By Mauro Giacca (auth.)
This e-book offers to the reader a accomplished and unique, but simply available, description of the entire box of gene treatment. It covers themes starting from vector improvement to the result of the latest medical trials. It comprises descriptions of lentiviral and AAV vector improvement, of healing gene choice (including siRNAs, shRNAs, and microRNAs), and of the newest scientific functions of gene remedy for illnesses of the attention, the cardiovascular procedure, and the primary frightened procedure. Of word, the e-book in basic terms considers stipulations for which move of genetic fabric has already stepped forward to scientific purposes or is with regards to doing so. The language is apparent and, each time attainable, non-technical. because the ebook is meant to be a textbook within the box of gene remedy in either the elemental technology and medical fields, every time technical descriptions are required those are supplied. Readers will comprise graduate scholars within the lifestyles sciences zone of their ultimate years of analysis, PhD scholars, and docs focusing on assorted fields of inner and expert medication, as well as study scientists operating in numerous parts of present biomedical research.
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Extra info for Gene Therapy
Example text
Although each of these bonds is non-covalent and thus weak, their overall number renders the overall affinity between two complementary DNA strands very high: the Kd of double-stranded DNA is in the order of 1×10–15 mol/l, far higher than that, for example, of an antibody for its ligand (Kd between 22 Therapeutic Nucleic Acids 2 Fig. 4 Therapeutic nucleic acids. The site of action of the various nucleic acid-based therapeutics is shown along the pathway of gene expression 1×10–6 and 1×10–10) or a growth factor for its receptor (Kd between 1×10–8 and 5×10–10).
In addition, the regulator often consists of two different proteins, as in the case of the ecdysone- or rapamycin-regulated systems. 2 Non-Coding Nucleic Acids 21 cloning of large DNA inserts. In addition, the regulatory proteins must be expressed in a constitutive manner; however, these proteins derive from different organisms (for example, the tetracycline repressor from E. coli, the VP16 transactivator from HSV-1, the EcR from D. melanogaster, and so on) and are thus potentially immunogenic.
In most cases, however, this is not possible, since natural promoters are usually very large and thus cannot be accommodated in most viral vectors. 1 Tissue-Specific Promoters The first strategy to tackle the issue of regulated transgene expression is to limit its transcription to the tissue of interest, using a promoter specific for this tissue and sufficiently short to be cloned in a gene transfer vector. Examples of such transcriptional elements are the muscle creatine-kinase (MCK) enhancer or the β-actin gene promoter for the skeletal muscle, the α-myosin heavy chain gene promoter (α-MHC) for the heart, the insulin gene promoter for the pancreas, the albumin gene promoter for the liver, the transthyretin gene promoter for the retina, and so on.