𝔖 Bobbio Scriptorium
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Gene therapy for rare diseases – the case for acute intermittent Porphyria

✍ Scribed by Grosios, Konstantina

Book ID
125404101
Publisher
BioMed Central
Year
2014
Tongue
English
Weight
79 KB
Volume
5
Category
Article
ISSN
1878-5077

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## Abstract ## Background Acute intermittent porphyria (AIP) is an autosomal dominant disorder caused by the half‐normal activity of hydroxymethylbilane synthase (HMB‐synthase). Affected individuals can experience episodic, life‐threatening, acute neurological attacks that are precipitated by vari