Mechanistic Insights into Factor VIII Immune Tolerance Induction via Prenatal Cell Therapy in Hemophilia A
Sign Up to like & getrecommendations! Published in 2019 at "Current Stem Cell Reports"
DOI: 10.1007/s40778-019-00165-y
Abstract: Prenatal stem cell and gene therapy approaches are among the few therapies that can promise the birth of a healthy infant with specific known genetic diseases. This review describes fetal immune cell signaling and its… read more here.
Keywords: tolerance; therapy hemophilia; cell; prenatal cell ... See more keywords
Gene therapy for hemophilia: what does the future hold?
Sign Up to like & getrecommendations! Published in 2018 at "Therapeutic Advances in Hematology"
DOI: 10.1177/2040620718791933
Abstract: Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have reported remarkable success in ameliorating disease phenotype in hemophilia A and B. These trials, which highlight the challenges overcome through decades of preclinical and… read more here.
Keywords: therapy hemophilia; future hold; gene therapy; hemophilia future ... See more keywords
The effectiveness and value of gene therapy for hemophilia: A Summary from the Institute for Clinical and Economic Review's California Technology Assessment Forum.
Sign Up to like & getrecommendations! Published in 2023 at "Journal of managed care & specialty pharmacy"
DOI: 10.18553/jmcp.2023.29.5.576
Abstract: DISCLOSURES: Dr Tice and Mr Sarker received ICER grants during the conduct of the study. Dr Moradi, Ms Herce-Hagiwara, Dr Faghim, Dr Agboola, Dr Rind, and Dr Pearson reports grants from Arnold Ventures, grants from… read more here.
Keywords: therapy hemophilia; health; hemophilia summary; effectiveness value ... See more keywords
Development of an AAV DNA-based synthetic vector for the potential gene therapy of hemophilia in children
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Microbiology"
DOI: 10.3389/fmicb.2022.1033615
Abstract: Recombinant AAV serotype vectors and their variants have been or are currently being used for gene therapy for hemophilia in several phase I/II/III clinical trials in humans. However, none of these trials have included children… read more here.
Keywords: therapy hemophilia; aav; gene therapy; vector ... See more keywords