Development of a gene targeting strategy for liver metabolic diseases using the Crispr/Cas9 platform
Many inborn errors of metabolism require life-long treatments and, in severe conditions, organ transplantation remains the only curative treatment. Non-integrative AAV-mediated gene therapy in the liver has shown to be efficient in different clinical trials of adult patients, but treatment in pediat...
| Main Author: | |
|---|---|
| Format: | Doctoral Thesis |
| Language: | English |
| Published: |
Università degli studi di Trento
2021
|
| Online Access: | http://hdl.handle.net/11572/324574 |