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Alternative Strategies for Gene Therapy of Hemophilia

Hemophilia A and B are monogenic disorders that were felt to be ideal targets for initiation of gene therapy. Although the first hemophilia gene therapy trial has been over 10 years ago, few trials are currently actively recruiting. Although preclinical studies in animals were promising, levels achi...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: Montgomery, Robert R., Shi, Qizhen
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2010
Gaiak:
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC3383974/
https://ncbi.nlm.nih.gov/pubmed/21239794
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/asheducation-2010.1.197
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