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.
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.
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.
Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue
Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: The use of retroviral vectors for gene therapy-what are the risks? A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery
Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: Improved gene delivery to human saphenous vein cells and tissue using a peptide-modified adenoviral vector
Lorraine M Work1, Paul N Reynolds2 and Andrew H
Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: In vivo gene targeting of IL-3 into immature hematopoietic cells through CD117 receptor mediated antibody gene delivery
Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice
Tuyển tập các báo cáo nghiên cứu về y học được đăng trên tạp chí y học Wertheim cung cấp cho các bạn kiến thức về ngành y đề tài: Substitution of the Rev-response element in an HIV-1-based gene delivery system with that of SIVmac239 allows efficient delivery of Rev M10 into T-lymphocytes...
NovelN,N¢-diacyl-1,3-diaminopropyl-2-carbamoyl bivalent cationic lipids
were synthesized and their physicochemical properties in lamellar assemblies
with and without plasmid DNA were evaluated to elucidate the structural
requirements of these double-chained pH-sensitive surfactants for potent
non-viral gene delivery and expression.
Certain natural peptides and proteins of mammalian origin are able to bind
and condense plasmid DNA, a prerequisite for the formation of transfec-tion-competent complexes that facilitate nonviral gene delivery. Here we
have generated recombinant derivatives of the human high-mobility group
(HMG) protein HMGB2 and investigated their potential as novel protein-based transfection reagents.
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. These vehicles have generally been engineered from viruses by deleting some or all of the viral genome and replacing it with the therapeutic gene of interest under the control of a suitable promoter (Table 65-1). ...
Malonyl-CoA, a potent inhibitor of carnitine pamitoyl
transferase-I (CPT-I), plays a pivotal role in fuel selection in
cardiac muscle. Malonyl-CoA decarboxylase (MCD) cata-lyzes the degradation of malonyl-CoA, removes a potent
allosteric inhibition on CPT-I and thereby increases fatty
acid oxidation in the heart. Although MCDhas several Ser/
Thr phosphorylation sites, whether it is regulated by AMP-activated protein kinase (AMPK) has been controversial.
We therefore overexpressed MCD(Ad.MCD) and consti-tutively active AMPK (Ad.
This contribution book collects reviews and original articles from eminent experts working in the interdisciplinary arena of novel drug delivery systems and their uses. From their direct and recent experience, the readers can achieve a wide vision on the new and ongoing potentialities of different drug delivery systems. Since the advent of analytical techniques and capabilities to measure particle sizes in nanometer ranges, there has been tremendous interest in the use of nanoparticles for more efficient methods of drug delivery.
Synthetic polymers fulfill many functions in biotechnology and medicine. In
cell culture technology and tissue engineering they provide the surfaces to
which cells may attach. Cross-linked polymer networks are used for drug
delivery and cell encapsulation. Polymer-based porous membranes can be used to
shield implanted cells from the immune system of the host, while allowing for the
exchange of nutrients and metabolic waste products thus keeping the cells alive
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.
PHI has often financed the delivery of larger treatment volumes by offering higher payments to
providers. Financial incentives linked to payment mechanisms exert a direct impact upon doctors’
productivity.32 This has contributed to a growth in the volumes of private hospital treatments in several
countries where doctors have both public and private sector engagements, as in Australia and Ireland
(Colombo and Tapay, 2003 and 2004b).