By Daniel Scherman

This certain complicated textbook presents a transparent and finished description of the sphere of gene supply, gene treatment and genetic pharmacology, with descriptions of the most gene move vectors and a collection of chosen healing functions, in addition to security concerns. using gene move is exponentially starting to be within the clinical and scientific groups for daily mobile biology experiments and fast improvement of progressive gene treatment techniques. during this complex textbook, greater than 25 top scientists, world-renowned of their respective fields, come jointly to supply a transparent and accomplished description of gene supply, gene treatment and genetic pharmacology. This academic creation to the most gene move vectors and chosen healing purposes presents the heritage fabric had to extra discover the topic in addition to suitable examine literature. it's going to therefore be valuable to grasp, PhD or MD scholars, post-doctoral scientists or doctors, in addition to any scientist wishing to bring a gene or man made nucleotide, or boost a gene treatment procedure. moreover, the textbook's easy and artificial content material may be of price to any reader drawn to the organic and clinical revolution derived from the elucidation of the human genome.

Readership: grasp, PhD or MD scholars, post-doctoral scientists or docs, and any scientists utilizing gene move suggestions or enforcing gene remedy thoughts.

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Additional info for Advanced Textbook on Gene Transer, Gene Therapy and Genetic Pharmacology: Principles, Delivery and Pharmacological and Biomedical Applications of Nucleotide-Based Therapies

Sample text

This concept has been approached experimentally in the mdx mouse model, in which it has been possible to study the effect on muscle function of a whole series of deletions of increasing size. These studies indicated that not every part of the dystrophin protein is necessary for its function, leading to a therapeutic approach by modification of RNA splicing. indd 24 11/8/2013 7:18:26 AM B1574 Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology History of Gene Therapy „ 25 been envisaged.

We will begin this section with a point of nomenclature. Within a DNA gene, two strands can be defined. indd 35 11/8/2013 12:05:29 PM B1574 Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology 36 „ Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology nucleotide information for the amino acid sequence of the protein and is therefore called the sense strand. The second strand is complementary to the sense strand and is referred to as the antisense strand.

This drawback turns out to be of interest for non-genetic disorders, through the skipping of necessary exons of, for instance, oncogenes or viral proteins. Antisense oligonucleotides will most certainly give rise to a whole new class of drugs. Antisense therapy is not strictly a form of gene therapy (except under the form of viral transduction), but is a related, genetically mediated therapy. More details on exon skipping are given in Chapters 6 and 24. 5 Concluding Remarks Gene therapy has gone through several ten-year cycles.

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