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Showing 1 to 10 of 10 publications
Pub Name
Title with link
Gene Therapy
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Pub Name
Title with link
Gene Therapy
Praesent ac sem eget est egestas volutpat. Ut varius tincidunt libero. Vestibulum ante ipsum primis in faucibus orci luctus et ultrices posuere cubilia Curae; In ac dui quis mi consectetuer lacinia. Phasellus magna. Aenean posuere, tortor sed cursus feugiat, nunc augue blandit nunc, eu sollicitudin urna dolor sagittis lacus.
Pub Name
Title with link
Gene Therapy
Praesent ac sem eget est egestas volutpat. Ut varius tincidunt libero. Vestibulum ante ipsum primis in faucibus orci luctus et ultrices posuere cubilia Curae; In ac dui quis mi consectetuer lacinia. Phasellus magna. Aenean posuere, tortor sed cursus feugiat, nunc augue blandit nunc, eu sollicitudin urna dolor sagittis lacus.
Pub Name
Title with link
Gene Therapy
Praesent ac sem eget est egestas volutpat. Ut varius tincidunt libero. Vestibulum ante ipsum primis in faucibus orci luctus et ultrices posuere cubilia Curae; In ac dui quis mi consectetuer lacinia. Phasellus magna. Aenean posuere, tortor sed cursus feugiat, nunc augue blandit nunc, eu sollicitudin urna dolor sagittis lacus.
Pub Name
Title with link
Gene Therapy
Praesent ac sem eget est egestas volutpat. Ut varius tincidunt libero. Vestibulum ante ipsum primis in faucibus orci luctus et ultrices posuere cubilia Curae; In ac dui quis mi consectetuer lacinia. Phasellus magna. Aenean posuere, tortor sed cursus feugiat, nunc augue blandit nunc, eu sollicitudin urna dolor sagittis lacus.
Gene Therapy
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Gene Therapy
The evaluation demonstrated preclinical proof-of-concept of FLT190 for the treatment of Fabry disease. In vivo studies demonstrated several features of liver-directed gene transfer for Fabry disease that may have considerable therapeutic value, including α-Gal A over-expression that drives cellular uptake; stable long-term α-Gal A expression; normalization of Lyso-Gb3 storage material in plasma and key disease tissues; normalization of Gb3 storage material in plasma, heart, urine; and significant clearance of Gb3 storage material from kidney.
The Journal of Gene Medicine
Evaluation of life cycle defective adenovirus mutants for production of adeno‐associated virus vectors
The Journal of Gene Medicine
The study indicates the advantages of using a ∆pTP mutant adenovirus rather than adenovirus wild‐type as a helper virus for AAV2 production and also indicates that temperature shifts to lower temperatures may improve AAV2 vector production rates.
Molecular Genetics and Metabolism
Liver-directed gene therapy corrects Fabry disease in mice
Elsevier
We examined the therapeutic efficacy of GLA gene therapy in Fabry mice using recombinant adeno-associated virus (rAAV) vectors, directed by the liver-specific promoter (FRE1). The data resulting from this study provides strong evidence that our liver-directed AAV-mediated gene therapy approach holds considerable therapeutic potential for the treatment of Fabry disease.
Molecular Genetics and Metabolism
Liver directed AAV gene therapy to treat Gaucher disease
Elsevier
We used an adeno-associated viral vector (AAV) to drive the expression of human GBA gene in the liver of mice and observed noticeable uptake of human GCase in affected organs. Overall, the data showed that upon a single injection of a liver-expression-directed AAV vector it is possible to achieve elevation of GCase in the bloodstream that results in greater GCase bioavailability compared to current ERT.
Blood Journal
A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of more than 40% and has the Potential to Provide a Functional Cure for Patients with Haemophilia B
Blood Journal
This research was originally published in Blood. Chowdary, Pratima; Shapiro, Susie; Davidoff, Andrew M.; Reiss, Ulrike; Alade, Rebecca; Brooks, Geoff; Dane, Allison; McIntosh, Jenny; Short, Gerard; Tuddenham, Edward and Nathwani, Amit C. A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B. Blood. 2018; 132: 631. © the American Society of Hematology.
Blood Journal
Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B
Blood Journal
This research was originally published in Blood. Dane, Allison; McIntosh, Jenny; Lee, Doyoung; Sheridan, Rose; Maginn, Mark; Corbau, Romuald; Davidoff, Andrew M. and Nathwan, Amit C. Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B. Blood. 2018; 132: 2197. © the American Society of Hematology.
Blood Journal
Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
Blood Journal
This research was originally published in Blood. Kia, Azadeh; McIntosh, Jenny; Rosales, Cecilia; Hosseini, Paniz; Sheridan, Rose; Spiewak, Justyna; Mills, Kevin; Corbau, Romuald and Nathwani, Amit C. Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice. Blood. 2018; 132: 2209. © the American Society of Hematology.
The New England Journal of Medicine
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
The New England Journal of Medicine
In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months.
The New England Journal of Medicine
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
The New England Journal of Medicine
Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder.
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