Hemophilia gene therapy: the end of the beginning?
D De Wolf, K Singh, MK Chuah… - Human Gene …, 2023 - liebertpub.com
Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …
Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells
B Handyside, AM Ismail, L Zhang, B Yates, L **e… - Molecular Therapy, 2022 - cell.com
Recombinant adeno-associated virus (rAAV) vectors are often produced in HEK293 or
Spodoptera frugiperda (Sf)-based cell lines. We compared expression profiles of" …
Spodoptera frugiperda (Sf)-based cell lines. We compared expression profiles of" …
Long‐term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post‐treatment
Introduction Valoctocogene roxaparvovec uses an adeno‐associated virus serotype 5
(AAV5) vector to transfer a factor VIII (FVIII) coding sequence to individuals with severe …
(AAV5) vector to transfer a factor VIII (FVIII) coding sequence to individuals with severe …
Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A
Introduction Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5
(AAV5)‐mediated gene therapy approved for severe haemophilia A (HA). Aim To report the …
(AAV5)‐mediated gene therapy approved for severe haemophilia A (HA). Aim To report the …
AAV-mediated co-expression of an immunogenic transgene plus PD-L1 enables sustained expression through immunological evasion
TB McMurphy, A Park, PJ Heizer, C Bottenfield… - Scientific Reports, 2024 - nature.com
Adeno-associated virus (AAV) vectors can mediate long-term expression of immunogenic
transgenes in vivo through transduction of tolerogenic cells in the liver. Tissue-targeted AAV …
transgenes in vivo through transduction of tolerogenic cells in the liver. Tissue-targeted AAV …
The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice
Adeno-associated virus (AAV)-based vectors are used clinically for gene transfer and persist
as extrachromosomal episomes. A small fraction of vector genomes integrate into the host …
as extrachromosomal episomes. A small fraction of vector genomes integrate into the host …