Journal
HUMAN MOLECULAR GENETICS
Volume 25, Issue -, Pages R36-R41Publisher
OXFORD UNIV PRESS
DOI: 10.1093/hmg/ddv475
Keywords
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Funding
- Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia
- Howard Hughes Medical Institute
- US National Institutes of Health [HL64190, HL078810, HV78203]
- Spark Therapeutics, Inc.
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Gene transfer studies for the treatment of hemophilia began more than two decades ago. A large body of pre-clinical work evaluated a variety of vectors and target tissues, but by the start of the new millennium it became evident that adeno-associated viral (AAV)-mediated gene transfer to the liver held great promise as a therapeutic tool. The transition to the clinical arena uncovered a number of unforeseen challenges, mainly in the form of a human-specific immune response against the vector that poses a significant limitation in the application of this technology. While the full nature of this response has not been elucidated, long-term expression of therapeutic levels of factor IX is already a reality for a small number of patients. Extending this success to a greater number of hemophilia B patients remains a major goal of the field, as well as translating this strategy to clinical therapy for hemophilia A. This review summarizes the progress of AAV-mediated gene therapy for the hemophilias, along with its upcoming prospects and challenges.
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