The New Biology set consists of the following six volumes: The Cell,
Animal Cloning, Stem Cell Research, Gene Therapy, Cancer, and Aging.
The set is intended primarily for middle and high school students, but
it is also appropriate for first-year university students and the general
public. In writing this set, I have tried to balance the need for a comprehensive
presentation of the material, covering many complex fields,
against the danger of burying—and thereby losing—young students
under a mountain of detail.
Harrison's Internal Medicine Chapter 65. Gene Therapy in Clinical Medicine
Gene Therapy in Clinical Medicine: Introduction
Gene transfer is a novel area of therapeutics in which the active agent is a nucleic acid sequence rather than a protein or small molecule. Because delivery of naked DNA or RNA to a cell is an inefficient process, most gene transfer is carried out using a vector, or gene delivery vehicle.
In the last 10 years gene therapy has experienced a renascence thanks to the development of
safer and more efficient gene transfer vectors and to the advances in the cell therapy field.
This book brings together a comprehensive collection of gene therapy tools and their thera‐
peutic applications. The first part of the book covers different gene therapy vectors focusing
on their advantages and disadvantages.
Other Diseases The power and versatility of gene transfer approaches are such that there are few serious disease entities for which gene transfer therapies are not under development. Besides those already discussed, other areas of interest include gene therapies for HIV and for neurodegenerative disorders.
Indications in gene therapy clinical trials. The chart divides clinical gene transfer studies by disease classification. A majority of trials have addressed cancer, with monogenic disorders and cardiovascular diseases the next largest
categories. (Reproduced with permission from J Gene Med. New Jersey, Wiley, 2006.)
Gene Transfer for Genetic Disease
Gene transfer strategies for genetic disease generally involve gene addition therapy. This approach most commonly involves transfer of the missing gene to a physiologically relevant target cell. ...
The general meaning of gene therapy is to correct defective genes that are responsible
for disease development. The most common form of gene therapy involves the
insertion, alteration or removal of genes within an individual's cells and biological
tissues. Many of gene transfer vectors are modified viruses. The ability for the delivery
of therapeutic genes made them desirable for engineering virus vector systems.
Recently, the viral vectors in laboratory and clinical use have been based on RNA and
DNA viruses processing very different genomic structures and host ranges.
In the first section of this book, ‘Retroviral Vector’, chapter one discusses the efficiency
of retroviral DNA integration, the preferences of integration for certain regions, and
advances on integration site selection and gene therapy. Chapter two reviews and
discusses the current cell lines and bioreaction platforms used for production of
retroviral and lentiviral vectors, focusing on the current bottlenecks and future
directions with a particular emphasis in the metabolic constrains.
Long-Term Expression in Genetic Disease: In Vivo Gene Transfer with Recombinant Adeno-Associated Viral (AAV) Vectors
Recombinant AAV vectors have emerged as attractive gene delivery vehicles for genetic disease. Engineered from a small replication-defective DNA virus, they are devoid of viral coding sequences and trigger very little immune response in experimental animals. They are capable of transducing nondividing target cells, and the donated DNA is stabilized primarily in an episomal form, thus minimizing risks associated with insertional mutagenesis.
The molecular definition of tumor antigens, costimulatory signals, and the
possibility to genetically engineer tumor cells as well as simple protocols for
efficient isolation and preparation of dendritic cells (DC) renew the interest in
tumor immunotherapy and vaccination, in particular. Engineering of tumor
cells with the gene of a particular cytokine is a way of releasing that cytokine at
the tumor site. In contrast to bolus administration, it provides a constant supply
Gene therapy has the potential to be a tailor-made therapeutic with increased specificity and decreased side effects that can offer a "cure" for many disorders. The aim of this book is to provide up-to-date reviews of the rapidly growing field of gene therapy. Chapters cover a large range of topics including methods of gene delivery, and identification of targets with several papers on cancer gene therapy.
The research field of gene therapy and the clinical practice of medicine evolving
from the research are fast moving, ever-changing disciplines. On an almost daily
basis, there appears in the media a “breaking story” of a gene-based research finding.
An implication of the story is that this research breakthrough will speedily transform,
in the next few years, into a marvelous new therapy in molecular genetic
Another local approach uses adenoviral-mediated expression of the tumor suppressor p53, which is mutated in a wide variety of cancers. This strategy has shown complete and partial responses in squamous cell carcinoma of the head and neck, esophageal cancer, and non-small cell lung cancer after direct intratumoral injection of the vector. Response rates (~15%) are comparable to those of other single agents.
Modification of the
surface of these vectors is a key element for their successful research and clinical use.
Chapter three discusses the methods to modify surfaces of retroviral vectors, and the
applications for surface modification of retroviral vectors, such as targeting and
immune modulation. Chapter four reviews the role of the nuclear glucocorticoid
receptor in controlling retroviral infection and function, and highlights its potential
importance in retroviral-based gene therapy applications....
According to the most recent updates, retroviral and lentiviral vectors represent 23% of all
the vector types and 33% of the viral vectors used in Gene Therapy clinical trials. Moreover,
retroviral vectors are currently the blockbuster vectors for the treatment of monogenic and
infectious diseases and gene marking clinical trials (Edelstein 2010).
Retroviruses are double stranded RNA enveloped viruses mainly characterized by the
ability to “reverse-transcribe” their genome from RNA to DNA.
Tuyển tập báo cáo các nghiên cứu khoa học quốc tế ngành y học dành cho các bạn tham khảo đề tài: Stable replication of the EBNA1/OriP-mediated baculovirus vector and its application to anti-HCV gene therapy
Tuyển tập báo cáo các nghiên cứu khoa học quốc tế ngành hóa học dành cho các bạn yêu hóa học tham khảo đề tài: Anti-viral state segregates two molecular phenotypes of pancreatic adenocarcinoma: potential relevance for adenoviral gene therapy
Tuyển tập báo cáo các nghiên cứu khoa học quốc tế ngành hóa học dành cho các bạn yêu hóa học tham khảo đề tài: Strategy Escalation: An emerging paradigm for safe clinical development of T cell gene therapies
Richard Paul Junghan
Tuyển tập báo cáo các nghiên cứu khoa học quốc tế ngành hóa học dành cho các bạn yêu hóa học tham khảo đề tài: Alpha-1 antitrypsin protein and gene therapies decrease autoimmunity and delay arthritis development in mouse model