Medical genetics : a legal frontier / by Lori B. Andrews.

  • Lori Andrews
Date:
1987
Catalogue details

Licence: Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0)

Credit: Medical genetics : a legal frontier / by Lori B. Andrews. Source: Wellcome Collection.

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    278 I. Description of Proposal A. Objectives and rationale of the proposed research State concisely the overall objectives and rationale of the propsed study. Please provide information on the following specific points; 1. Why is the disease selected for treatment by means of gene therapy a good candidate for such treatment? 2. Describe the natural history and range of expression of the disease selected for treatment What objective and/or quantitative measures of disease activity are available? In your view, are the usual effects of the disease predictable enough to allow for meaningful assessment of the results of gene therapy? 3. Is the protocol designed to prevent all manifestations of the disease, to halt the progression of the disease after symptoms have begun to appear, or to reverse manifestations of the disease in seriously ill victims? 4. What alternative therapies exist? In what groups of patients are these therapies effective? What are their relative advantages and disadvantages as compared with the proposed gene therapy? B. Research design, anticipated risks and benefits 1. Structure and characteristics of the biological system Provide a full description of the methods and reagents to be employed for gene delivery and the rationale for their use. The following are specific points to be addressed; a. What is the structure of the cloned DNA that will be used? (1) Describe the gene (genomic or cDNA), the bacterial plas- mid or phage vector, and the delivery vector (if any). Pro¬ vide complete nucleotide sequence analysis or a detailed restriction enzyme map of the total construct. (2) What regulatory elements does the construct contain (e.g., promo¬ ters, enhancers, polyadenylation sites, replication origins, etc.)? (3) Describe the steps used to derive the DNA construct b. What is the structure of the material that will be administered to the patient? ( 1 ) Describe the preparation, structure, and composition of the materials that will be given to the patient or used to treat the patient's cells. (a) If DNA, what is the purity (both in terms of being a single DNA species and in terms of other contain¬ ments)? What tests have been used and what is the sen¬ sitivity of the tests? (b) If a virus, how is it prepared from the DNA construct? In what cell is the virus grown (any special features)? What medium and serum are used? How is the virus purified? What is its structure and purity? What steps
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