Abstract
This chapter describes the history and development of gene-based therapies for alpha-1-antitrypsin deficiency (AATD), starting with the general principles of gene therapy, advantages, and disadvantages of vector types, and progressing to results of animal and human studies. It includes consideration of the problems encountered in current human studies and ways in which these might be overcome, learning from the lessons of gene therapy for other conditions, such as cystic fibrosis. Examples of such issues are immune reaction against the vector, and optimal route of delivery. Future developments are also discussed, such as gene silencing/editing and modification of pathology by targeting genes other than SERPINA1.
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