Gene therapy: a) what disease is it used for? b) what genetic defect causes the disease? c) what and how viral vector is used AND/OR CRISPR-Cas is used?

Biology (MindTap Course List)
11th Edition
ISBN:9781337392938
Author:Eldra Solomon, Charles Martin, Diana W. Martin, Linda R. Berg
Publisher:Eldra Solomon, Charles Martin, Diana W. Martin, Linda R. Berg
Chapter16: Human Genetics And The Human Genome
Section16.4: Gene Therapy
Problem 1C
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Gene therapy:

a) what disease is it used for?

b) what genetic defect causes the disease?

c) what and how viral vector is used AND/OR CRISPR-Cas is used?

d) what is in the horizen re. development of new virus-based gene delivery vesicle?

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