Year: 2003
Type: article
Abstract: The results of Zhang and colleagues provide further support for the notion that gene therapy might be used for the induction of immune tolerance to clotting factors.However, this study also poses several questions that remain to be addressed, including whether certain anatomic sites of transgene expression preferentially result in tolerance induction, 5 and why results using a similar neonatal retroviral delivery protocol for factor VIII resulted in a 50% incidence of inhibitor development.6 Despite repeated challenges to the progress of gene therapy over the past few years, preclinical progress in the treatment of hemophilia using this therapeutic modality continues to be very encouraging.There remains optimism that this will eventually be converted to clinical success.
Source: Blood
Author: Diane C. Arthur
Institution: National Cancer Institute
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: bronze
OpenAlex ID: W2034125527