An Introduction to Molecular Medicine and Gene Therapy by Thomas F. Kresina

By Thomas F. Kresina

Gene treatment, or using genetic manipulation for ailment therapy, is derived from advances in genetics, molecular biology, scientific medication, and human genomics. Molecular medication, the appliance of molecular organic options to ailment therapy and prognosis, is derived from the improvement of human organ transplantation, pharmacotherapy, and elucidation of the human genome. An Introduction to Molecular medication and Gene Therapy offers a foundation for reading new scientific and simple study findings within the parts of cloning, gene move, and focusing on; the functions of genetic medication to medical stipulations; ethics and governmental laws; and the burgeoning fields of genomics, biotechnology, and bioinformatics. via dividing the fabric into 3 sections - an creation to simple technology, a evaluation of medical purposes, and a dialogue of the evolving concerns with regards to gene treatment and molecular medicine-this accomplished handbook describes the elemental techniques to the extensive diversity of exact and power genetic-based treatments.

In addition, An advent to Molecular medication and Gene Therapy:

  • Covers new frontiers in gene remedy, animal versions, vectors, gene concentrating on, and ethical/legal considerations
  • Provides organ-based stories of present stories in gene remedy for monogenetic, multifactoral or polygenic issues, and infectious diseases
  • Includes bold-faced phrases, key options, summaries, and lists of priceless references through topic in each one chapter
  • Contains appendices on advertisement implications and a assessment of the background of gene therapy

This textbook bargains a transparent, concise writing type, drawing upon the services of the authors, all well known researchers of their respective specialties of molecular drugs. Researchers in genetics and molecular drugs will all locate An Introduction to Molecular drugs and Gene Therapy to be a necessary consultant to the quickly evolving box of gene treatment and its functions in molecular medicine.

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They result from a complex series of events involving changes in the level of expression of many genes and/or environmental factors and behavior. While many individual interventions may be partially effective at treating complex diseases, the greatest benefits are likely to be derived from combination therapies. 1a. Such strategies, for example, have been used to introduce a specific gene into the liver cells of patients with familial hypercholesterolemia (see Chapters 6 and 7). But, it is estimated that only 2% of human diseases are thought to be caused by direct one-to-one Mendelian expression of a single gene.

There is a broad array of diseases in which specific protocols of gene therapy could provide novel therapeutic approaches. 1). They consist of chromosomal disorders that are inherited as a single gene, Mendelian disorder (autosomal dominant, autosomal recessive, sex-linked recessive, or sex-linked dominant), and result from a mutation at a single locus. These compare to the multifactorially inherited disorders that involve multiple genes working in concert with known or enigmatic environmental factors.

In a recent clinical trial performed after successful preclinical studies, gene therapy was shown to provide full correction of a disease phenotype in two patients with severe combined immunodeficiency-X1 (SCID-X1). The data presented showed clinical benefit for a ten month follow-up period. For long-term data on clinical benefit, await further follow-up of this study. Research efforts are needed to develop new vectors for gene transfer, to improve current viral and nonviral vectors, and to enhance genomic technology.

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