Lecture 26: Using Cloned Genes for GeneTherapy

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since 4 October 1999

(version 25 September, 1999)

Some of the technical issues

1. Getting replacement gene into patient's cells.

2. Targeting the correct tissues

3. Levels of expression.

4. Replacing the defective gene early enough in development

5. Somatic versus germline gene therapy.

Early attempts at gene therapy

ADA gene therapy. Defect results in Severe Combined ImmuneDeficiency (SCID) - movie

Potential for diseases such as sickle-cell anemia, DMD (Duchennes muscular dystrophy), and CF (cystic fibrosis)

Some ethical issues with gene therapy

1. what characters should be corrected?

2. Somatic versus germ-line therapy?

MOVIE!