Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins
Author:
Publisher
Proceedings of the National Academy of Sciences
Subject
Multidisciplinary
Reference18 articles.
1. Unfolding the role of protein misfolding in neurodegenerative diseases
2. Adeno-associated virus vectors: potential applications for cancer gene therapy
3. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
4. Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2α1-Antitrypsin (AAT) Vector in AAT-Deficient Adults
5. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors
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