Genetic Basis of Haemophilia

Abstruct ………………………………………………………………………………………………………………………… 3
Introduction……………………………………………………………………………………………………………………. 4
Genetic Basis of Haemophilia ………………………………………………………………………………………….. 5
Success and Failures in the Application of Gene Therapy to Haemophilia in Studies using Nonhuman
Subjects………………………………………………………………………………………………………………. 6
Retroviral vector gene delivery strategies…………………………………………………………………………… 6
Adenoviral vectors gene delivery strategies ……………………………………………………………………… 10
Adeno-associated virus (AAV) vector gene delivery studies………………………………………………. 12
Non-viral vector gene delivery methods…………………………………………………………………………… 13
Recent Progresses and Roadblocks in the Application of Gene Therapy in the Treatment of
Haemophilia in Humans ………………………………………………………………………………………………… 14
Adenoviral vectors delivery……………………………………………………………………………………………. 14
Retroviral delivery ………………………………………………………………………………………………………… 15
Adeno-associated vectors delivery ………………………………………………………………………………….. 16
Opportunities for Future Improvement of Gene Therapy to Haemophilia …………………………….. 18
Conclusion …………………………………………………………………………………………………………………… 22
References …
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