Book Synopsis Immunopharmacology by : Manzoor M. Khan
Download or read book Immunopharmacology written by Manzoor M. Khan and published by Springer Science & Business Media. This book was released on 2008-12-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.
Book Synopsis Advanced Gene Delivery by : Alain Rolland
Download or read book Advanced Gene Delivery written by Alain Rolland and published by CRC Press. This book was released on 2003-09-02 with total page 300 pages. Available in PDF, EPUB and Kindle. Book excerpt: A practical resource for everyone involved in the gene therapy field and in the design of effective gene delivery systems, this volume presents an overview and update of recent advances in the field of non-viral methods for the in vivo transfer of therapeutic genes to biological targets using conventional routes of administration. Methods to control the spatial and temporal modulation of gene function in vivo as well as the level, duration, specificity, and fidelity of gene expression are described. The rational design and the applications of a variety of non-viral gene delivery systems, such as cationic lipid-, polymer-, and (poly) peptide-based systems, are exemplified for the control of location of therapeutic genes administered by various routes. Current and potential clinical applications of gene-based medicines are presented for the prevention, correction or modulation of diseases. Examples of current applications of plasmid-based systems for genetic vaccination, treatment of genetic disorders such as cystic fibrosis, and treatment of acquired diseases such as cancer are also provided.
Book Synopsis Molecular Basis and Gene Therapies of Cystic Fibrosis by : John Engelhardt
Download or read book Molecular Basis and Gene Therapies of Cystic Fibrosis written by John Engelhardt and published by . This book was released on 2020 with total page 210 pages. Available in PDF, EPUB and Kindle. Book excerpt: Of Genes. Thirty years ago, the gene responsible for cystic fibrosis (CF), a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator gene, was identified. This progress has considerably changed our understanding of the pathophysiology of CF and has paved the way for the development of novel and specific therapies for the disease. The CFTR gene contains 27 exons and is characterized by a frequent three base pair deletion of the p.Phe508del. As a result of collaborative work, today more than 2000 mutations have been reported in the gene, and their impact on protein function is now more evident and useful in designing new strategies to correct the gene defect. The field of gene therapy, as illustrated by Ziying Yan in this book, has worked on identifying an efficient vector system for the delivery of the wild-type CFTR gene to the lung. At the same time, animal models have been developed in mice, rats, rabbits, zebrafish, ferrets, and pigs to establish the efficacity of gene delivery. These animals are also of the utmost importance in testing new molecules as modulators or correctors to improve the CFTR lung function. During the last three decades, the epidemiology of CF has dramatically changed, as today cystic fibrosis is now a chronic adult pulmonary disease.
Book Synopsis Adenoviral Vectors for Gene Therapy by : David T. Curiel
Download or read book Adenoviral Vectors for Gene Therapy written by David T. Curiel and published by Academic Press. This book was released on 2016-03-10 with total page 870 pages. Available in PDF, EPUB and Kindle. Book excerpt: Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. - Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors - Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement - Demonstrates noninvasive imaging of adenovirus-mediated gene transfer - Discusses utility of adenoviral vectors in animal disease models - Considers Federal Drug Administration regulations for human clinical trials
Book Synopsis Gene Therapy for Cystic Fibrosis by : Gustavo Puras
Download or read book Gene Therapy for Cystic Fibrosis written by Gustavo Puras and published by . This book was released on 2018 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cystic fibrosis (CF) is a genetic disease that hampers the lung function. Despite that the main defective gene has been deeply characterized, some relevant concerns still need to be resolved before considering gene therapy as a realistic medical choice. One of the major issues that need to be strongly considered in order to succeed in the search for an effective gene therapy approach for CF is the design of the appropriate genetic material to be delivered. Other relevant factors to take into consideration include the design of safe and effective gene delivery systems, the biological barriers that need to be overcome in order to reach the nucleus of the target cells, and the problems related to the design of a drug formulation suitable for lung delivery purposes. Furthermore, some problems related to the commercialization of gene therapy products also need to be resolved. In this chapter, we discuss the up-to-date strategies to overcome such hurdles in order for gene therapy to become a routine treatment modality for CF.
Book Synopsis Investigation and Application of Novel Adeno-associated Viral Vectors for Cystic Fibrosis Gene Therapy by : Benjamin Richard Steines
Download or read book Investigation and Application of Novel Adeno-associated Viral Vectors for Cystic Fibrosis Gene Therapy written by Benjamin Richard Steines and published by . This book was released on 2015 with total page 103 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cystic Fibrosis (CF) is a lethal autosomal recessive genetic disorder caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR transports anions at the apical surface of epithelial membranes and functions in many areas of the body. However in CF, loss of CFTR function in the lungs is the major source of morbidity and mortality. Replacing the defective CFTR in the lungs through gene therapy has the potential to cure the disease. Recombinant adeno-associated virus (AAV) is an effective gene transfer vector and has been used extensively to deliver genes to cells in culture. A number of clinical trials using AAV have been attempted for a variety of diseases, including CF, albeit with limited success. Poor vector transduction efficiency prevents effective gene therapy. We have previously used a technique to greatly increase the transduction efficiency of AAV in human lung tissues by selecting from a library of AAVs using a directed evolution technique. However, this evolution was performed in cultured cells and did not fully represent the in vivo environment in which the AAV would be used. In 2008, a CF pig model was developed to develop a further understanding of the mechanisms of CF and CFTR function. We hypothesized that we could use directed evolution to select for a vector in vivo using the pig, allowing gene therapy studies to be conducted in a physiologically relevant model of CF. We selected a novel AAV variant, called AAV2H22, which is closely related to AAV2 but with greatly increased transduction efficiency in pig airway epithelia. AAV2H22 displayed specific tropism for pig airway epithelia and saturated cell surface receptors, indicating specific binding in those cells. We found that AAV2H22-mediated gene transfer corrected chloride and bicarbonate transport defects both in vitro and in vivo. Importantly, bicarbonate transport was sufficient to normalize pH in the airway surface liquid, resulting in increased bacterial killing likely due to increased activity of antimicrobial peptides. To investigate the mechanics of the increased transduction of AAV2H22, capsid mutants were assayed for transduction efficiency. Two of the five amino acid differences between AAV2 and AAV2H22 lie at the surface and are predicted to alter capsid binding. This is consistent with the results showing specific binding in cultured airway epithelia. This research has important implications for gene therapy and investigations using AAV2H22 will increase our understanding of the biology needed to successfully treat CF.
Book Synopsis Pharmaceutical Biotechnology by : Daan J. A. Crommelin
Download or read book Pharmaceutical Biotechnology written by Daan J. A. Crommelin and published by CRC Press. This book was released on 2002-11-14 with total page 456 pages. Available in PDF, EPUB and Kindle. Book excerpt: The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.
Book Synopsis Understanding Gene Therapy by : Prof Nick Lemoine
Download or read book Understanding Gene Therapy written by Prof Nick Lemoine and published by Taylor & Francis. This book was released on 2023-05-09 with total page 142 pages. Available in PDF, EPUB and Kindle. Book excerpt: This title explains what gene therapy is, how genes are delivered and how they are targeted. It discusses recent gene therapy trials, future applications and considers the ethical and safety issues surrounding gene therapy. Understanding Gene Therapy is a
Book Synopsis Nanotechnology for Nucleic Acid Delivery by : Manfred Ogris
Download or read book Nanotechnology for Nucleic Acid Delivery written by Manfred Ogris and published by Humana Press. This book was released on 2012-10-16 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Nanotechnology and nucleic acid based therapies are two emerging fields in science whose combination has the potential to improve quality of life for patients suffering from various diseases that can so far only be treated in an unsatisfactory way. Nucleic acids offer the potential for highly selective treatment of such diseases or the highly specific modulation of gene expression with RNA interference. A key issue for successful nucleic acid therapies is the availability of a suitable delivery system. Here, the field of nanotechnology offers a multitude of possibilities to develop nanosized delivery vectors tailor-made for various local and systemic approaches. In Nanotechnology for Nucleic Acid Delivery: Methods and Protocols, experts in the field cover the area of nanoparticulate delivery of nucleic acids in terms of biosafety, particle synthesis as well as its application in cell culture. Written in the successful Methods in Molecular BiologyTM 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. Authoritative and easily accessible, Nanotechnology for Nucleic Acid Delivery: Methods and Protocols seeks to serve both professionals and novices with its well-honed methodologies.
Book Synopsis Polymeric Gene Delivery Systems by : Yiyun Cheng
Download or read book Polymeric Gene Delivery Systems written by Yiyun Cheng and published by Springer. This book was released on 2018-09-04 with total page 361 pages. Available in PDF, EPUB and Kindle. Book excerpt: The series Topics in Current Chemistry Collections presents critical reviews from the journal Topics in Current Chemistry organized in topical volumes. The scope of coverage is all areas of chemical science including the interfaces with related disciplines such as biology, medicine and materials science. The goal of each thematic volume is to give the non-specialist reader, whether in academia or industry, a comprehensive insight into an area where new research is emerging which is of interest to a larger scientific audience. Each review within the volume critically surveys one aspect of that topic and places it within the context of the volume as a whole. The most significant developments of the last 5 to 10 years are presented using selected examples to illustrate the principles discussed. The coverage is not intended to be an exhaustive summary of the field or include large quantities of data, but should rather be conceptual, concentrating on the methodological thinking that will allow the non-specialist reader to understand the information presented. Contributions also offer an outlook on potential future developments in the field. The chapter "Polymeric Nanoparticle-Mediated Gene Delivery for Lung Cancer Treatment" is available open access under a Creative Commons Attribution 4.0 International License via link.springer.com.
Book Synopsis Mammalian Artificial Chromosomes by : Vittorio Sgaramella
Download or read book Mammalian Artificial Chromosomes written by Vittorio Sgaramella and published by Springer Science & Business Media. This book was released on 2008-02-02 with total page 277 pages. Available in PDF, EPUB and Kindle. Book excerpt: In 1996, we organized a workshop, inter alia, at the National Research Co- cil in Milan under the generous sponsorship of the European Science Foun- tion. On that occasion, a small group of investigators convened from many countries and presented early evidence of the possibility of assembling basic units of mammalian chromosomes into artificial constructs (or, indeed, red- ing the relevant components to more manageable dimensions and defined c- stitution). Progress in the following years has been slow but steady. Many scientists who took part in the workshop have since been engaged in active and prod- tive research. It goes to the credit of Humana Press to have realized the need for a book on artificial chromosomes that aims to provide better tools to all scientists committed to this field who are confronted with very difficult tech- cal problems. We have strived to cover in Mammalian Artificial Chromosomes: Methods and Protocols all relevant areas of artificial chromosome research, from basic genetics to daring attempts to build new tools for genetic therapy. We are of course grateful to the authors who have accepted the task of describing the technical steps and pitfalls that can be encountered in their research. Rarely has a very delicate methodology been presented with such meticulous care. We have been helped in this enterprise by the excellent librarian of the LITA Institute in Segrate, Italy, Ms. Claudia Piergigli, whom we thank warmly. Ms.
Book Synopsis A Guide to Human Gene Therapy by : Roland W. Herzog
Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne
