AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. - Wikidata - wikimedia - TriplyDB

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.

http://www.wikidata.org/entity/Q35153203

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Clinical development of gene therapy: results and lessons from recent successes Emerging and future therapies for hemophilia Current animal models of hemophilia: the state of the art Gene therapy in an era of emerging treatment options for hemophilia B Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models Profile of efraloctocog alfa and its potential in the treatment of hemophilia A.Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A.Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models.Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.Obstacles and future of gene therapy for hemophilia.New approaches to gene and cell therapy for hemophilia.In vivo induction of regulatory T cells for immune tolerance in hemophilia.New and improved AAVenues: current status of hemophilia B gene therapy.Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies.Gene therapy for immune tolerance induction in hemophilia with inhibitors.Specific factor IX mRNA and protein features favor drug-induced readthrough over recurrent nonsense mutations.Complexity of immune responses to AAV transgene products - Example of factor IX.In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.Dynamics of antigen presentation to transgene product-specific CD4+ T cells and of Treg induction upon hepatic AAV gene transfer.Moving forward toward a cure for hemophilia B.Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD/SCIDγc-/- mice.A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice.Partnering to change the world for people with haemophilia: 7th Haemophilia Global Summit, Madrid, Spain 22-24 September 2016.Novel approaches to hemophilia therapy: successes and challenges.Gene Therapy for Hemophilia: Progress to Date.Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.Emerging Issues in AAV-Mediated In Vivo Gene Therapy.Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy.Combination Therapy Is the New Gene Therapy?Gene Therapy With Regulatory T Cells: A Beneficial Alliance.Emerging therapies for hemophilia: controversies and unanswered questions.Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX.Gene therapy for hemophilia: what does the future hold?

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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.

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2015年の論文

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AAV liver expression of FIX-Pa ...... in hemophilia B dogs and mice.

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Clinton D Lothrop

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Federico Mingozzi

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Glenn P Niemeyer

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Jonathan D Finn

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Joshua I Siner

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Julie M Crudele

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Nicholas B Martin

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Shangzhen Zhou

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Valder R Arruda

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P2860

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

A zymogen-like factor Xa variant corrects the coagulation defect in hemophilia

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

Canine hemophilia B resulting from a point mutation with unusual consequences

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy.

Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Role of IFN-gamma in induction of Foxp3 and conversion of CD4+ CD25- T cells to CD4+ Tregs.

Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.

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